PDUFA IV Information Technology Plan

**DRAFT**

[1.0 Introduction 3](#introduction)

[2.0 Purpose 3](#purpose)

[3.0 Vision 5](#vision)

[4.0 Goals and Objectives 5](#goals-and-objectives)

[4.1 Department Goals 5](#department-goals)

[4.2 FDA Strategic Goals and Objectives
5](#fda-strategic-goals-and-objectives)

[4.2.2 Information Management/Information Technology Strategy
6](#information-managementinformation-technology-strategy)

[5.0 PDUFA IV IT Strategy 6](#pdufa-iv-it-strategy)

[5.1 Business Modernization and Transformation
8](#business-modernization-and-transformation)

[5.1.1 Business Process Improvement 9](#business-process-improvement)

[5.2 Target Architecture 9](#target-architecture)

[5.3 Guidance, Policy and Regulation
12](#guidance-policy-and-regulation)

[5.4 Data Standards 14](#data-standards)

[6.0 Programs 19](#programs)

[6.1 Pre-Market Activities 19](#pre-market-activities)

[6.2 Post-Market Activities 26](#post-market-activities)

[7.0 Appendices 29](#appendices)

[7.1 PDUFA IV Metrics 29](#pdufa-iv-metrics)

[7.2 PDUFA Information Management/IT Goals and Objectives
30](#pdufa-information-managementit-goals-and-objectives)

[7.3 PDUFA IV Goals Mapped to FDA Initiatives
32](#pdufa-iv-goals-mapped-to-fda-initiatives)

[7.4 Business Review Boards 5-year Goals, Priorities and Current
Projects
34](#business-review-boards-5-year-goals-priorities-and-current-projects)

[7.5 Summary Schedule 35](#summary-schedule)

[7.6 Acronym List 36](#acronym-list)

#   {#section .unnumbered}

# 1.0 Introduction

As a part of the Department of Health and Human Services (DHHS), the
Food and Drug Administration's (FDA's) mission is to advance the public
health by helping to speed innovations that make products more
effective, safer, and more affordable, and to monitor products for
continued safety after they are in use. Decisions made by the FDA affect
every single American every day. Consumers spend more than 20 percent of
all consumer expenditures on FDA regulated products. Operating as a
modern, scientifically up-to-date, responsive, and efficient Agency, the
FDA can provide better protection for consumers and more effectively
promote their health.

In the last decade, the FDA has achieved great success in reforming and
modernizing its regulatory processes and responsibilities as a result of
changes and improvements driven by the requirements of the Prescription
Drug User Fee Act (PDUFA), the 1997 FDA Modernization Act (FDAMA), and
other legislation. The additional resources provided by user fees, when
combined with appropriations, have enabled the FDA to modernize its
information technology infrastructure and begin a monumental
transformation from a paper-based to an electronic work environment.
With the reauthorization of PDUFA, the FDA plans to make even greater
progress during the PDUFA IV timeframe (FY2008 -- FY2012), building on
the foundation established in previous years.

The Prescription Drug User Fee Act, or as it is commonly called, PDUFA,
allows the Agency to help fund the review of new human drugs through
fees paid by the sponsors/applicants that develop and market human drugs
and therapeutic biologics. PDUFA was first enacted in 1992, and has been
reauthorized, each time for five years, in 1997, 2002, and 2007. The
drugs user fee program was reauthorized by the Food and Drug
Administration Modernization Act of 1997, by the Public Health Security
and Bioterrorism Preparedness and Response Act of 2002, and recently by
the FDA Amendments Act of 2007.

PDUFA authorizes the FDA to collect fees from companies that produce
certain human drug and biologic products. To market a new drug or
biologic, a company must submit an application along with a fee. In
addition, companies are assessed annual fees for each prescription drug
product marketed and for each manufacturing location of the product.
Under PDUFA, industry provides funding that is added to the FDA\'s
appropriated budget, and the FDA commits to certain performance goals.
More information on the PDUFA program and performance goals is available
at <http://www.fda.gov/oc/pdufa/>.

The PDUFA III re-authorization included the Electronic Applications and
Submission Goals that included FDA's commitment to implement the
electronic Common Technical Document (eCTD) and a common solution for
the secure exchange of content including secure email and electronic
submissions. The FDA met these requirements by implementing a review
system for the evaluation of submissions in the eCTD format
<http://www.fda.gov/cder/regulatory/ersr/ectd.htm> and the
implementation of the Electronic Submissions Gateway (ESG)
<http://www.fda.gov/esg/>. In addition, the FDA implemented the first
phase of the electronic labeling rule in the Center for Drug Evaluation
and Research (CDER) that will be expanded to the Center for Biologics
Evaluation and Research (CBER)
<http://www.fda.gov/oc/datacouncil/spl.html>.

The PDUFA IV agreement builds upon the progress made in PDUFA III and
will commit the FDA to develop, implement, and maintain new information
systems consistently across all organizational divisions participating
in the process for human drug review throughout the product lifecycle.
To help meet this goal, there is an ongoing effort to document the
business processes in CBER and CDER, building upon the FDA Business
Process Framework developed in 2004 and updated in 2006. As part of the
PDUFA IV commitment, the FDA is publishing this PDUFA Information
Technology (IT) Plan for comment to allow the public to provide feedback
as the FDA moves towards a fully electronic standards-based submission
and review environment. At the end of the comment period the FDA will
review the comments, update the plan, and publish the final version no
later than May 30, 2008.

# 2.0 Purpose

This Plan demonstrates how the FDA will improve the automation of
business processes and acquire and maintain information systems to
achieve the objectives defined in the PDUFA IV Commitment Letter
transmitted from the Secretary of Health and Human Services to Congress
<http://www.fda.gov/oc/pdufa4/pdufa4goals.html> . This plan also
provides a future-state vision for the FDA standards and technical
infrastructure supporting the process for the review of human drugs
throughout the product lifecycle. Specifically, this Plan details how
the FDA intends to:

-   strengthen and improve information management within the new drug
    and biologic products review processes;

-   strengthen the IT infrastructure to improve capacity for post market
    safety data management and analysis;

-   improve the FDA's ability to communicate, share, and disseminate
    information more clearly within the Agency and with other government
    organizations, the regulated industry, and the American Public; and

-   seek more efficient and effective means for supplying technology
    tools and services to the FDA user community.

This plan will help guide the direction and implementation of IT
projects initiated to meet Agency program objectives and specific PDUFA
IV IT goals. Among the principal IT planning documents to be developed
by the Agency during the PDUFA IV timeframe, this plan will be the
mechanism to communicate the steps the FDA plans to take to achieve its
objectives to stakeholders, both internal and external to the Agency.

The CDER and the CBER have collaborated with the Office of the Chief
Information Officer (OCIO) and the Office of Planning (OPL) in the
Office of the Commissioner (OC) to develop this FDA PDUFA IV Information
Technology Plan. Together, these offices will address a key objective of
PDUFA IV: applying technology to the FDA regulatory review process in
the most efficient and effective way possible to ensure reviewers have
the information and tools that will allow them to make more informed and
timely decisions.

The FDA considers the first year of the PDUFA IV timeframe to be a
period of considerable transition. The Agency must resolve many
near-term planning activities and strategic investment decisions prior
to committing resources to future, long-range systems development plans
for the out years of PDUFA IV. For example, due to a variety of external
pressures, the FDA is conducting studies to determine a strategy for
modernizing IT infrastructure and services. Similarly, the FDA is
working to shift its IT decision-making and governance to an
Agency-wide, less de-centralized model. This governance structure will
institute an enterprise approach to automating common or special purpose
IT solutions by defining roadmaps for each business process area that
will be further refined into discrete IT solutions. Further, the FDA
must resolve technical and policy issues in order to establish standard,
Agency-wide solutions for secure exchange of information with Industry.
In the first 12 to 24 months of PDUFA IV, the FDA will focus on
completing these plans to ensure that they are developed, published, and
widely understood. Once these foundational plans are implemented, the
FDA will be in a position to expand planning of specific systems
development and infrastructure projects into the PDUFA IV out-years.

Therefore, the purpose of this document is to communicate the FDA's
long-range goals under PDUFA IV, and to present tactical strategies for
accomplishing near-term objectives toward those goals. The intent of
this plan is to:

-   communicate the link between IT efforts and the expected business
    outcomes and benefits;

-   communicate vision and strategies the FDA will follow for meeting
    the goals and objectives;

-   ensure the FDA's ability to baseline plans and measure future
    progress;

-   communicate the framework that governs PDUFA IV IT decision-making;

-   provide an understanding for how this plan links to other Agency and
    Departmental planning documents; and

-   track progress using objective measures.

The purpose of this draft plan is to solicit comments from the public.
The FDA will review and analyze the comments, make revisions to the IT
Plan, and complete and publish the final version of the IT Plan no later
than May 30, 2008. The Plan will be revised periodically over the course
of PDUFA IV as strategies and approaches are defined and clarified.

The FDA will conduct an annual assessment of progress against the plan
and publish on the FDA website a summary of the assessment within two
months after the close of each fiscal year. Updates to the plan will be
published as the FDA deems necessary to achieve the objectives defined
in PDUFA Information Technology Goals. The FDA will publish on its web
site draft revisions to the IT plan, solicit comments from the public on
those draft revisions, and consider the public comments before
completing and publishing updates to the IT plan.

# 3.0 Vision 

The FDA is committed to achieve the long-term goal of an automated
standards-based information technology environment for the exchange,
review, and management of information supporting the process for the
review of human drug applications throughout the product lifecycle. The
FDA vision is a fully electronic submission and review environment of
all regulatory documents and data; and the elimination of future
paper-based submissions. While FDA does not expect to completely achieve
this vision during the PDUFA IV timeframe, meeting the PDUFA IV
Information Technology commitments will allow the Agency and regulated
stakeholders to make tremendous progress towards implementing the
vision.

# 4.0 Goals and Objectives

This section presents the strategic goals and objectives of the various
governing layers within which FDA operates. First, it presents the
goals, objectives, and strategic planning progress of the Department of
Health and Human Services. FDA Agency level goals and objectives, under
the leadership of the FDA Commissioner, are then presented. Next,
specific information management/information technology goals and
objectives for the FDA are presented. It is important to understand how
the PDUFA Program, and in particular, the PDUFA Information Technology
Goals are linked to the HHS and FDA strategic goals. Accomplishment of
these goals will be critical to the success of the Agency and
Departmental goals.

## 4.1 Department Goals

The Department of Health and Human Services recently published its
Strategic Plan for FY 2007 -- 2012. Complete details can be found at the
following link: <http://www.hhs.gov/strategic_plan/>. FDA directly
supports 3 of the 4 HHS strategic goals:

> **Goal 1**: Improve the safety, quality, affordability and
> accessibility of health care, including behavioral health care and
> long-term care.
>
> **Goal 2**: Prevent and control disease, injury, illness and
> disability across the lifespan, and protect the public from
> infectious, occupational, environmental and terrorist threats.
>
> **Goal 4**: Advance scientific and biomedical research and development
> related to health and human services.

## 4.2 FDA Strategic Goals and Objectives

> The FDA published its most recent Strategic Action Plan in the Fall of
> 2007, (<http://www.fda.gov/ope/stratplan07/stratplan07.htm>). FDA's
> strategic goals and objectives address the entire life cycle of
> FDA-regulated products. Information management is an important theme
> that cuts across numerous goals and objectives.
>
> **Goal 1**: Strengthen FDA for Today and Tomorrow

-   Strengthen the scientific foundation of FDA's regulatory mission.

-   Cultivate a culture that promotes transparency, effective teamwork,
    and mutual respect, and ensures integrity and accountability in
    regulatory decision making.

-   Enhance partnerships and communications.

-   Strengthen FDA's base of operations. (Includes action items to
    improve FDA's information management infrastructure.)

**Goal 2**: Improve Patient and Consumer Safety

-   Strengthen the science that supports product safety.

-   Improve information systems for problem detection and public
    communication about product safety.

-   Provide patients and consumers with better access to clear and
    timely risk-benefit information for medical products.

-   Provide consumers with clear and timely information to protect them
    from food-borne illness and promote better nutrition.

**Goal 3**: Increase Access to New Medical and Food Products

-   Increase the number of safe and effective new medical products
    available to patients.

-   Improve the medical product review process to increase the
    predictability and transparency of decisions using the best
    available science.

-   Increase access to safe and nutritious new food products.

**Goal 4**: Improve the Quality and Safety of Manufactured Products and
the Supply Chain.

-   Prevent safety problems by modernizing science-based standards and
    tools to ensure high-quality manufacturing, processing, and
    distribution.

-   Detect safety problems earlier and better target interventions to
    prevent harm to consumers.

-   Respond more quickly and effectively to emerging safety problems,
    through better information, better coordination and better
    communication.

### 4.2.2 Information Management/Information Technology Strategy 

The Office of Chief Information Officer is realigning operations to
support the Agency goals and objectives. A critical factor in achieving
these goals and objectives requires data interoperability. The IT
strategy considers both short- and long-term initiatives to provide the
mechanisms to establish an appropriate environment that facilitates data
interoperability and identifying data assets. A planned infrastructure
will incorporate standards at many levels, including application
development, terminology, content exchange and content where
appropriate. The underlying standards will be promoted from a central
organization to ensure all Centers are reusing code effectively as well
as managing master data elements and data sources in a similar fashion.

# 5.0 PDUFA IV IT Strategy

The PDUFA IV IT strategy is one component of the overall FDA IT
strategy. In order to accomplish the goals in the PDUFA commitment
letter, the FDA through the PDUFA Review Board, has developed the PDUFA
IV IT Strategy, which incorporates efforts that are currently underway
to improve general IT processes and practices, alongside efforts that
have been developed specifically to satisfy PDUFA-driven goals. By doing
so, overall efficiency is increased and the FDA's ability to further
enhance the Agency mission is enabled. The FDA is committed to achieve
the long-term goal of an automated standards-based IT environment for
the exchange, review, and management of information supporting the
process for the review of human drug applications and continued risk and
benefit assessment throughout the product life cycle. To realize this
goal, the Agency's strategy is to evaluate current business processes,
IT Applications, and the overall IT architecture to define a target
enterprise architecture that will achieve the IT goals defined in the
PDUFA IV Commitment Letter.

