Source: https://regulations.justia.com/regulations/fedreg/2013/01/15/2013-00607.html
Timestamp: 2020-02-19 23:46:17
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Matched Legal Cases: ['art 2', 'art 314', 'art 601', 'art 15', 'art 15', 'art 15', '§ 15', 'art 15', 'art 10', 'art 10', '§ 10', '§ 15', 'art 15', '§ 15', 'art 314', 'art 601', 'art 15', 'art 15', 'art 15', 'art 10', 'art 10', 'art 15']

Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need; Public Hearing; Request for Comments, 3005-3008 [2013-00607] :: Food And Drug Administration :: Department Of Health And Human Services :: Regulation Tracker :: Justia
Justia Regulation Tracker Department Of Health And Human Services Food And Drug Administration Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need; Public Hearing; Request for Comments, 3005-3008 [2013-00607]
Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need; