Critical Path 
Innovation Meetings  
 
Guidance for Industry 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
U.S. Department of Health and Human Services 
Food and Drug Administration 
Center for Drug Evaluation and Research (CDER) 
 
April 2015 
Procedural 
 
   
 
 Critical Path 
Innovation Meetings  
 
Guidance for Industry 
 
 
 
 
 
Additional copies are available from: 
 
Office of Communications, Division of Drug Information 
Center for Drug Evaluation and Research 
Food and Drug Administration 
10001 New Hampshire Ave., Hillandale Bldg., 4th Floor  
Silver Spring, MD  20993-0002 
Tel: 855-543-3784 or 301-796-3400; Fax: 301-431-6353; Email: druginfo@fda.hhs.gov 
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm 
 
 
 
 
 
 
 
 
 
 
 
U.S. Department of Health and Human Services 
Food and Drug Administration 
Center for Drug Evaluation and Research (CDER) 
 
April 2015 
Procedural 
   
TABLE OF CONTENTS 
 
I.  INTRODUCTION............................................................................................................. 1 
II.  BACKGROUND ............................................................................................................... 2 
III.  SCOPE, CONTENT, AND OUTCOMES ...................................................................... 2 
A.  Scope ............................................................................................................................................... 2 
B.  Potential Topics for a CPIM ......................................................................................................... 2 
C.  Outcomes ........................................................................................................................................ 3 
IV.  PROCEDURES ................................................................................................................. 4 
A.  General Considerations ................................................................................................................. 4 
B.  CPIM Requests .............................................................................................................................. 4 
C.  CPIM Preparation Packages ........................................................................................................ 4 
D.   Postmeeting Summaries ................................................................................................................ 5 
i 
  Contains Nonbinding Recommendations 
 
Critical Path Innovation Meetings 
1
Guidance for Industry  
 
 
 
 
 
This guidance represents the current thinking of the Food and Drug Administration (FDA or Agency) on 
this topic.  It does not create any rights for any person and is not binding on FDA or the public.  You can 
use an alternative approach if it satisfies the requirements of the applicable statutes and regulations.  To 
discuss an alternative approach, contact the FDA staff responsible for this guidance as listed on the title 
page.   
 
 
 
 
 
I.  INTRODUCTION  
 
This guidance describes the purpose, scope, documentation, and administrative procedures for a 
Critical Path Innovation Meeting (CPIM), including how to request such a meeting. The CPIM is a 
means by which the Center for Drug Evaluation and Research (CDER or we) and investigators from 
industry, academia, patient advocacy groups, and government can communicate to improve 
efficiency and success in drug development. The goals of the CPIM are to discuss a methodology or 
technology proposed by the meeting requester and for CDER to provide general advice on how this 
methodology or technology might enhance drug development. CDER will identify some of the larger 
gaps in existing knowledge that requesters might consider addressing in the course of their work. The 
discussions and background information submitted through the CPIM are nonbinding on both FDA 
and CPIM requesters.  
 
This guidance provides some examples of topics appropriate for a CPIM. It also describes the 
information that should be provided to CDER in preparation for a meeting and potential outcomes 
from the CPIM. 
 
In general, FDAs guidance documents do not establish legally enforceable responsibilities.  
Instead, guidances describe the Agencys current thinking on a topic and should be viewed only 
as recommendations, unless specific regulatory or statutory requirements are cited.  The use of 
the word should in Agency guidances means that something is suggested or recommended, but 
not required.  
 
 
 
                                                 
1 This guidance has been prepared by the Office of Translational Sciences and the Office of New Drugs in the 
Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration.  
  1  Contains Nonbinding Recommendations 
II.  BACKGROUND 
 
In 2004, FDA published the report Innovation or Stagnation: Challenge and Opportunity on the 
Critical Path to New Medical Products, which called attention to a slowdown . . . in innovative 
medical therapies reaching patients. 2  The report identified several areas of product 
development in need of improvement, including technical methods such as animal or computer-
based predictive models, biomarkers for safety and effectiveness, and new clinical evaluation 
techniques, and cited a need to create better tools for developing medical technologies [and] a 
knowledge base built not just on ideas from biomedical research, but on reliable insights into the 
pathway to patients.  Through the Critical Path Initiative (CPI), FDA works to foster innovation 
through collaborations among government, industry, academia, patient advocacy groups, and 
other external stakeholders.  CDER has created programs such as the Voluntary eXploratory 
Data Submission program (VXDS)3 and Drug Development Tools (DDT) qualification 
programs4,5 for biomarkers, clinical outcome assessments, and animal models under the Animal 
Rule.6  FDA continues to work to identify opportunities to advance drug development efforts.7  
 
III.  SCOPE, CONTENT, AND OUTCOMES     
 
A.  Scope 
 
The CPIM is broad in scope. It is a general discussion of challenges in drug development and 
innovative strategies to address them. Appropriate FDA experts from CDER offices and other centers 
will participate as resources and time permit. 
 
B.  Potential Topics for a CPIM 
 
Potential topics for a CPIM include, but are not limited to, the following:  
 
                                                 
2 Available at www.fda.gov/ScienceResearch/SpecialTopics/CriticalPathInitiative/CriticalPathOpportunitiesReports 
under Challenges and Opportunities Report  March 2004. 
 
3 See the guidance for industry Pharmacogenomics Data Submissions.  The guidances referenced in this document 
are available on the FDA Drugs guidance Web page at 
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm.  We update 
guidances periodically.  To make sure you have the most recent version of a guidance, check the FDA Drugs 
guidance Web page. 
 