The formation of the FDA Bioinformatics Board (BiB) in February 2006
addressed a growing number of business automation challenges facing FDA,
and was intended to ensure that Agency planning for future business
automation meets the needs of FDA programs while satisfying external
demands on the Agency.

The BiB works under a strategic framework for automation established by
the Commissioner and implemented by the FDA Management Council. The BiB
coordinates and oversees all activities and decisions related to
business automation planning, acquisition, and implementation throughout
FDA, and ensure that the activities related to its charge are
communicated to all levels of the Agency. The BiB also ensures
coordination of activities among FDA representatives to the Federal
Health Architecture program and other federal health informatics
initiatives, the FDA Regulation Policy Council, the FDA Data Standards
Council, and the Enterprise Architecture Review Board, particularly with
regard to business process planning and regulatory policies. The BiB
reports directly to the FDA Management Council.

Business Review Boards (BRBs) that correspond to the core business areas
identified in the Agency's common business process model serve as
standing subcommittees of the BiB. In addition, a BRB to support the
scientific computing and computational science work of the FDA has been
established. Each BRB supports the BiB in its respective areas of
expertise.

The business areas include the following:

-   Pre-Market Review

-   Product Quality

-   Post-Market Safety

-   Scientific Computing /Computational Science

-   Administrative Services

The BRBs are supported by a multidisciplinary team which ensures that
every information management project takes a comprehensive view. The
disciplines in this team include:

-   Regulation policy analysis and development

-   Strategic and performance planning

-   Business process modeling and analysis

-   Data standards development and adoption

-   Information technology

-   Finance

The structure of the BiB and BRBs are depicted in a diagram on the next
page.

> **The structure of the BiB is depicted below in Figure 2**

![](media/image3.png){width="5.9944444444444445in"
height="4.051388888888889in"}

For more information regarding the Bioinformatics Board Organization,
the charter can be found at:
<http://www.fda.gov/smg/vol3/2000/2010_7.html>

The Data Standards Council charter is located at:
<http://www.fda.gov/smg/vol3/2000/2010_3.html>

## 5.1 Business Modernization and Transformation

The FDA has embarked on a business modernization and transformation
effort to improve how the Agency achieves its mission. The FDA initiated
Agency-wide business process planning in 2003 to articulate the FDA's
mission-critical activities and to develop a strategically-aligned
common business model. With support from external consultants, the FDA
analyzed current regulatory business processes and supporting management
and administrative processes, which led to the development of a common
FDA business process framework that was ratified by the FDA's Management
Council in 2005. The business process framework describes work processes
at a high level, using general language and concepts that demonstrate
the commonality of core mission functions among all of the FDA product
centers and other programs and organizational units. In addition,
analyses of business processes were completed to assess the importance
of: business process vs. capability to perform, and importance vs. IT
system capability.

The business process framework was revised in 2006 to improve alignment
with the FDA's new strategic goal framework. This alignment helps ensure
FDA's business process improvement initiatives support the Agency's
strategy. The FDA adopted a consistent methodology for modeling business
processes for Agency-wide initiatives. Having a consistent methodology
enables the FDA to evaluate and assess business processes coherently
throughout the Agency.

The BiB, through the work of the BRBs, engaged in business process
modeling to better define cross-Agency opportunities. Strategic roadmaps
have been developed by the BRBs and integrated together to meet the
FDA's vision of modern, integrated systems, cutting edge analytic tools,
access to high quality data, and effective communication vehicles
necessary to achieve the FDA's mission of protecting and promoting
public health.

Appendix 7.4 lists each of the BRBs 5-year goals, priorities, and
current projects.

### 5.1.1 Business Process Improvement

While considering the role of information technology and automation, the
FDA's general approach to business process improvement is to:

-   identify a target area for improvement

-   establish performance goals

-   model the business processes using the Agency-wide standard
    methodology; and

-   identify opportunities for improvement through analysis and
    collaborative problem-solving

This approach includes active involvement of senior management and
operational business owners who understand and champion business process
improvement initiatives that improve the effectiveness and efficiency of
FDA. An important component of these activities will be a continuing
focus on the quality management aspects of FDA operations.

## 5.2 Target Architecture 

The Target Enterprise Architecture (EA) for the FDA will provide a
business-driven plan that describes the desired end-state for the FDA's
business architecture, data architecture, applications architecture,
technical architecture, security architecture, and standards profile.
The primary purpose of the Target EA is to effectively plan a course for
achieving the FDA's strategic vision and goals. It is one element in a
broader set of interrelated activities that collectively enable the FDA
managers and staff to define a vision, develop strategies and plans for
achieving the vision, make resource decisions, implement strategies and
evaluate performance.

By defining the end-state from several distinctive perspectives (e.g.
business, data, etc.), the Target EA will also provide stakeholders with
a view into the complex relationships that exist among these different
perspectives. For example, the Target EA will provide insight into how a
particular need translates into a set of target FDA business processes,
and how those business processes will be supported by a common set of
technologies.

The FDA has numerous information systems, executes overlapping business
and information processes, and relies on a number of technologies that
are expensive to maintain. To reduce costs and streamline operations,
the FDA is migrating toward a more service-oriented and component-based
approach to architecture. This approach, consistent with government and
industry best practice, will enable the FDA to "build once, use often."
In other words, by separating out the functionality or capabilities of a
business process or application into discrete pieces, components can be
shared and reused across the enterprise. As a result of this approach,
the FDA Target EA will:

-   Improve Program Performance -- The overarching benefit of the Target
    EA is that it provides opportunities to improve the efficiency and
    effectiveness of the FDA's programs. It ensures that data is
    optimized in support of the business, and applications and
    technology solutions are driven by business needs. It also allows
    FDA to more readily share services/data across organizational and
    functional lines.

-   Improve Interoperability -- The Target EA establishes
    enterprise-wide standards that promote platform and vendor
    independence, enabling greater interoperability across disparate
    applications, both internal and external.

-   Improve Utilization of Resources -- The Target EA reduces system
    development and operation and maintenance costs by eliminating
    duplicative investments, promoting sharing of common services, and
    establishing Agency-wide standards.

-   Accelerate System Implementation -- The Target EA equips the
    Agency's system developers and architects with an inventory of
    component-based services from which to choose that provide well
    defined functionality, thus maximizing reuse and portability of
    previously developed processes, components, code, etc.

-   Simplify Investment Decisions -- The Target EA provides a view from
    strategy to business function to technology, allowing
    decision-makers to be able to more quickly assess the relative value
    of initiatives, and to identify duplicative and misaligned
    initiatives.

-   Reduce IT Diversity and Complexity -- The Target EA simplifies the
    FDA's IT environment by promoting standards and the sharing and
    reuse of common technologies.

The FDA EA program intends to accomplish this by addressing the EA in
segments: Post-Market, Pre-Market, Product Quality, Scientific, and
Administrative. The corporate governance structure of the FDA
Bioinformatics Board and the subordinate Business Review Boards will be
leveraged to architect these segments.

The Agency's approach to target architecture development will follow
OMB's "Analyze-Architect-Implement" model. Under the "Analyze" phase,
the Agency is executing three parallel initiatives to analyze and assess
current regulatory business processes and the IT systems that support
them. These enterprise initiatives are:

1.  Business Modernization / Transformation (BMT) -- This initiative is
    described above in Section 5.1.

2.  IT Assessment -- The FDA is conducting an IT Application Assessment
    to identify potential Agency-wide applications. This initiative is
    using a set of agreed upon criteria to assess existing IT
    Applications. It is sponsored through the Agency's Office of the CIO
    and assesses the IT applications from two perspectives, business
    value and technical viability. The outcome of the assessment will be
    recommendations and supporting analysis that identify systems to be
    enhanced to satisfy common business needs, systems to be expanded
    and/or maintained to satisfy special purposes, and systems to be
    retired from the Agency's IT portfolio. The primary focus of the
    assessment will be the Agency's pre-market activities.

3.  Electronic Platform (e-platform) -- On December 18, 2006, the FDA
    held a Part 15 hearing requesting public comment on transitioning to
    an all-electronic submissions environment and an electronic platform
    (<http://www.fda.gov/ohrms/dockets/dockets/06n0464/06n0464.htm>).
    The FDA requested the public to comment on the following issues
    related to an all-electronic environment.

    i.  The feasibility issues related to the electronic submission of
        pre-market submissions and other regulatory information; and

    ii. The issues related to the concept and feasibility of an
        electronic platform that would facilitate the exchange of
        clinical research information and other regulatory product
        information, the role of a public/private partnership helping
        the creation and assessment of such an electronic platform, and
        the management of the platform after its creation by a private
        entity with the relevant technological expertise.

> The table below provides an update on the e-platform activities and
> two ongoing efforts that have a potential to become components of an
> e-platform.

**E-Platform Initiatives**

+-----------------------------+------------+--------------------------+
| **Project Name and          | **Current  | **Strategy /             |
| Description**               | Status**   | Milestones**             |
+=============================+============+==========================+
| E-platform: a common        | In March   | A strategy to move       |
| electronic platform for the | 2007 the   | forward is being         |
| exchange of clinical        | FDA and    | developed in             |
| research data (i.e., the    | NIH        | collaboration with the   |
| data normally collected     | jointly    | NIH and the NCI with     |
| during the course of a      | issued a   | plans to finalize        |
| clinical trial, as well as  | request    | strategy in the first    |
| the submission, receipt,    | for        | quarter of 2008.         |
| and management of           | i          |                          |
| regulatory product          | nformation |                          |
| information)                | (RFI) on   |                          |
|                             | the        |                          |
|                             | formation  |                          |
|                             | of a       |                          |
|                             | publ       |                          |
|                             | ic-private |                          |
|                             | p          |                          |
|                             | artnership |                          |
|                             | whose goal |                          |
|                             | it would   |                          |
|                             | be to      |                          |
|                             | establish  |                          |
|                             | and        |                          |
|                             | maintain   |                          |
|                             | the        |                          |
|                             | e-platform |                          |
|                             | (          |                          |
|                             | [[http://w |                          |
|                             | ww.fbo.gov |                          |
|                             | /servlet/S |                          |
|                             | olicitatio |                          |
|                             | n/R/HHS/FD |                          |
|                             | A/DCASC/e- |                          |
|                             | Platform-R |                          |
|                             | FI]{.under |                          |
|                             | line}](htt |                          |
|                             | p://www.fb |                          |
|                             | o.gov/serv |                          |
|                             | let/Solici |                          |
|                             | tation/R/H |                          |
|                             | HS/FDA/DCA |                          |
|                             | SC/e-Platf |                          |
|                             | orm-RFI)). |                          |
|                             | Public     |                          |
|                             | comments   |                          |
|                             | and        |                          |
|                             | responses  |                          |
|                             | to the RFI |                          |
|                             | have been  |                          |
|                             | reviewed.  |                          |
+-----------------------------+------------+--------------------------+
| FIREBIRD, Federal           | Pilot      | Complete harmonized FDA  |
| Investigator Registry of    | completed  | requirements for CDER,   |
| Biomedical Informatics      | and        | CBER and CDRH.           |
| Research Data, is a         | limited    | Functionality includes   |
| partnership between the     | production | 1572 data extraction,    |
| National Cancer Institute   | impl       | inspection data entry,   |
| (NCI) and the FDA to create | ementation | query and reporting      |
| an infrastructure to        | in 2007,   | capabilities,            |
| support the electronic      | for        | integration with Center  |
| dissemination of clinical   | NC         | application tracking     |
| research information.       | I/Division | systems, and data        |
| FIREBIRD enables            | of Cancer  | migration from existing  |
| investigators to register   | Prevention | clinical investigator    |
| online with clinical trial  | DCP and    | and bioresearch          |
| sponsors, allows sponsors   | in         | monitoring systems.      |
| to electronically maintain  | vestigator |                          |
| and manage 1572             | community  |                          |
| registrations, and          |            |                          |
| eliminates the paper-based, | Begin      |                          |
| manual process for 1572     | re         |                          |
| forms by providing the FDA  | quirements |                          |
| with electronic access to   | gathering  |                          |
| the information.            | for FDA    |                          |
|                             | fun        |                          |
|                             | ctionality |                          |
+-----------------------------+------------+--------------------------+
| CP -- The Collaboration     | Re         | Prototype will be tested |
| Portal will provide a       | quirements | by FDA and industry      |
| web-based collaboration     | completed  | users starting January   |
| platform where applicants   | and        | 2008. If approved by     |
| and the FDA can review and  | d          | business stakeholders,   |
| negotiate SPL-based labels. | evelopment | Release 1.0 will be in   |
| This online collaboration   | underway   | Production late 2^nd^    |
| should enhance the FDA's    | with CRADA | quarter of 2008.         |
| ability to approve final    | partner    |                          |
| labeling at the time of the |            |                          |
| application approval.       |            |                          |
+-----------------------------+------------+--------------------------+

As these enterprise initiatives progress, the FDA governance process
(e.g., Bioinformatics Board, PDUFA Review Board, Business Review Boards,
etc.) will evaluate the aggregate recommendations and collaborate with
the FDA Chief Information Officer (CIO) to define the enterprise target
architecture. This Plan reflects what is known today and be updated
periodically to reflect the priorities and direction of the FDA
governance bodies. The following diagram graphically depicts the plan's
dependencies on two of these initiatives.

**Figure 3 depicts PDUFA IV planning and analysis and how it is
coordinated with planning activities across the Agency**

Once the enterprise target architecture is defined and business has
prioritized and sequenced the Agency's priorities, the Office of the CIO
will design the IT solutions that will implement the business needs
within the target architecture. As the enterprise architecture matures,
FDA will focus on the development of common IT solutions that support
multiple process areas (e.g., Application Submissions, Review
Workflow/Tracking, Electronic Document Repositories, etc.). FDA
recognizes that specific business needs exist that may not be satisfied
through common software and will develop IT solutions that support these
specific needs.

The Agency will leverage the outcome from the IT Assessment and other
business process modeling efforts to determine which solutions can be
built upon with new or additional functionality, maintained as an
ongoing IT investment (steady state) or retired from service. Once this
assessment has taken place, the Agency will refine the PDUFA IV IT Plan
to reflect greater detail.

## 5.3 Guidance, Policy and Regulation

During the PDUFA III timeframe from fiscal years 2002 through 2007, the
FDA developed regulations and published guidance to improve the
consistency of electronic submission of regulatory documents and data.
During this timeframe, there was a significant increase in the number of
submissions sent to the Agency electronically. The increase in the
number of electronic submissions received by the FDA can be directly
attributed to the PDUFA III strategy to implement the Electronic Common
Document (eCTD) submission format, the implementation of the FDA
Electronic Submissions Gateway (ESG), and the implementation of the
Electronic Labeling Rule (ELR) and the Physicians Labeling Rule (PLR).
The development and publishing of guidance to industry and regulation
changes were critical to the success of these initiatives.

During PDUFA IV, the FDA will continue to work with Industry to increase
the number of submissions sent to the Agency electronically. The FDA
will develop regulations and guidance to improve the consistency of data
organization, to improve submission processing, to improve access to
documents and data, and to improve the evaluation of submission
information. The FDA will continue the work that has already begun to
establish an electronic architecture for enhanced information
management. This directly supports the FDA strategy for implementing an
all-electronic environment.

Format and data standards are integral to the receipt of electronic
submissions. The FDA will continue to work with our stakeholders to
coordinate the implementation of standards through public meetings,
pilot testing, external training and tutorial sessions. As standards are
approved through the various standard organizations and adopted
internally, the FDA will update our guidance and modify our regulations
to utilize the new standards.

This section describes the FDA's strategy for managing all policy
throughout its life cycle. All important FDA policy is documented in the
form of (1) regulation, (2) guidance, or (3) Manual of Policies and
Procedures (MaPP) and Standard Operating Procedure and Policy (SOPP).

**Regulation, Rule**

A Regulation or Rule is a policy that is legally binding and
enforceable. It is promulgated under the procedures set forth in the
Administrative Procedure Act (5 U.S.C. 551), usually with notice and
comment rulemaking.

The Unified Agenda of Federal Regulatory and Deregulatory Actions, (also
know as the semi-annual regulatory agenda) is published in the spring
and fall of each year. Since 1978, Federal agencies have been required
by Executive Orders to publish agendas of regulatory and deregulatory
activities. The Regulatory Plan, which is published as part of the fall
edition of the Agenda, identifies regulatory priorities and contains
additional detail about the most important significant regulatory
actions that agencies expect to take in the coming year. More
information can be found at the following link:
<http://www.fda.gov/oc/industry/unifiedagenda/agenda.html>.

The FDA is working on the following proposed rules pertaining to
electronic submissions:

-   Electronic Registration and Drug Listing Rule

-   Submission of Standardized Electronic Study Data from Clinical
    Studies Evaluating Human Drugs and Biologics

**Guidance**

A Guidance document is a nonbinding recommendation or guidance that is
intended primarily to assist industry or other regulated entities. A
Guidance document refers to any written communication that describes or
explains an Agency or Center policy on a regulatory issue (See 20 CFR
10.115(b)). The term guidance generally refers to guidance for regulated
entities (e.g., the pharmaceutical industry). In some instances, Centers
have developed reviewer guidance or guidance for industry and reviewers.
Guidance documents do not include (1) FDA reports; (2) general
information documents provided to consumers; (3) documents relating
solely to internal FDA procedures (e.g., where there is no external
interaction); (4) speeches, journal articles, editorials, press
materials or media interviews; (5) warning letters; (6) memoranda of
understanding; or (7) other communications or actions taken by
individuals at the FDA directed to individual persons or firms.

Guidance documents must be developed according to good guidance
practices. The Food and Drug Administration Modernization Act of 1997
(FDAMA) amends the Federal Food, Drug, and Cosmetic Act by incorporating
aspects of good guidance practices, including the provision for public
participation in the development of significant guidance documents and
the opportunity for public comment upon issuance of all guidance. In
response to FDAMA, the FDA codified its policies and procedures for the
development and issuance of guidance documents in 21 CFR 10.115 in
September 2000.

Guidance documents provide assistance to the regulated industry and the
FDA by clarifying requirements imposed by Congress or promulgated by the
FDA and by explaining how industry and the FDA may comply with those
statutory and regulatory requirements. Guidance documents are prepared
to establish clarity and consistency in the FDA policies, regulatory
activities, and inspection and enforcement procedures. Guidance
documents provide industry with specific details that often are not
included in the relevant statutes and regulations, and are intended to
assist the pharmaceutical industry in carrying out its obligations under
laws and regulations on subjects such as the processing, content,
evaluation, and approval of drug and biologic product applications and
the design, production, manufacturing, and testing of regulated
products. These documents also provide specific review and enforcement
approaches to help ensure that the FDA\'s employees implement the FDA\'s
mandate in an effective, fair, and consistent manner. Guidance documents
do not establish legally enforceable rights or responsibilities and, as
such, are not binding on the Agency or the public. Rather, they explain
how the Agency believes the statutes and regulations apply to regulated
activities and reflect the FDA\'s current thinking on the subject
addressed in the document.

The Agency recognizes the importance of maintaining a transparent
guidance development process. Therefore, the Agency has implemented
various practices intended to obtain input at the earliest stages of
guidance document development and abide by good guidance practice (GGP)
regulation (21 CFR 10.115).

-   The Agency is required to annually publish in the Federal Register
    an Agency guidance agenda with the goal of soliciting comment on
    Agency intentions to develop guidance.

-   CDER and CBER maintain Guidance Agendas on their Internet sites
    listing the Guidance documents they intend to issue in the current
    year. This enables the public to see what the Centers are working
    on. The link to the CDER Guidance Agenda is
    <http://www.fda.gov/cder/guidance/> and the link to the CBER
    Guidance Agenda is <http://www.fda.gov/cber/guidelines.htm>.

-   The Agency may solicit or accept early input on the need for a new
    or revised guidance, or assistance in the development of a
    particular guidance document, from individual governmental and/or
    nongovernmental groups (e.g., National Institutes of Health,
    consumer groups, trade associations, patient groups, public interest
    groups).

-   The Agency may participate in meetings with these various parties to
    obtain each party\'s views on priorities for developing guidance
    documents.

-   The Agency may hold meetings and workshops to obtain input from
    interested parties on the development or revision of guidance
    documents on a particular subject area.

-   The Agency may hold a public workshop to discuss a draft and/or
    present a draft to an advisory committee when there are highly
    controversial or unusually complex new scientific issues.

-   The Agency may issue a notice in the Federal Register soliciting
    public input before developing draft guidance.

Comments will be accepted at any time pertaining to all final guidance
documents. Comments on guidance documents in use should be submitted to
the Division of Dockets Management or to the relevant division. Guidance
documents will be revised in response to such comments, as appropriate.

**Policy, Procedure**

Policies and procedures primarily intended to provide direction to
reviewers or other staff within the Centers on how they are to do their
work will be issued in a MaPP or SOPP. Instructions and templates for
the proper development, formatting, processing, routing, and use of
policy documents are published and utilized for each of the Centers.
These instructions and templates provide consistency in the policies and
procedures that are published, and decrease the time to develop, review
and implement the policies and procedures in the Centers.

## 5.4 Data Standards

The FDA recognizes the importance of, and is committed to, using data
standards for regulatory submissions wherever possible. For the purposes
of this discussion, data standards can be divided into two broad
categories: exchange standards and terminology standards. Exchange
standards provide a consistent way to exchange information between
organizations and computer systems. Exchange standards help ensure that
the both sending and the receiving system both understand unambiguously
what information is being exchanged. For example, Structured Product
Labeling (SPL) is an exchange standard for product information.
Terminology standards, on the other hand, provide a consistent way to
describe concepts. For example, Unique Ingredient Identifiers (UNII)
provides a consistent way to describe substances in products.

This section describes the FDA's strategy for managing data standards
throughout their life-cycle. The important principles in standards
management at the FDA are described below. From the FDA's perspective,
standards should:

-   Use voluntary consensus based standards developed in accredited
    standards development organizations in place of government unique
    standards unless such standards are either inconsistent with
    applicable laws and procedures.[^1]

-   Align with existing health information technology initiatives, laws,
    regulations, and mandates (e.g. executive orders) and

-   Coordinate with other standards currently in use.

The FDA recognizes that not all of the principles can be met in all
cases. The FDA will strive to adhere to as many principles as possible
when selecting a standard for implementation.[^2] The discussion that
follows applies equally to both exchange and terminology standards,
unless otherwise noted.

The life-cycle of a data standard can be divided into the following
steps:

1.  Needs Assessment and Requirements Gathering

2.  Development, Adoption and Maintenance

3.  Implementation

**Needs Assessment and Requirements Gathering**

An FDA business component identifies the need for a standard and
identifies a business sponsor to represent the business community during
subsequent phases.

The appropriate Business Review Board reviews the need and, if it
concurs, raises it to the Bioinformatics Board for review.

Upon concurrence, the Bioinformatics Board instructs the Data Standards
Council to identify a standard that will meet the business need.

The Data Standards Council works with the business sponsor to create a
working group of the FDA subject matter experts to gather business
requirements.

The end-product or deliverable at the conclusion of this phase is a
document that describes the business needs or defines the business
processes that the standard is intended to support (e.g., scenarios, use
cases, or storyboards) in sufficient detail to begin standards
development and adoption.

**Development, Adoption, and Maintenance**

The Data Standards Council first attempts to identify an existing
standard that will meet the business need. Priority is given to
standards that adhere to the principles described previously. If a
standard is not already available, then the DSC begins development
activity. The DSC identifies and works with a well-recognized standards
development organization (SDO), when appropriate to develop and adopt
the standard. Priority is given to accredited, open consensus SDOs.
Examples of SDOs are:

-   Accredited, open consensus SDO

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-   International Standards Organization (ISO)

-   American Nation al Standards Institute (ANSI)

-   Health Level Seven (HL7)

-   National Council for Prescription Drug Programs (NCPDP)

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```
-   Others

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```
-   Clinical Data Interchange Standards Consortium (CDISC)

> Global regulatory standards groups (ICH, VICH, GHTF)

In instances where work with these organizations are inconsistent with
applicable FDA processes or otherwise impractical or inappropriate, then
the DSC may develop the standard. During this phase, the FDA tests the
standard to ensure that the standard is capable of meeting the business
requirements. The figure below depicts the interactions of various FDA
components during this phase.

**Figure 4: Standards Development and Adoption at the FDA**

For new health information exchange standards, the FDA works within HL7.
The FDA also encourages other business experts, such as CDISC, ICH,
other government agencies, and international regulatory bodies to bring
their business requirements to HL7 to ensure interoperability among
health information exchange standards.

**Figure 5: Developing Health Information Exchange Standards within
HL7**

For terminology standards, the FDA uses existing terminologies whenever
possible (rather than create new terminologies). Priority is given to
terminologies that adhere to the principles described previously. The
FDA recognizes its role in maintaining certain terminologies (e.g.,
Unique Ingredient Identifier).

The Development and Adoption phase ends when the Data Standards Council
has identified or developed a standard with the appropriate conformance
specifications that meets the business requirements.

The DSC works with the FDA business community and the appropriate SDO or
terminology standards maintenance organization to update the standard as
needed.

**Implementation**

The DSC presents to the Bioinformatics Board the standard that meets the
business needs described during the Needs Assessment and Requirements
Gathering process. The BiB seeks the advice of the appropriate BRBs in
determining whether to implement a standard.

If the BiB decides to implement, then it directs the appropriate BRB to
develop and execute an implementation plan, with appropriate BiB
oversight and DSC interaction throughout the process. This will often
require updating existing systems or developing new systems in close
coordination with the Office of the CIO.

The DSC works with the business community to transition to new standards
as technology advances and previous standards become outdated.

For terminology standards, the FDA partners with the National Cancer
Institute Enterprise Vocabulary Services (EVS). The NCI EVS hosts the
FDA terminologies and makes them freely available to the public.

In general, the implementation of standards can be difficult due to the
vast number of stakeholders using or planning to use a standard.
Therefore, there is a great deal of uncertainty about specific
timelines.

The FDA is committed to working throughout the standards development and
implementation process describe below with the business community to
bring important improvements in information management that provide
significant performance benefits and improve public health and safety.
The DSC standards efforts underway are illustrated in the following two
graphics. More specifics about each standard is outlined in Section 6.0.

**Figure 6: Data Exchange Standards Process**

![](media/image4.png){width="6.277165354330709in"
height="2.0984251968503935in"}

**Figure 7: Terminology Standards Process**

![](media/image5.png){width="6.277165354330709in"
height="2.090944881889764in"}

[]{.mark}![](media/image6.png){width="1.1041666666666667in"
height="2.2423611111111112in"}

[]{.mark}

[]{.mark}

[]{.mark}

[]{.mark}

[]{.mark}

[]{.mark}

[]{.mark}

An important measure of success is how well the standard is implemented
according to a well-described, well-designed, publicly available
implementation plan.

Another important measure of a successful standard is the extent to
which the standard improves existing business processes. This measure
depends on the existence of business performance metrics and data before
and after standards implementation.

**Data Standards Investment Strategy**

As previously described, the life-cycle management of data standards at
the FDA is a complex process that requires careful planning, execution,
and assessment. Not surprisingly, effective data standards management at
the FDA requires a coordinated investment strategy across various FDA
components to achieve success. Overall, the FDA must achieve:

-   An adequate number of FTEs dedicated to data standards management,
    as well as

-   Sufficient funding to support data standards projects

During needs assessment, the Bioinformatics Board and the associated
Business Review Boards play a dominant role in assessing the FDA's needs
for a new standard. The Data Standards Council provides a data standards
liaison to the BRBs to provide guidance and/or mitigate risks related to
existing or new standards work within the DSC or relevant Standards
Development Organizations.