4 See the Drug Development Tools (DDT) Qualification Programs Web page, 
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/. 
 
5 See the guidance for industry and FDA staff Qualification Process for Drug Development Tools.  
 
6 See the draft guidance for industry Product Development Under the Animal Rule.  When final, this guidance will 
represent the FDAs current thinking on this topic. 
 
7  Identifying CDERs Science and Research Needs Report (2011), available at 
http://www.fda.gov/Drugs/ScienceResearch/ucm264327.htm. 
 
  2  Contains Nonbinding Recommendations 
  Biomarkers in the early phase of development and not yet ready for the Biomarker 
Qualification Program (BQP):  The CPIM can be a venue for a discussion of the 
potential of proposed biomarkers.  The discussion can help requesters understand some of 
the more important questions FDA may have related to proposed biomarkers and prepare 
prospective submitters for the BQP.  
  Clinical outcome assessments in the early phase of development and not yet ready 
for the Clinical Outcome Assessment Qualification Program: Clinical outcome 
assessments (COAs) include patient-reported outcomes, clinician-reported outcomes, 
observer-reported outcomes, and performance outcomes.  The CPIM can be a venue for a 
discussion of the potential approaches to developing COAs to provide evidence of 
treatment benefit to support marketing approval and labeling claims.  The discussion can 
help requesters understand the needs and goals for COA qualification and answer 
questions they may have related to the development or selection of COAs in preparation 
for the qualification process. 
  Natural history study designs and implementation: The CPIM can assist in the design 
of natural history studies to increase the potential for the data generated by these studies 
to help in the design of interventional clinical trials and drug development programs. 
 
  Emerging technologies (other than manufacturing technology) or new uses of 
existing technologies:  The CPIM may help developers understand the strengths and 
weaknesses of these technologies in relation to the various potential uses at different 
stages of drug development. 
  Innovative conceptual approaches to clinical trial design and analysis: The CPIM can 
be a forum for the discussion of conceptual and general regulatory issues concerning 
various design and analytical approaches to clinical trials. 
 
FDA will not give regulatory advice on specific product development programs at a CPIM.  
Meetings relating to a specific drug development program should be requested through the 
appropriate review division in accordance with the FDA guidance for industry Formal Meetings 
Between the FDA and Sponsors or Applicants.  The CPIM is not intended to replace meetings 
that should be held through formal DDT qualification programs (see Background).  Requesters 
interested in discussions related to the DDT qualification programs should contact the relevant 
DDT qualification program directly.  CPIMs are not intended to discuss therapeutic product-
specific data or result in binding agreements.  
 
C.  Outcomes 
 
Through the CPIM, CDER intends to provide our perspective on the potential for use of 
proposed new tools and methods in drug development.  Based on CDER experience, we may 
advise requesters of issues to consider in pursuing their work, propose joint efforts through 
existing consortia, or discuss the potential to form new consortia.  The CPIM may also lead to 
recommendations for public workshops or other avenues for engaging with the wider scientific 
community.  CDER expects that the CPIM will also provide FDA with exposure to methods and 
techniques that may have value in drug development.   
 
  3  Contains Nonbinding Recommendations 
The CPIM is not intended to be a venue for ongoing, recurrent discussions with FDA.  However, 
we will consider requests for a subsequent meeting on a given topic on a case-by-case basis.    
 
CDER will post the topics discussed on a quarterly basis at 
http://www.fda.gov/drugs/developmentapprovalprocess/druginnovation/ucm395888.htm 
 
 
IV.  PROCEDURES 
 
A.  General Considerations 
 
The CPIM is administered by the Office of Translational Sciences (OTS), which is the point of 
contact for all communications.  
 
CDER will discuss with the requester, as needed, whether the issues raised in the CPIM request 
would be better addressed through other venues (see Scope).  A request for a CPIM should not 
be submitted to a particular regulatory application (e.g., investigational new drug application 
(IND), new drug application (NDA), biologics license application (BLA)).    
 
Requesters should clearly identify confidential or other proprietary information.  
 
B.  CPIM Requests  
 
The CPIM request should be submitted electronically.  Information about how to submit the 
request electronically is available at: 
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm395888.htm . 
 
The request should include the following information: 
  Name of requester  
  Date of request 
  Description of organization 
  A document, no more than 5-6 pages in length, containing the background and purpose of 
the meeting, steps taken in advancing the project, and specific questions for FDA (if 
needed) 
  Desired outcome of the meeting 
 
Requests should provide enough information appropriate for a discussion of conceptual drug 
development issues and should not be focused on a particular regulatory submission.  
CDER plans to respond to the requester within 14 days of receipt of the meeting request and 
discuss with the requester the appropriateness of the CPIM.   
 
C.  CPIM Preparation Packages 
  
If a request for a CPIM is granted, the requester should submit a final preparation package no 
later than 2 weeks before the meeting date.   
 
  4  Contains Nonbinding Recommendations 
The CPIM preparation package should be submitted to the CDER Document Room and include 
the following elements: 
  Objective of the meeting 
  Proposed agenda 
  Presentation slides, if any 
  Proposed attendees and respective affiliations 
 
Send the CPIM package to the following address: 
Food and Drug Administration 
10903 New Hampshire Avenue 
WO Building 21-4547 
Silver Spring, MD 20993-0002 
 
 
D.   Postmeeting Summaries 
 
We will send a meeting summary to the requester within 60 days of the meeting.  
 
 
 
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