During requirements gathering, development, adoption, and maintenance of
a data standard, the DSC plays the dominant role. The DSC staffing
consists of core experts in data standards development and
volunteers/representatives from the various programs with expertise in
the respective business processes. The DSC support activities associated
with this phase of data standards management include;

-   Interaction with standards development and standards maintenance
    organizations

-   Exchange standards development

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-   Data standards requirements gathering / use case development

-   Modeling requirements and use cases (e.g., modeling to HL7 Reference
    Information Model)

-   Testing model against requirements and use cases to include
    development of visualization tools (e.g., stylesheets, XForm)
    documentation and coordination assistance

-   Balloting (e.g., ballot preparation, presentation and
    reconciliation)

-   Accreditation

-   Conformance specifications (implementation guide)

```{=html}
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```
-   Terminology standards development

-   Standards maintenance (e.g., Unique Ingredient Identifier, NCI
    Enterprise Vocabulary Services)

-   Training and implementation support

```{=html}
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```
-   Support for training or other related IT development activities
    associated with standards adoption and implementation (e.g. data
    type specification, message instance examples or data standards
    harmonization)

During the Implementation phase, the Bioinformatics Board and the Office
of the Chief Information Officer play the dominant role in data
standards implementation, with substantial support from the Data
Standards Council and the Office of Planning. Implementation activities
include;

-   Business and IT impact analyses

-   Development or enhancement of an IT system to use the standard

-   Business process re-engineering

-   Training

-   Change management

-   Outreach activities to the FDA stakeholders

## 

# 6.0 Programs

This section is divided into two sub-sections; Pre-Market Activities and
Post Market Activities. The purpose of this section is to describe the
current IT environment at a high-level and to show the FDA's current
vision for the PDUFA IV target environment. As stated in previous
sections, there are a number of ongoing planning activities that may
impact how (e.g. the role of the e-Platform) and when (i.e. ensuring
alignment with Agency strategic goals) the FDA is moving towards an
automated standards-based IT environment. Although the FDA is continuing
to address the strategy to fully implement the standards-based
environment, the FDA has made a number of important strategic decisions
in moving towards this vision and the initiatives described below
reflect those decisions and the direction of the PDUFA Program. The
division of this section into Pre-Market and Post-Market has been done
for readability purposes, the FDA's plans and governance structure has
been setup to ensure that information is shared throughout the product
life-cycle. Examples of this are FDA Electronic Submissions Gateway and
the FDA Common EDR initiative, these are described in the Pre-Market
section but the scope of these efforts includes all regulatory
documents.

## 6.1 Pre-Market Activities

In the past, most Centers in the FDA have developed and implemented
software developed by their Center IT organizations. During the PDUFA
III timeframe the FDA implemented the FDA Electronic Submissions Gateway
across the Agency and implemented the eCTD Review System, but both CBER
and CDER continue to have separate systems to track and report on PDUFA
goals and timelines. Many times there are separate systems to track
PDUFA goals (e.g. meeting request). The diagram below represents the
current environment at a high-level without providing the details on all
the current systems supporting CBER and CDER.

**Figure 8: E Submission Tracking and Archiving Premarket Current
State**

![](media/image7.wmf){width="0.7338582677165354in"
height="0.6716535433070866in"}![](media/image8.png){width="0.9909448818897638in"
height="0.9901574803149606in"}![](media/image9.png){width="0.8570866141732284in"
height="0.8570866141732284in"}![](media/image9.png){width="0.8570866141732284in"
height="0.8570866141732284in"}![](media/image10.png){width="0.6425196850393701in"
height="0.6425196850393701in"}![](media/image7.wmf){width="0.5354330708661418in"
height="0.48976377952755906in"}![](media/image7.wmf){width="0.5354330708661418in"
height="0.48976377952755906in"}

The Pre-Market target diagram represents FDA's current approach in
developing and implementing an automated standards-based IT environment
that will support the Agency's strategic goals and enable the FDA to
meet the PDUFA IT Goals.

**Figure 9: E Submission Tracking and Archiving Premarket Target State
based on Standards Based Submissions**

![](media/image11.wmf){width="0.8834645669291339in"
height="0.9192913385826772in"}![](media/image7.wmf){width="1.0118110236220472in"
height="0.9263779527559055in"}![](media/image8.png){width="1.000787401574803in"
height="1.000787401574803in"}![](media/image12.wmf){width="0.7035433070866142in"
height="0.7188976377952756in"}

The table below describes the various initiatives and activities that
are being performed or planned to move towards the target IT
environment. The table describes the project, gives some background on
the current status, discusses the FDA strategy and milestones for the
project, and provides information on the PDUFA IT Goals that the project
supports.

+-----------------------------+------------+--------------------------+
| **Project Name and          | **Current  | **Strategy /             |
| Description**               | Status**   | Milestones**             |
+=============================+============+==========================+
| Regulated Product           | Release 1  | -   Implement/accept RPS |
| Submission, RPS, is a       | of the RPS |     submissions in the   |
| Health Level Seven (HL7)    | standard   |     first quarter of     |
| standard to facilitate the  | was passed |     2008 for;            |
| processing and review of    | in May     |                          |
| regulated product           | 2007.      |     -   SPL submissions  |
| information. The next       |            |         to a paper       |
| generation of processing    |            |         NDA/BLA.         |
| the eCTD format will be     |            |                          |
| transitioned to the RPS     |            |     -   Electronic       |
| standard. The FDA plans on  |            |         datasets to a    |
| using the RPS standard to   |            |         paper            |
| meet the PDUFA goal to      |            |         IND/NDA/BLA.     |
| cross-reference to          |            |                          |
| previously submitted        |            |     -   Single           |
| electronic materials        |            |         Investigator     |
| through standardized        |            |         IND.             |
| automated links and to      |            |                          |
| standardize the two-way     |            | ```{=html}               |
| communication between the   |            | <!-- -->                 |
| sponsor and the FDA by      |            | ```                      |
| incorporating these         |            | -   RPS Release 1 HL7    |
| requirements into RPS       |            |     Implementation Guide |
| Release 2. Release 2 will   |            |     ready for ballot in  |
| incorporate the following   |            |     2^nd^ quarter of     |
| requirements;               |            |     2008.                |
|                             |            |                          |
| -   Two-way communication   |            | -   Target for           |
|                             |            |     addressing PDUFA     |
|     -   Minutes and general |            |     requirements RPS     |
|         correspondence      |            |     DSTU Release 2 --    |
|         (related to two-way |            |     HL7 ballot 3^rd^     |
|         communication)      |            |     quarter of 2008.     |
|         including           |            |                          |
|         pre-submission      |            | *Additional RPS          |
|         information         |            | activities:*             |
|                             |            |                          |
| -   Referencing             |            | -   The Office of        |
|                             |            |     Combination Products |
|     -   in backbone (Master |            |     (OCP) is finalizing  |
|         Files, Other        |            |     the common format    |
|                             |            |     for combination      |
|     submission/application, |            |     product submissions, |
|         Pre-submission)     |            |     leveraging the CTD   |
|                             |            |     format and the PMA & |
|     -   Hyperlink content   |            |     510(k) submission    |
|         to other content    |            |     formats. The goal is |
|                             |            |     to complete the      |
| -   Provide information     |            |     submission format in |
|     about the submission    |            |     the first quarter of |
|     (e.g. information       |            |     2008 and to test the |
|     currently collected on  |            |     submission format in |
|     application forms)      |            |     2008.                |
|                             |            |                          |
|     -   information about   |            | -   CDRH has defined the |
|         the product         |            |     submission format    |
|                             |            |     for Premarket        |
|     -   Contact Information |            |     Approval (PMA) and   |
|                             |            |     Premarket            |
| As of January 1, 2008 CDER  |            |     Notification 510(k)  |
| will only accept electronic |            |     applications and     |
| submissions in the eCTD     |            |     plans on testing the |
| format. To facilitate the   |            |     RPS standard during  |
| transition to the CTD       |            |     2008.                |
| format FDA will start       |            |                          |
| accept CTD submissions      |            | -   CFSAN is currently   |
| based on the RPS standard   |            |     working on guidance  |
| for a limited set of        |            |     for Food Additive    |
| submissions. These          |            |     Petition and Color   |
| submissions have been part  |            |     Additive Petition    |
| of a paper submission with  |            |     submissions and      |
| the electronic portion      |            |     plans on testing and |
| submitted in the eNDA       |            |     accepting production |
| format. Applicants and      |            |     submissions in 2008. |
| sponsors will be permitted  |            |                          |
| to submit RPS using the CTD |            |                          |
| format for the following    |            |                          |
| types of submissions;       |            |                          |
|                             |            |                          |
| -   SPL to a paper NDA/BLA  |            |                          |
|                             |            |                          |
| -   Electronic datasets to  |            |                          |
|     a paper IND/NDA/BLA     |            |                          |
|                             |            |                          |
| -   Single Investigator IND |            |                          |
|                             |            |                          |
| FDA will provide            |            |                          |
| applicants/sponsors access  |            |                          |
| to web-based forms to       |            |                          |
| create and submit the       |            |                          |
| submission types above.     |            |                          |
| This strategy will allow    |            |                          |
| the PDUFA program to start  |            |                          |
| to transition to the RPS    |            |                          |
| standard and permit current |            |                          |
| applicants/sponsors using   |            |                          |
| the eNDA format or paper to |            |                          |
| move to the CTD format.     |            |                          |
+-----------------------------+------------+--------------------------+
| The FDA Electronic          | In FY2007, | -   In the first quarter |
| Submissions Gateway (ESG),  | the ESG    |     of 2008 the FDA      |
| an FDA-wide solution that   | received   |     plans to upgrade the |
| enables the secure          | and        |     ESG, by providing a  |
| submission of electronic    | processed  |     method to include    |
| regulatory submissions has  | over       |     the Center &         |
| been in production since    | 147,000    |     Submission Type      |
| May 2006, the ESG provides  | pre-market |     attributes in the    |
| the single point of entry   | and        |     AS2 Routing ID. This |
| for the receipt and         | p          |     upgrade will enable  |
| processing of all PDUFA     | ost-market |     the FDA to phase out |
| submissions.                | su         |     the AS1 submission   |
|                             | bmissions. |     method for drug      |
| Both CBER and CDER fully    | Most of    |     safety reporting.    |
| automated the electronic    | these      |                          |
| submission process by       | s          | ```{=html}               |
| implementing automated      | ubmissions | <!-- -->                 |
| systems to expedite the     | were       | ```                      |
| processing and increase the | post       | -   As stated in the     |
| availably of properly       | -marketing |     PDUFA IT Goals, the  |
| formatted ESG submissions.  | safety     |     FDA will extend the  |
| The electronic submission   | reports,   |     capability of the    |
| process encompasses the     | during the |     secure single point  |
| receipt, acknowledgment of  | last six   |     of entry to include  |
| receipt and any processing  | months of  |     two-way transmission |
| errors (to the sender),     | FY2007 the |     of regulatory        |
| routing, notification (to a | ESG was    |     correspondence. The  |
| receiving Center or         | processing |     FDA has had          |
| Office), and providing      | over       |     preliminary planning |
| access to the review team   | 13,800     |     discussions on       |
| of the electronic           | p          |     expanding the ESG    |
| submission.                 | ost-market |     functionality to     |
|                             | safety     |     meet this goal. The  |
|                             | reports    |     FDA does not plan on |
|                             | per month. |     expanding the ESG    |
|                             | In the     |     functionality in     |
|                             | pre-market |     2008.                |
|                             | area, the  |                          |
|                             | ESG was    |                          |
|                             | averaging  |                          |
|                             | over 1100  |                          |
|                             | s          |                          |
|                             | ubmissions |                          |
|                             | per month. |                          |
|                             | I          |                          |
|                             | nformation |                          |
|                             | on the ESG |                          |
|                             | process    |                          |
|                             | and        |                          |
|                             | re         |                          |
|                             | quirements |                          |
|                             | is at:     |                          |
|                             | <htt       |                          |
|                             | p://www.fd |                          |
|                             | a.gov/esg> |                          |
+-----------------------------+------------+--------------------------+
| eCTD review system -- The   | The        | -   The current activity |
| current FDA eCTD review     | current    |     is focused on the    |
| system was implemented in   | review     |     validation component |
| 2005, and allows reviewers  | system is  |     of the software. The |
| to review submissions       | in         |     FDA plans to use     |
| submitted in the ICH eCTD   | operations |     this to validate     |
| format. The review system   | and        |     individual eCTD      |
| provides search             | ma         |     submissions and to   |
| capabilities and reviewers  | intenance, |     gather statistics on |
| are able to track the       | with the   |     the number of        |
| progress of the eCTD        | latest     |     submissions in       |
| submission review at the    | release    |     compliance with FDA  |
| section level. The eCTD     | providing  |     standards, along     |
| review system functionality | the FDA    |     with a distribution  |
| includes a validation       | with the   |     of the submission    |
| component that provides a   | capability |     failures by problem  |
| log of the submission       | to         |     type. The FDA has    |
| errors.                     | integrate  |     implemented this     |
|                             | the eCTD   |     functionality at the |
|                             | review     |     end of 2007.         |
|                             | system     |                          |
|                             | with the   | -   In relation to the   |
|                             | CBER and   |     RPS strategy, the    |
|                             | CDER       |     FDA plans on using   |
|                             | submission |     the eCTD review      |
|                             | tracking   |     system to review RPS |
|                             | databases. |     based submissions.   |
+-----------------------------+------------+--------------------------+
| Workflow tracking and       | Release    | -   Release 2.0 on       |
| information management      | 1.0 on     |     11/13/2007, for all  |
| system -                    | 1/28/2006, |     CDER INDs, Master    |
|                             | for        |     Files and Emergency  |
| Is a flexible, integrated,  | T          |     Use Authorizations   |
| fully electronic workflow   | herapeutic |     plus system          |
| tracking and information    | Biologic   |     enhancements and bug |
| management systems to       | Product    |     fixes.               |
| receive, log, track,        | INDs       |                          |
| assign, process, and manage |            | -   Release 3.0 end of   |
| official submissions with   | Release    |     2008 for all CDER    |
| internal and external       | 1.4 on     |     NDAs and ANDAs.      |
| stakeholders. The system    | 1/29/2007  |                          |
| maintains the official      | for Safety | -   Release 3.x after    |
| submission records and will | Issues     |     2008 for CBER and    |
| manage and track all        |            |     CDER BLAs.           |
| communications and          | Releases   |                          |
| documentation concerning a  | 1.1        |                          |
| submission.                 | through    |                          |
|                             | 1.6 also   |                          |
|                             | provided   |                          |
|                             | system     |                          |
|                             | en         |                          |
|                             | hancements |                          |
|                             | and bug    |                          |
|                             | fixes      |                          |
+-----------------------------+------------+--------------------------+
| FIREBIRD -- Please refer to |            |                          |
| the e-Platform Initiatives  |            |                          |
| in Section 5.2.             |            |                          |
+-----------------------------+------------+--------------------------+
| Information and Computer    | Completed  | -   Establish and        |
| Technologies for the 21st   | Baseline   |     implement Program    |
| Century, ICT21, investment  | Assessment |     Management Office.   |
| will enable the FDA,        |            |                          |
| through the development of  | Completed  | -   Complete detailed    |
| an Agency-wide              | Phase 1 of |     Alternatives         |
| bioinformatics initiative,  | Bioi       |     Analysis.            |
| to strengthen product       | nformatics |                          |
| development and approval,   | Transfer   | -   Complete Phase 2 of  |
| improve manufacturing and   | Plan       |     Bioinformatics       |
| product quality, strengthen |            |     Transfer Plan.       |
| post-approval surveillance  |            |                          |
| and safety, support         |            | -   Complete             |
| electronic prescribing, and |            |     Bioinformatics       |
| improve clinical decision   |            |     design.              |
| support. The FDA expects to |            |                          |
| see mature electronic       |            | -   Complete First Phase |
| health records, personal    |            |     of Bioinformatics    |
| health records, and         |            |     Platform migration.  |
| networks that connect them. |            |                          |
| To meet these challenges    |            |                          |
| and requirements, the FDA   |            |                          |
| must modernize its capacity |            |                          |
| and communication           |            |                          |
| capabilities by             |            |                          |
| establishing a standardized |            |                          |
| approach for delivering IT  |            |                          |
| services through this       |            |                          |
| Agency-wide bioinformatics  |            |                          |
| initiative to fulfill its   |            |                          |
| core public health          |            |                          |
| responsibilities and        |            |                          |
| respond to emerging         |            |                          |
| challenges.                 |            |                          |
+-----------------------------+------------+--------------------------+
| FDA's Common Electronic     | Concept    | -   Current activity is  |
| Document Room (EDR)         | proposal   |     to define the scope  |
| initiative is intended to   | approved   |     of the Common EDR.   |
| establish one common,       | by the     |                          |
| Agency-wide,                | Bioi       | -   Development and      |
| standards-based EDR as a    | nformatics |     Testing of the       |
| single platform database    | Board      |     Common EDR           |
| for all FDA-regulated       |            |     Functionality --     |
| product documents. Having a | Project    |     4^th^ quarter of     |
| single platform database    | Charter    |     2008.                |
| that contains all documents |            |                          |
| related to the              | IT Project | -   Full Implementation  |
| FDA-regulated products will | Team was   |     -- 3^rd^ quarter of  |
| improve access to all FDA   | formed to  |     2009.                |
| documents, data, and        | define the |                          |
| metadata across center      | document   |                          |
| lines, thus enhancing the   | the        |                          |
| ability of Agency           | current    |                          |
| pre-market reviewers and    | (as-is)    |                          |
| others to perform their     | en         |                          |
| jobs. In addition, having   | vironment. |                          |
| an Agency-wide EDR offers   |            |                          |
| the opportunity to reduce   |            |                          |
| redundancy and related      |            |                          |
| costs and complexities      |            |                          |
| associated with maintaining |            |                          |
| multiple electronic         |            |                          |
| document rooms.             |            |                          |
|                             |            |                          |
| Benefits/Strategy: A Common |            |                          |
| EDR provides the FDA with   |            |                          |
| the capability to           |            |                          |
| streamline the submission   |            |                          |
| process, provide reviewers' |            |                          |
| additional collaboration    |            |                          |
| capabilities, provide       |            |                          |
| reviewers access and search |            |                          |
| for information across      |            |                          |
| traditional organizational  |            |                          |
| boundaries, and position    |            |                          |
| the FDA to share and        |            |                          |
| interact with external      |            |                          |
| networks/systems as an      |            |                          |
| Agency (e.g. e-Platform).   |            |                          |
+-----------------------------+------------+--------------------------+
| The Facts@FDA program is    |            |                          |
| part of the broader US      |            |                          |
| effort to achieve           |            |                          |
| electronic prescribing and  |            |                          |
| other e-health information  |            |                          |
| technology initiatives:     |            |                          |
| ELIPS, e-List, CP, and      |            |                          |
| SRSID                       |            |                          |
+-----------------------------+------------+--------------------------+
| Electronic Labeling Review  | Release    | -   Address the system   |
| System -- The Electronic    | 1.0 on     |     performance issue.   |
| Labeling Review System      | October,   |     Continue with        |
| receives and processes      | 2005 to    |     operation and        |
| electronic labeling         | support    |     maintenance.         |
| information through the     | receipt,   |                          |
| Structured Product Label    | store,     | -   Interface with the   |
| (SPL) standard format. The  | review,    |     workflow and         |
| labels received by the FDA  | and        |     information          |
| in SPL format are available | tr         |     management system to |
| on the Internet at          | ansmission |     access (migrated)    |
| DailyMed, part of the       | of SPLs to |     NDAs and ANDAs.      |
| National Library of         | NLM.       |                          |
| Medicine (NLM).             |            |                          |
|                             | Release    |                          |
| The Data Warehouse (DW)     | 3.0 on     |                          |
| functionality provides      | February,  |                          |
| users ad-hoc report and     | 2007 to    |                          |
| query capability on         | support    |                          |
| transmitted SPLs.           | PLR SPLs.  |                          |
|                             |            |                          |
|                             | DW release |                          |
|                             | 1.0 on     |                          |
|                             | October,   |                          |
|                             | 2006       |                          |
|                             | provides   |                          |
|                             | initial    |                          |
|                             | query and  |                          |
|                             | report     |                          |
|                             | capability |                          |
|                             | via an     |                          |
|                             | Agency     |                          |
|                             | Reporting  |                          |
|                             | tool.      |                          |
+-----------------------------+------------+--------------------------+
| Electronic Listing --       | Re         | -   Prototype will be    |
| Electronic listing will     | quirements |     tested by FDA and    |
| provide the ability to      | completed  |     industry users       |
| automate drug listing       | and        |     starting 1^st^       |
| information and validation  | d          |     quarter of 2008. If  |
| processes. The SPL data     | evelopment |     approved by business |
| elements from the labels    | underway   |     stakeholders,        |
| will be extracted and       | with CRADA |     Release 1.0 will be  |
| reused. The listing         | partner    |     in Production in     |
| information will be         |            |     2^nd^ quarter of     |
| available to the public     |            |     2008.                |
| through DailyMed and other  |            |                          |
| electronic means.           |            |                          |
+-----------------------------+------------+--------------------------+
| CP (Collaboration Portal)   |            |                          |
| -- Please refer to the      |            |                          |
| e-Platform Initiatives in   |            |                          |
| Section 5.2                 |            |                          |
+-----------------------------+------------+--------------------------+
| Substance Registration      | Release    | -   Continue with        |
| System -- The overall       | 1.0 on     |     operation and        |
| purpose of the joint        | September, |     maintenance.         |
| FDA/USP Substance           | 2005 to    |                          |
| Registration System is to   | support    | -   Interface with the   |
| support health information  | re         |     workflow and         |
| technology initiatives by   | gistration |     information          |
| generating Unique           | and review |     management system    |
| Ingredient Identifiers      | of         |     release 2.0 to       |
| (UNII) for substances in    | substances |     access (migrated)    |
| drugs, biologics, foods and | and        |     Drug Master File.    |
| devices. The UNII is a      | ingredient |                          |
| non-proprietary, free,      | names.     |                          |
| unique, unambiguous,        | This       |                          |
| non-semantic, alphanumeric  | release    |                          |
| identifier based on a       | provides   |                          |
| substance's molecular       | Unique     |                          |
| structure and/or            | Ingredient |                          |
| descriptive information.    | Identifier |                          |
|                             | (UNII)     |                          |
|                             | standard   |                          |
|                             | t          |                          |
|                             | erminology |                          |
|                             | for use in |                          |
|                             | SPL.       |                          |
|                             |            |                          |
|                             | Release    |                          |
|                             | 1.4 allows |                          |
|                             | all FDA    |                          |
|                             | personnel  |                          |
|                             | to query   |                          |
|                             | and view   |                          |
|                             | approved   |                          |
|                             | s          |                          |
|                             | ubstances. |                          |
+-----------------------------+------------+--------------------------+

The diagram and table below describes the FDA's direction in moving
towards XML based exchange messages to submit clinical and preclinical
information to the FDA. The diagram is the FDA current thinking on how
the process might work by leveraging the CDISC efforts by the end of
PDUFA IV, September 30, 2012. As stated in the Guidance, Policy and
Regulation section (5.5) the FDA is currently working on a proposed rule
that would require the electronic submission of clinical data to the
FDA. The FDA is also working with the National Coordinator for Health
Information Technology within the Office of the Secretary of the
Department of Health and Human Services to coordinate FDA efforts with
the Federal Government effort to develop and implement an interoperable
electronic medical record by 2014.

**Figure 10: PDUFA IV Target Clinical Data Flow**

![](media/image13.png){width="0.4507874015748031in"
height="0.45354330708661417in"}![](media/image14.wmf){width="0.5354330708661418in"
height="0.6492125984251969in"}![](media/image15.png){width="0.7192913385826771in"
height="0.4688976377952756in"}![](media/image14.wmf){width="0.5700787401574803in"
height="0.6484251968503937in"}![](media/image16.png){width="0.44724409448818897in"
height="0.18740157480314962in"}![](media/image16.png){width="0.4484251968503937in"
height="0.18740157480314962in"}![](media/image16.png){width="0.44724409448818897in"
height="0.18661417322834645in"}

+-----------------------------+--------------+-------------------------+
| **Project Name and          | **Current    | **Strategy /            |
| Description**               | Status**     | Milestones**            |
+=============================+==============+=========================+
| Clinical/Preclinical Data   |              |                         |
| Standards & Initiatives --  |              |                         |
|                             |              |                         |
| The FDA receives massive    |              |                         |
| amounts of clinical         |              |                         |
| research data in extremely  |              |                         |
| disparate formats using a   |              |                         |
| variety of proprietary      |              |                         |
| standards. This makes it    |              |                         |
| extremely difficult, if not |              |                         |
| impossible, to do           |              |                         |
| cross-study and application |              |                         |
| reviews. The FDA has been   |              |                         |
| working towards a           |              |                         |
| standardized approach to    |              |                         |
| capture, receive, and       |              |                         |
| analyze clinical study      |              |                         |
| data. The standardization   |              |                         |
| of clinical data is vital   |              |                         |
| to the FDA strategic        |              |                         |
| initiatives to integrate    |              |                         |
| pre-marketing clinical      |              |                         |
| trial data and              |              |                         |
| post-marketing safety data  |              |                         |
| to improve public health    |              |                         |
| and patient safety. The     |              |                         |
| goal of these efforts are   |              |                         |
| to;                         |              |                         |
|                             |              |                         |
| -   Enhance FDA regulatory  |              |                         |
|     decision making and     |              |                         |
|     address complex public  |              |                         |
|     health questions        |              |                         |
|     through improved data   |              |                         |
|     management through;     |              |                         |
|                             |              |                         |
|     -   Standardize data -  |              |                         |
|         exchange and        |              |                         |
|         terminology         |              |                         |
|         standards to        |              |                         |
|         facilitate data     |              |                         |
|         aggregation,        |              |                         |
|         analysis, data      |              |                         |
|         mining and signal   |              |                         |
|         detection           |              |                         |
|                             |              |                         |
|     -   Improved access to  |              |                         |
|         aggregate data      |              |                         |
|                             |              |                         |
|     -   User friendly tools |              |                         |
|         for review          |              |                         |
|                             |              |                         |
| -   Support of the FDA      |              |                         |
|     Critical Path           |              |                         |
|     Initiatives supporting  |              |                         |
|     regulatory research     |              |                         |
|                             |              |                         |
|     -   Safer, effective    |              |                         |
|         products            |              |                         |
|                             |              |                         |
|     -   More efficient      |              |                         |
|         product development |              |                         |
|                             |              |                         |
| The foundation for the      |              |                         |
| standardized clinical       |              |                         |
| content is the Clinical     |              |                         |
| Data Interchange Standards  |              |                         |
| Consortium (CDISC) Study    |              |                         |
| Data Tabulation Model       |              |                         |
| (SDTM). The SDTM will also  |              |                         |
| include nonclinical         |              |                         |
| requirements based on the   |              |                         |
| Standard for Exchange of    |              |                         |
| Nonclinical Data (SEND)     |              |                         |
| models that is being        |              |                         |
| harmonized with the SDTM.   |              |                         |
| The CDISC content will be   |              |                         |
| sent to FDA as an XML       |              |                         |
| message using the Health    |              |                         |
| Level Seven (HL7) Reference |              |                         |
| Information Model (RIM) and |              |                         |
| harmonized with the         |              |                         |
| Biomedical Research         |              |                         |
| Integrated Domain Group     |              |                         |
| (BRIDG) Model.              |              |                         |
|                             |              |                         |
| SDTM version 3.1.1          |              |                         |
| submissions are accepted by |              |                         |
| FDA. A draft implementation |              |                         |
| guide for SDTM 3.1.2 is     |              |                         |
| currently under review by   |              |                         |
| CDISC and FDA. FDA and      |              |                         |
| CDISC are in the process of |              |                         |
| forming a communications    |              |                         |
| team that will ensure SDTM  |              |                         |
| meets FDA's scientific      |              |                         |
| requirements.               |              |                         |
+-----------------------------+--------------+-------------------------+
| CDISC - HL7 Project - The   | CDISC        | -   The FDA is          |
| FDA plans to transition to  | Content to   |     proposing the       |
| HL7 exchange messages for   | Message      |     development of four |
| submission of all study     | Project      |     messages that map   |
| data. This initiative is    | initiation   |     to content areas    |
| based on the outcomes of    | was approved |     identified above.   |
| the CDISC Content to HL7    | by the HL7   |                         |
| Message Exploratory         | Regulated    |     -   Study Design    |
| Project. The objective of   | Clinical     |                         |
| the Exploratory Project was | Research     |     -   Study           |
| to;                         | Information  |         Participation   |
|                             | Management   |                         |
| -   Harmonize the SDTM into | (RCRIM)      |     -   Subject Data    |
|     the BRIDG model (see    | Technical    |                         |
|     below).                 | Committee    |     -   Individual Case |
|                             | 11/2007.     |         Safety          |
| -   To identify HL7         |              |         Reporting       |
|     exchange message        |              |         (ICSR)          |
|     content for submission  |              |                         |
|     to a regulatory         |              | -   This process also   |
|     authority that          |              |     includes the        |
|     addresses; a) study     |              |     completion of the   |
|     summary (clinical trial |              |     BRIDG Model         |
|     registry), b)           |              |     harmonization, to   |
|     eligibility             |              |     ensure that all     |
|     criteria, c) trial      |              |     content has been    |
|     design (including parts |              |     identified and      |
|     I and II: arms,         |              |     harmonized with the |
|     elements visits,        |              |     model before        |
|     planned assessments,    |              |     achieving normative |
|     and planned             |              |     status.             |
|     intervention(s)), d)    |              |                         |
|     statistical analysis    |              |                         |
|     plan, e) collected      |              |                         |
|     data/study data         |              |                         |
|     tabulations and f)      |              |                         |
|     derived data/analysis   |              |                         |
|     datasets, all of which  |              |                         |
|     are currently defined   |              |                         |
|     by the CDISC standard.  |              |                         |
+-----------------------------+--------------+-------------------------+
| BRIDG Model - The           | As of        | -   As mentioned above, |
| Biomedical Research         | Release 1.1, |     any content         |
| Integrated Domain Group,    | in October   |     identified as part  |
| BRIDG Model, is a domain    | 2007, the    |     of the CDISC - HL7  |
| analysis model representing | content has  |     Project will be     |
| protocol-driven             | been drawn   |     harmonized with the |
| biomedical/clinical         | from six     |     BRIDG Model in      |
| research. The BRIDG Model   | projects:    |     coordination with   |
| is a collaborative effort   |              |     the BRIDG scheduled |
| of stakeholders from the    | -   Study    |     releases.           |
| Clinical Data Interchange   |     Data     |                         |
| Standards Consortium        |              |                         |
| (CDISC), the HL7 Regulated  |   Tabulation |                         |
| Clinical Research           |     Model    |                         |
| Information Management      |     (SDTM)   |                         |
| Technical Committee (RCRIM  |     **--**   |                         |
| TC), the National Cancer    |     CDISC    |                         |
| Institute (NCI), and the    |              |                         |
| FDA to produce a shared     | -   caXC     |                         |
| view of the dynamic and     | hange/LabHub |                         |
| static semantics that       |              |                         |
| collectively define the     |   (including |                         |
| shared domain of clinical   |     Periodic |                         |
| and pre-clinical            |              |                         |
| protocol-driven research    |    Reporting |                         |
| and its associated          |     of CT    |                         |
| regulatory artifacts. In    |              |                         |
| the case of the BRIDG       |   Laboratory |                         |
| model, the domain is        |     Results  |                         |
| defined as:                 |     and Lab  |                         |
|                             |     Model)   |                         |
| *Protocol-driven research   |     **\--**  |                         |
| and its associated          |     N        |                         |
| regulatory artifacts, i.e.  | CI/HL7/RCRIM |                         |
| the data, organization,     |     TC/CDISC |                         |
| resources, rules, and       |              |                         |
| processes involved in the   | -            |                         |
| formal assessment of the    |    Regulated |                         |
| utility, impact, or other   |     Product  |                         |
| pharmacological,            |              |                         |
| physiological, or           |   Submission |                         |
| psychological effects of a  |     (RPS)    |                         |
| drug, procedure, process,   |     **\--**  |                         |
| or device on a human,       |     HL7      |                         |
| animal, or other biologic   |     RCRIM TC |                         |
| subject or substance plus   |              |                         |
| all associated regulatory   | -   Cancer   |                         |
| artifacts required for or   |     Trial    |                         |
| derived from this effort.*  |     Object   |                         |
|                             |     Model    |                         |
| The BRIDG Model serves to   |     (CTOM)   |                         |
| bridge standards, as well   |     **--**   |                         |
| as organizations and        |     NCI      |                         |
| various communities,        |     (a       |                         |
| including academic research | pproximately |                         |
| institutions and            |     50% of   |                         |
| pharmaceutical product      |     the      |                         |
| development organizations   |     total    |                         |
| and related service and     |     content) |                         |
| technology providers. It is |              |                         |
| also bridging the gap       | -   Trial    |                         |
| between clinical research   |     Design   |                         |
| and healthcare.             |     Model    |                         |
|                             |     (TDM) -- |                         |
|                             |     CDISC    |                         |
|                             |              |                         |
|                             | Patient      |                         |
|                             | Study        |                         |
|                             | Calendar,    |                         |
|                             | Phase II     |                         |
|                             | (PSC) **--** |                         |
|                             | NCI          |                         |
+-----------------------------+--------------+-------------------------+
| The JANUS data warehouse    | Memorandum   | -   Plan for and        |
| for both animal and human   | of           |     implement a Phase 3 |
| study data is being         | U            |     pilot that includes |
| developed by the National   | nderstanding |     extensions of the   |
| Cancer Institute (NCI) with | with NCI     |     Janus logical data  |
| the FDA participating       | signed in    |     model and a         |
| through its Interagency     | March 2007.  |     service-oriented    |
| Oncology Task Force         |              |     architecture        |
| activities. The NCI and the | Established  |     designed to support |
| FDA are collaborating to    | Janus Change |     the submission of   |
| implement a common,         | Control      |     HL7 messages and    |
| standards-based electronic  | Board (CCB)  |     leveraging of NCI's |
| infrastructure for          | with         |     Enterprise          |
| regulatory data and         | re           |     Vocabulary Service  |
| document submission,        | presentation |     (EVS) to begin to   |
| review, and analysis. The   | from NCI,    |     address controlled  |
| standard for the submission | FDA, CDISC,  |     vocabulary issues.  |
| of study data for Janus is  | and          |                         |
| the Clinical Data           | industry.    | -   Establish two-way   |
| Interchange Standards       |              |     data exchange       |
| Consortium (CDISC) Study    | Im           |     between FDA and NCI |
| Data Tabulation Model       | plementation |     using FDA's         |
| (SDTM) which includes the   | of Phase 2   |     electronic gateway. |
| standard for animal         | pilot, which |                         |
| toxicity data being         | integrates   | -   Have the CDISC-HL7  |
| developed by CDISC's SEND   | two reviewer |     messages completed  |
| Team. SEND data is designed | tools        |     and accepted as     |
| to work with data viewer    | (WebSDM,     |     HL-7 draft          |
| (ToxVisionTM) developed     | JReview)     |     standards for trial |
| through CRADA with          | with the     |     use Draft Standard  |
| PharmQuest, Inc.            | Janus        |     for Trial Use       |
| (PharmQuest is now          | repository.  |     (DSTU).             |
| Pointcross)                 | Phase 2      |                         |
|                             | involves     |                         |
|                             | development  |                         |
|                             | of a data    |                         |
|                             | validation   |                         |
|                             | and import   |                         |
|                             | facility,    |                         |
|                             | loading of   |                         |
|                             | validated    |                         |
|                             | SDTM         |                         |
|                             | datasets     |                         |
|                             | into Janus   |                         |
|                             | repository,  |                         |
|                             | and creation |                         |
|                             | of           |                         |
|                             | analytical   |                         |
|                             | views based  |                         |
|                             | on used      |                         |
|                             | cases using  |                         |
|                             | reviewer     |                         |
|                             | tools. Phase |                         |
|                             | 2 completed  |                         |
|                             | in the 4^th^ |                         |
|                             | quarter      |                         |
|                             | 2007.        |                         |
+-----------------------------+--------------+-------------------------+
| [Standard for Exchange of   | CDER         | -   The 3-year pilot    |
| Nonclinical Data (SEND)     | recently     |     project is to test  |
| Pilot](http://a257.g.akamai | completed a  |     the CDISC SEND      |
| tech.net/7/257/2422/01jan20 | related      |     model with the      |
| 071800/edocket.access.gpo.g | pilot        |     long-term goal of   |
| ov/2007/pdf/E7-19468.pdf) - | project      |     replacing the       |
| CDER is conducting a pilot  | (phase 1)    |     existing paper/PDF  |
| project to test, in a       | that asked   |     based listings of   |
| regulatory setting, the     | for          |     nonclinical study   |
| electronic submission of    | volunteers   |     data.               |
| nonclinical study data      | from         |                         |
| using the CDISC Standard    | industry to  | -   For this 3-year     |
| for Exchange of Nonclinical | submit       |     phase 2 pilot, full |
| Data (SEND). The purpose of | sample       |     study reports of    |
| this pilot is to test the   | nonclinical  |     the following types |
| ability of a new electronic | datasets in  |     of animal toxicity  |
| data format to support      | the SEND     |     studies will be     |
| nonclinical review          | format       |     requested for       |
| activity. The pilot also    | outside of a |     submission to an    |
| will involve a              | regulatory   |     existing IND in the |
| collaboration of FDA,       | setting (68  |     appropriate CDER    |
| available pilot             | FR 3885;     |     review              |
| participants, and the SEND  | January 27,  |     division: (1)       |
| team to update and create a | 2003). The   |     Repeat-dose         |
| new draft SEND              | phase 1      |     toxicity studies of |
| implementation guide that   | pilot also   |     14 days duration to |
| will harmonize SEND with    | evaluated    |     12 months duration  |
| SDTM. FDA anticipates that  | data         |     in any species, (2) |
| a successful pilot will     | validation   |     lifetime            |
| enable CDER to routinely    | and analysis |     carcinogenicity     |
| accept nonclinical study    | tools        |     studies in rats or  |
| data electronically in SEND | specifically |     mice, or (3)        |
| format, instead of paper or | designed to  |     6-month             |
| portable document format    | validate     |     carcinogenicity     |
| (PDF), in investigational   | datasets     |     studies in          |
| new drug applications       | according to |     transgenic mice.    |
| (INDs), new drug            | the current  |     Studies should      |
| applications (NDAs), and    | SEND         |     include             |
| biologics licensing         | standard and |     toxicokinetic data, |
| applications (BLAs).        | to enable a  |     if available.       |
|                             | reviewer to  |     Depending on the    |
|                             | efficiently  |     ongoing efforts of  |
|                             | display and  |     the SEND Consortium |
|                             | evaluate     |     to expand the SEND  |
|                             | data from    |     implementation      |
|                             | animal       |     guide, additional   |
|                             | toxicity     |     nonclinical study   |
|                             | studies      |     types may be        |
|                             | submitted in |     piloted in the      |
|                             | the SEND     |     future. If so, FDA  |
|                             | format. The  |     will post on the    |
|                             | phase 1      |     FDA SEND Web page   |
|                             | pilot        |     an updated list of  |
|                             | resulted in  |     study types the     |
|                             | development  |     Agency will accept  |
|                             | of a SEND    |     in this and any     |
|                             | Im           |     future pilots. We   |
|                             | plementation |     anticipate that a   |
|                             | Guide        |     successful phase 2  |
|                             | (Version     |     pilot, which        |
|                             | 2.3;         |     includes            |
|                             | November     |     implementation of   |
|                             | 2005), which |     any needed changes  |
|                             | is available |     to the SEND         |
|                             | on the CDISC |     implementation      |
|                             | Website      |     guide and/or the    |
|                             | (            |     data validation,    |
|                             | *<http://www |     viewing, and        |
|                             | .cdisc.org/m |     analysis tools,     |
|                             | odels/send/> |     will allow CDER to  |
|                             | v2.          |     routinely accept    |
|                             | 3/SENDV2.3Im |     specific types of   |
|                             | plementation |     nonclinical study   |
|                             | Guide.pdf*). |     data provided       |
|                             | The SEND     |     electronically as   |
|                             | Im           |     SAS transport file  |
|                             | plementation |     (XPT version 5)     |
|                             | Guide        |     datasets based on   |
|                             | describes    |     the SEND format. In |
|                             | the process  |     the case of         |
|                             | for          |     carcinogenicity     |
|                             | formatting   |     studies, a          |
|                             | nonclinical  |     successful phase 2  |
|                             | data from    |     pilot will enable   |
|                             | single and   |     submission of the   |
|                             | repeat-dose  |     entire              |
|                             | animal       |     carcinogenicity     |
|                             | toxicity and |     study data in the   |
|                             | car          |     electronic SEND     |
|                             | cinogenicity |     format, thus        |
|                             | studies for  |     eliminating the     |
|                             | submission   |     need for a separate |
|                             | purposes.    |     submission of the   |
|                             | The pilot    |     electronic tumor    |
|                             | also         |     dataset (i.e.,      |
|                             | resulted in  |     tumor.xpt).         |
|                             | the          |                         |
|                             | development  |                         |
|                             | of           |                         |
|                             | specialized  |                         |
|                             | software     |                         |
|                             | tools for    |                         |
|                             | validating,  |                         |
|                             | displaying,  |                         |
|                             | and          |                         |
|                             | analyzing    |                         |
|                             | SE           |                         |
|                             | ND-formatted |                         |
|                             | nonclinical  |                         |
|                             | data.        |                         |
+-----------------------------+--------------+-------------------------+
| Electronic Case Report Form | Six pilot    | -   Based on our        |
| eCRF Pilot - The purpose of | participants |     experience,         |
| the eCRF pilot project is   | have been    |     PDF-based CRFs from |
| to obtain experience with   | identified   |     clinical trials     |
| the CDISC Operational Data  | and a pilot  |     that employ EDC are |
| Model (ODM) based CRFs,     | schedule is  |     not ideal to        |
| with the goal of replacing  | being        |     support all review  |
| the existing portable       | developed.   |     activity. Although  |
| document format (PDF)-based |              |     the PDF-based CRFs  |
| CRFs derived from clinical  |              |     for trials that use |
| trials that use EDC and,    |              |     EDC can provide a   |
| therefore, lack paper CRFs. |              |     record of the       |
| A successful pilot will     |              |     observations        |
| allow CDER and CBER to      |              |     collected during    |
| routinely accept CRFs from  |              |     the trial (i.e.,    |
| studies that employ         |              |     the data) and       |
| electronic data capture     |              |     additional          |
| (EDC) in ODM format in      |              |     information about   |
| marketing applications      |              |     what was collected  |
| submitted in electronic     |              |     (metadata), they    |
| format.                     |              |     typically do not    |
|                             |              |     provide an audit    |
|                             |              |     trail. CDER and     |
|                             |              |     CBER are interested |
|                             |              |     in adopting a new,  |
|                             |              |     standard format     |
|                             |              |     that can replace    |
|                             |              |     the PDF-based CRF   |
|                             |              |     and that can        |
|                             |              |     reliably provide    |
|                             |              |     all three           |
|                             |              |     components of the   |
|                             |              |     CRF in an           |
|                             |              |     electronic format:  |
|                             |              |     Data, metadata, and |
|                             |              |     audit trail.        |
|                             |              |                         |
|                             |              | -   The ODM is an       |
|                             |              |     XML-based standard  |
|                             |              |     that facilitates    |
|                             |              |     the electronic      |
|                             |              |     exchange of         |
|                             |              |     clinical trial      |
|                             |              |     data, metadata, and |
|                             |              |     audit trail. We are |
|                             |              |     working with CDISC  |
|                             |              |     to develop the      |
|                             |              |     capabilities within |
|                             |              |     CDER and CBER to    |
|                             |              |     review CRFs using   |
|                             |              |     ODM. CDISC employed |
|                             |              |     the current         |
|                             |              |     production version  |
|                             |              |     (Version 1.2) of    |
|                             |              |     the ODM on the      |
|                             |              |     CDISC Web site, and |
|                             |              |     we performed some   |
|                             |              |     initial testing of  |
|                             |              |     limited CRF data in |
|                             |              |     ODM. To help in     |
|                             |              |     this development,   |
|                             |              |     we are launching    |
|                             |              |     this pilot project  |
|                             |              |     and seeking         |
|                             |              |     sponsors willing to |
|                             |              |     provide CRFs in ODM |
|                             |              |     format to test our  |
|                             |              |     capabilities to     |
|                             |              |     review these files. |
|                             |              |     However, data       |
|                             |              |     supplied during the |
|                             |              |     pilot project will  |
|                             |              |     not replace any     |
|                             |              |     regulatory          |
|                             |              |     requirements for    |
|                             |              |     submitting CRFs.    |
|                             |              |                         |
|                             |              | At this time, the pilot |
|                             |              | has not defined set     |
|                             |              | timeframes for the      |
|                             |              | submission/analysis of  |
|                             |              | eCRFs.                  |
+-----------------------------+--------------+-------------------------+
| CDISC CDASH (Clinical Data  | These        | -   The next steps are  |
| Acquisition Standards       | 'safety      |     for the CDASH       |
| Harmonization) - The        | d            |     project team to     |
| project goal is to develop  | ata/domains' |     assemble the        |
| a set of \"content          | were divided |     comments on each of |
| standards\" (element name,  | into four    |     the packages. After |
| definition, and related     | separate     |     there is agreement  |
| metadata) for a basic set   | packages or  |     on the content the  |
| of global data collection   | work         |     CDASH 'safety       |
| fields (also known as CRF,  | streams. The |     data/domain'        |
| or Case Report Form,        | CDASH        |     information will be |
| variables) that will        | project has  |     released for public |
| support clinical research   | addressed    |     comment in the      |
| studies.                    | all of the   |     first quarter of    |
|                             | 'safety      |     2008.               |
| The initial scope of the    | data/domain' |                         |
| project is the development  | areas and    |                         |
| of 16 CRF content 'safety   | FDA has      |                         |
| data/domains'; Adverse      | provided     |                         |
| Events, (Prior and)         | comments on  |                         |
| Concomitant Medications,    | all four     |                         |
| Comments, Demographics,     | packages to  |                         |
| Disposition/End of Study,   | ensure that  |                         |
| Drug Accountability, ECG,   | regulatory   |                         |
| Exposure, Inclusion and     | requirements |                         |
| Exclusion Criteria, Lab,    | will be met. |                         |
| Medical History, Physical   |              |                         |
| Examination, Protocol       |              |                         |
| Violations, Subject         |              |                         |
| Characteristics, Substance  |              |                         |
| Use, and Vital Signs. These |              |                         |
| safety domains are common   |              |                         |
| to all therapeutic areas.   |              |                         |
| The initial scope is not    |              |                         |
| the physical layout of the  |              |                         |
| CRF or terminology;         |              |                         |
| terminology is incorporated |              |                         |
| through collaboration with  |              |                         |
| the CDISC Terminology Team. |              |                         |
|                             |              |                         |
| Basic data collection       |              |                         |
| fields identified by CDASH  |              |                         |
| project work streams are    |              |                         |
| mapped into the Study Data  |              |                         |
| Tabulated Model (SDTM) and  |              |                         |
| are compliant with the SDTM |              |                         |
| Implementation Guide (SDTM  |              |                         |
| IG).                        |              |                         |
|                             |              |                         |
| FDA's role in this effort   |              |                         |
| is to ensure that the CRF   |              |                         |
| regulatory requirements are |              |                         |
| being addressed.            |              |                         |
+-----------------------------+--------------+-------------------------+
| CDISC ADaM - Analysis Data  | ADaM         |                         |
| Model-The ADaM datasets are | datasets     |                         |
| designed to provide a clear | have been    |                         |
| and unambiguous             | pilot rested |                         |
| communication of the        | by CDER      |                         |
| content, source and quality | review       |                         |
| of the datasets supporting  | staff.       |                         |
| the statistical analyses    |              |                         |
| performed in a clinical     |              |                         |
| study. They provide a       |              |                         |
| standard for transferring   |              |                         |
| analysis datasets between   |              |                         |
| sponsors and FDA.           |              |                         |
+-----------------------------+--------------+-------------------------+
| Terminology - Terminology   |              |                         |
| binding and RIM             |              |                         |
| harmonization will be done  |              |                         |
| following the HL7           |              |                         |
| Development Framework and   |              |                         |
| applicable stakeholder      |              |                         |
| processes.                  |              |                         |
+-----------------------------+--------------+-------------------------+

## 6.2 Post-Market Activities

As outlined in the Pre-Market Activities section, most Centers in the
FDA have developed and implemented software developed by their Center IT
organization to track and analyze spontaneous post-market safety
reports. The CDER Adverse Event Reporting System (AERS) has been the
exception to this rule. Although the drug safety reports are submitted
to and processed by CDER, both CBER and CDER use the same AERS
application to view the ICSR and the same data warehouse to perform
analysis on the safety reports. Within the PDUFA program there is a
separate reporting mechanism for the submission of vaccine adverse
reports; this process is handled by the Center for Disease Control (CDC)
with the information transferred to CBER for analysis.

**Figure 11: E Submission Tracking and Archiving Postmarket Current
State**

![](media/image8.png){width="1.000787401574803in"
height="1.000787401574803in"}![](media/image17.wmf){width="0.6in"
height="1.1346456692913385in"}![](media/image17.wmf){width="0.5984251968503937in"
height="0.738976377952756in"}![](media/image7.wmf){width="0.7401574803149606in"
height="0.6771653543307087in"}![](media/image7.wmf){width="0.7401574803149606in"
height="0.6771653543307087in"}

The Post-Market target diagram represents FDA's future approach in
developing and implementing an automated standards-based IT environment
that will support the Agency's strategic goals and enable the FDA to
meet the PDUFA drug safety IT Goals. As described below the FDA is
taking an Agency approach in capturing, tracking, and analyzing drug
safety reports through the MedWatch Plus initiative.

**Figure 12: E Submission Tracking and Archiving Postmarket Target
State**

![](media/image17.wmf){width="0.539763779527559in"
height="0.6673228346456693in"}![](media/image18.png){width="0.797244094488189in"
height="0.5519685039370079in"}![](media/image7.wmf){width="0.7216535433070866in"
height="0.6602362204724409in"}![](media/image11.wmf){width="0.5574803149606299in"
height="0.5799212598425196in"}![](media/image8.png){width="0.7118110236220473in"
height="0.7118110236220473in"}![](media/image19.wmf){width="0.7283464566929134in"
height="0.6653543307086615in"}![](media/image17.wmf){width="0.6299212598425197in"
height="0.6145669291338582in"}![](media/image7.wmf){width="0.6688976377952756in"
height="0.6118110236220472in"}![](media/image7.wmf){width="0.6688976377952756in"
height="0.6118110236220472in"}![](media/image12.wmf){width="0.6834645669291338in"
height="0.7236220472440945in"}

+-----------------------------+------------+--------------------------+
| **Project Name and          | **Current  | **Strategy /             |
| Description**               | Status**   | Milestones**             |
+=============================+============+==========================+
| The MedWatch ^Plus^         | High-level | -   Complete MedWatch    |
| initiative will enable the  | MW+        |     ^Plus^ requirements  |
| FDA to improve the          | Re         |     definition.          |
| timeliness, accuracy, and   | quirements |                          |
| usability of its product    | completed. | -   Award MedWatch       |
| safety surveillance data by |            |     ^Plus^ integration   |
| significantly reducing      | MOU with   |     contract.            |
| delays and errors           | NIH to     |                          |
| associated with manual data | develop    | -   Select FAERS COTS    |
| entry and coding of paper   | i          |     toolset.             |
| reports. It will provide a  | ntelligent |                          |
| user-friendly internet      | que        | -   Rollout CDER/CBER    |
| portal for anyone to report | stionnaire |     FAERS release.       |
| an adverse event resulting  | for the    |                          |
| from a FDA-regulated        | Internet   | -   Rollout CDRH/Office  |
| product. The portal will be | portal.    |     of Combination       |
| supported by an Agency-wide |            |     Products release.    |
| repository of adverse event | High-level |                          |
| reports (FAERS) with        | and        | -   Complete MedWatch    |
| integrated safety signal    | Detailed   |     Plus portal.         |
| management and analytical   | Business   |                          |
| tools.                      | and IT     | -   Integrate MedWatch   |
|                             | re         |     ^Plus^ portal with   |
|                             | quirements |     FAERS.               |
|                             | completed  |                          |
|                             | for FAERS. |                          |
|                             |            |                          |
|                             | A          |                          |
|                             | cquisition |                          |
|                             | active as  |                          |
|                             | of         |                          |
|                             | 9/24/2007. |                          |
+-----------------------------+------------+--------------------------+
| Sentinel System - The FDA,  | Sentinel   | -   Strengthen           |
| in coordination with other  | public     |     capability to draw   |
| Federal agencies,           | meeting    |     data from sources    |
| recommends assembling an    | held March |     like electronic      |
| integrated "virtual"        | 7 and 8,   |     health records and   |
| national medical product    | 2007 with  |     medical claims.      |
| safety system \-- the       | over 400   |                          |
| Sentinel System \-- which   | pages of   | -   Establish the        |
| would enable the electronic | t          |     ability of the FDA   |
| flow of safety information  | ranscripts |     to query other       |
| to and from the point of    | posted to  |     systems quickly and  |
| care. The system will build | public     |     securely for         |
| on existing public and      | docket.    |     relevant product     |
| private efforts through     |            |     safety information.  |
| multiple, broad-based,      |            |                          |
| public-private              |            | -   Establish            |
| collaborations.             |            |     methodologies to use |
|                             |            |     Sentinel data to     |
|                             |            |     support epidemiology |
|                             |            |     and other safety     |
|                             |            |     studies.             |
+-----------------------------+------------+--------------------------+

The modernized post-market safety related IT systems will ensure the
best collection, evaluation, and management of the vast quantity of
safety data that may be received by the FDA as noted below in figure 13.
Improvement in the infrastructure will support access to and the
analyses of externally linked databases, as well as enhancement of the
FDA's AERS and safety signal detection and management tools. The
MedWatch Plus initiative will result in a common FDA portal for
electronic receipt of adverse event reports from the public and will
provide direct electronic transfer of these reports to the database and
data analysis tools.

In addition to the enhancement and modernization of the drug safety
systems, the FDA will be expanding CBER's and CDER's acquisition/access
and analyses of externally-linked databases for purposes of targeted, or
active, post-marketing surveillance. The figure below includes both
passive surveillance data sources, and active surveillance data sources
that FDA will use to ensure drug safety.

**Figure 13: Sources of both Passive and Active Surveillance Data for
FDA Drug Safety Activities**
![](media/image20.wmf){width="4.995833333333334in"
height="3.751388888888889in"}

# 7.0 Appendices

## 7.1 PDUFA IV Metrics

The PDUFA IV Information Technology Performance Goals Metrics and
Measures subsection (Section XIV, D) states, 'FDA will measure progress
toward achievement of the objectives defined in PDUFA IT Goal A.' One of
the measures the FDA has agreed to track and report on is spending on
common IT systems, item 3 under the Metrics and Measures subsection. It
states 'Annual spending on maintenance of legacy IT systems and IT
systems that are common across the organizational divisions
participating in the process for the review of human drug applications.'
The FDA will report on the progress towards a common PDUFA IT
environment by reporting on the percentage of funding used for Common IT
Systems and Legacy IT Systems. Each of these categories is defined
below.

> **Common IT Systems** -- Development & maintenance spending on
> software applications, tools, and other products that both CDER and
> CBER use or plan to use to receive, track, and review PDUFA
> submissions. In addition, enterprise architecture activities and IT
> infrastructure consolidation activities are incorporated into this
> category of spending.
>
> **Legacy IT Systems** -- Development & maintenance spending on
> software applications that are used by a single Center and that
> overlaps with software functionality performed by another Center.
> These systems are not part of the target enterprise architecture.

The FDA will report on progress towards a fully electronic submission
process by reporting on NDA, BLA, and IND submissions that are totally
electronic and submitted through the FDA Electronic Submissions Gateway.
The FDA will provide overall progress towards this objective including
information based on the type of submissions. In addition, electronic
standards based submissions will be reported that fail to comply with
FDA electronic submission standards across categories of failure or
problem type.

## 7.2 PDUFA Information Management/IT Goals and Objectives

INFORMATION TECHNOLOGY GOALS (Section XIV)

**A. Objectives**

1\. FDA is committed to achieve the long-term goal of an automated
standards-based information technology (IT) environment for the
exchange, review, and management of information supporting the process
for the review of human drug applications throughout the product life
cycle. Towards this goal, FDA will work toward the accomplishment of the
following objectives by the end of FY 12:

> a\) Develop and periodically update an IT plan, as defined in Sections
> B) and C) below, covering a rolling five-year planning horizon.
>
> b\) Develop, implement, and maintain new information systems
> consistently across all organizational divisions participating in the
> process for the review of human drug applications, and in compliance
> with the IT plan, the FDA's program-wide governance process, the FDA's
> target enterprise architecture, and with HHS enterprise architecture
> standards. The consistency of development, implementation, and
> maintenance of new information systems will be determined by the FDA
> based on considerations of program efficiency and effectiveness.
> Emphasis will be placed on the consistency of interactions with
> regulated parties and other external stakeholders.
>
> c\) Update technical specifications and IT-related guidance documents
> as necessary to reflect consistent program-wide implementation of new
> information systems supporting electronic information exchange between
> FDA and regulated parties and other external stakeholders.
>
> d\) Extend the capability of the secure electronic single point of
> entry to include two-way transmission of regulatory correspondence.
>
> e\) Establish an automated standards-based regulatory submission and
> review environment for INDs, NDAs, and BLAs, and their supplements,
> that enables the following functions over the life cycle of the
> product:
>
> \(1\) Electronic IND, NDA, and BLA submissions received by FDA can be
> archived to enable retrieval through standardized automated links;
>
> \(2\) Electronic IND, NDA, and BLA submissions can include
> cross-references to previously submitted electronic materials through
> standardized automated links; and
>
> \(3\) Archived electronic IND, NDA, and BLA submissions can be
> retrieved through standardized automated links.
>
> f\) Establish a system for electronic exchange and management of human
> drug labeling information in a modular manner (e.g., at the label
> section level) that is based on FDA standards and that enables
> revision tracking.
>
> g\) Establish standards-based information systems to support how FDA
> obtains and analyzes post-market drug safety data and manages emerging
> drug safety signals, as described in Section VIII addressing the
> enhancement and modernization of the FDA drug safety system.

**B. Communications and Technical Interactions**

1\. FDA will develop and periodically update a five-year IT plan for
improving the automation of business processes and acquiring and
maintaining information systems to achieve the objectives defined above
in PDUFA IT Goal A. The plan will include measurable or observable
milestones toward achievement of those objectives.

2\. The IT plan will be reviewed and approved through the appropriate
FDA governance process to ensure it conforms to the Agency's overall
long-term automation strategy.

3\. The IT plan will be drafted, published on the FDA web site, and
updated as follows:

> a\) FDA will publish a draft of the IT plan by December 31, 2007. At
> that time, FDA will solicit and consider comments from the public on
> the draft IT plan. The public comment period will be at least 45
> calendar days. FDA will complete revisions to the IT plan and publish
> the final version no later than May 30, 2008.
>
> b\) FDA will conduct an annual assessment of progress against the IT
> plan and publish on the FDA web site a summary of the assessment
> within 2 months after the close of each fiscal year.
>
> c\) FDA will publish updates to the IT plan as FDA deems necessary to
> achieve the objectives defined in PDUFA IT Goal A. FDA will publish on
> the FDA web site draft revisions to the IT plan; solicit comments from
> the public on those draft revisions; and consider the public comments
> before completing and publishing updates to the IT plan.

4\. The FDA and industry stakeholders will meet on a quarterly basis to
discuss ongoing implementation of the IT plan, status of IT metrics as
available, and potential impacts that future activities may have on
stakeholders. These meetings will also be used to discuss potential FDA
revisions to the IT plan based on operational experience.

**C. Standards and IT Plan**

The IT plan referenced in PDUFA IT Goal B will provide a vision for FDA
standards and technical infrastructure supporting the process for the
review of human drug applications and will address the following:

1\. A description of the scope and approach for an evaluation and design
of the target enterprise architecture necessary to achieve the
objectives defined in PDUFA IT Goal A.

2\. The business processes targeted for automation to achieve
business-driven objectives.

3\. Which electronic data standards, including the associated Standards
Development Organization, are being considered for adoption or
development. (Note: The FDA's process for adopting or developing
standards includes the consideration of existing open consensus
standards prior to the development of new standards. FDA participates in
international Standards Development Organizations and supports global
harmonization of data standards through open structured processes.)

4\. Implementation of information systems that are based on the
electronic data standards.

5\. Training for system users, stakeholder adoption, and communications
for transitioning to new or reengineered information systems supporting
the process for the review of human drug applications.

6\. A description of FDA's processes for

> a\) evaluating business processes for electronic information exchange
> between FDA and regulated parties or external stakeholders;
>
> b\) evaluating, adopting or developing electronic data standards for
> information exchange between FDA and regulated parties or external
> stakeholders; and []{.mark}
>
> c\) developing, piloting, and deploying information systems that use
> those standards in supporting the process for the review of human drug
> applications.

**D. Metrics and Measures**

FDA will measure progress toward achievement of the objectives defined
in PDUFA IT Goal A. Measures will include:

1\. The number and percentage of IND, NDA, and BLA submissions received
in valid electronic format in compliance with FDA standards, categorized
by types of submissions. Increasing the number and percentage of IND,
NDA, and BLA submissions received in valid electronic format is a goal
that is supported by the FDA and industry stakeholders. Achievement of
this goal requires the cooperation of regulated industry. To support the
assessment of this goal, the following information will be tracked and
reported at least annually:

> a\) Total number of submissions categorized by type of submission;
>
> b\) Total number of submissions in valid electronic format in
> compliance with FDA standards
>
> c\) Total number of submissions received through the secure electronic
> single point of entry versus other methods; and
>
> d\) Total number of submissions received substantially on paper.

2\. Total number of standards-based electronic submissions that fail to
comply with FDA electronic submission standards, along with a
distribution of these submission failures across categories of failure
or problem type.

3\. Annual spending on maintenance of legacy IT systems and IT systems
that are common across the organizational divisions participating in the
process for the review of human drug applications.

4\. Other measures and milestones to be identified in the IT plan
addressed under Sections B and C above.

**Drug Safety Goals (Section VIII)**

**A. Development of 5-year plan, and Communications and Technical
Interactions**

> 1\. The FDA will develop and periodically update a 5-year plan
> describing activities that will lead to enhancing and modernizing
> FDA's drug safety activities/system. The activities described in the
> 5-year plan will include:
>
> c\) Expanding CBER/CDER's database acquisition and use for the
> purposes of targeted post-marketing surveillance and epidemiology;
>
> e\) Improving post-market IT systems (e.g., AERS 2, safety tracking
> system, and opportunities for linked data management).

**B. Conduct and support activities designed to modernize the process of
pharmacovigilance**

> 3\. Expanding Database Resources: A critical part of the
> transformation of the drug safety program is maximizing the usefulness
> of tools used for adverse event signal detection and risk assessment.
> To achieve this end, data other than spontaneous reports, including
> population-based epidemiological data and other types of observational
> data resources will be used and evaluated. Access to these types of
> data will expand the FDA's capability to carry out targeted
> post-marketing surveillance, look at class effects of drugs, and
> potentially carry out signal detection using data resources other than
> reports from AERS system.   PDUFA funds will be used to obtain access
> to additional databases and program staffing with epidemiologists and
> programmers who are able to use these new resources.

**D. Other Activities**

FDA will establish the following standards-based information systems to
support how FDA obtains and analyzes post-market drug safety data and
manages emerging drug safety information:

> 1\. Enhanced adverse event reporting system and surveillance tools;
>
> 2\. IT infrastructure to support access and analyses of
> externally-linked databases; and
>
> 3\. Workflow tracking system.

## 7.3 PDUFA IV Goals Mapped to FDA Initiatives

(On next page)

![](media/image1.wmf) Note: Goals section 'B. Communications and
Technical Interactions' and 'D. Metrics and Measures' are not included
on the goals listed above. Both goals are discussed in the plan and do
not directly map to programs.

## 7.4 Business Review Boards 5-year Goals, Priorities and Current Projects

**[Post-Market Safety]{.underline}**

**5-year goal:**

-   Strengthen capability to rapidly identify, assess and mitigate
    safety problems

**Priorities:**

-   Develop electronic receipt capabilities (i.e. improve receipt of
    spontaneous reporting, create a usable receipt interface, adopt,
    develop & implement data standards HL7 ICSR & SPL)

-   Enhance exploratory data analysis (i.e., strengthen signal detection
    & management of spontaneous reports)

-   Harmonize terminologies (i.e. create or adopt common terminology
    reference sources, implement terminology standards for all FDA
    product)

-   Improve knowledge base systems (i.e. Increase capacity to archive
    and search data & information, implement MedWatch plus -- FAERS)

-   Create supporting rule making (i.e. modify & update regulatory
    documentation (rules & guidance) to reduce / eliminate paper
    submissions)

**Major Project(s):**

-   MedWatch plus, including MedWatch plus portal project and FDA
    Adverse Event Reporting System (FAERS)

**[Product Quality and Compliance]{.underline}**

**5-year goal:**

-   Assure product quality and compliance through timely access to and
    better use of accurate FDA-related entity information across the
    Agency (entities are firms, facilities, points of contact, products,
    components/ingredients)

**Priorities:**

-   Implement Harmonized Business Processes and Systems for
    Identification and Tracking of FDA-Related Firms and Facilities
    across the Agency

-   Implement Harmonized Business Processes and Systems for
    Identification and Tracking of FDA-Regulated Products and
    Components/Ingredients across the Agency

-   Provide Single Portal Access to Comprehensive Entity Information

-   Harmonize FDA and Customs and Border Protection (CBP) Processes in
    order to Ensure Import Data Quality and Completeness

-   Enhance Automation of Import Screening Processes

**Major Project(s):**

-   Harmonized Inventory of FDA-Related Entities, including registration
    and listing.

**[Pre Market Review]{.underline}**

**5-year goal:**

-   Implement a standards-based end-to-end fully electronic receipt,
    review, dissemination and archival environment

**Priorities:**

-   Create or Adopt Standardized Structure and Formats for Data and
    Documents

-   Adopt HL7 Regulatory Product Submission (RPS) Standard for all FDA
    Regulated Products

-   Improve and Automate Electronic Receipt Functions

-   Improve Search Tools and Capabilities

-   Improve Automation of Workflow

-   Improve Document Management

**Major Project(s):**

-   Common Electronic Document Room (EDR)

-   Regulated Product Submission (RPS)

**[Administrative Services]{.underline}**

**Priorities:**

-   Human resources

-   Payroll

-   Budget formulation and planning

-   Tracking systems

-   Travel

They are now in the process of identifying priority initiatives.

**[Scientific Computing / Computational Science]{.underline}**

The SC/CS BRB has recently formed and is in the process of identifying
current best practices and areas that need improvement. They will define
their priorities in early 2008.

##  7.5 Summary Schedule

![](media/image2.wmf){width="6.1194444444444445in"
height="4.591666666666667in"}

## 

## 

## 7.6 Acronym List

  ------------------------ ----------------------------------------------
  (ICH, VICH, GHTF)        **Global regulatory standards groups**

  ADaM                     Analysis Data Model

  AERS                     Adverse Events Reporting System

  ANSI                     American Nation al Standards Institute

  BiB                      Bioinformatics Board

  BMT                      Business Modernization / Transformation

  BRBs                     Business Review Boards

  CBER                     Center for Biologics Evaluation and Research

  CDASH                    Clinical Data Acquisition Standards
                           Harmonization

  CDC                      Center for Disease Control

  CDER                     Center for Drug Evaluation and Research

  CDISC                    Clinical Data Interchange Standards Consortium

  CIO                      Chief Information Officer

  CRADA                    Cooperative Research and Development Agreement

  DHHS                     Department of Health and Human Services

  DSC                      Data Standards Council

  DT                       Developmental Test

  EA                       Enterprise Architecture

  eCTD                     electronic Common Technical Document

  EDSR                     Electronic Document Submission and Review

  ELR                      Electronic Labeling Rule

  EPLC7                    Enterprise Performance Life Cycle

  ESG                      Electronic Submissions Gateway

  EVS                      Enterprise Vocabulary Services

  FASTAR                   FDA Advanced Submission Tracking and Review
                           Framework

  FDA                      Food and Drug Administration

  FHA                      Federal Health Architecture

  FMAMA                    FDA Modernization Act

  FTE                      Full Time Equivalent

  GGP                      Good Guidance Practice

  HL7                      Health Level Seven

  ICSR                     Individual Case Safety Report

  IM                       Information Management

  ISO                      International Standards Organization

  IT                       Information Technology

  MaPP                     Manual of Policies and Procedures

  NCI                      National Cancer Institute

  NCPDP                    National Council for Prescription Drug
                           Programs

  OC                       Office of the Commissioner

  OCIO                     Office of the Chief Information Officer

  ODM                      Operational Data Model

  OMB                      Office of Management and Budget

  OPL                      Office of Planning

  ORA                      Office of Regulatory Affairs

  OT                       Operational Test

  PDUFA                    Prescription Drug User Fee Act

  PLR                      Physicians Labeling Rule

  SDLC                     System Development Lifecycle

  SDO                      Standards Development Organization

  SDTM                     Study Data Tabulation Model

  SEND                     Standard for Exchange of Nonclinical Data

  SIT                      System Integration Test

  SOPP                     Standard Operating Procedures and Policies

  SPL                      Structured Product Labeling

  SQT                      System Qualification Tests

  UNII                     Unique Ingredient Identifiers

  **VCS**                  Voluntary Consensus Standard
  ------------------------ ----------------------------------------------

[^1]: OMB Circular A-119

[^2]: An example of a standard widely used within FDA, which is not a
    VCS, is the portable document format (PDF) standard for electronic
    documents. Although a proprietary standard, it is in widespread use
    and no comparable VCS currently exists that meets the business
    requirements.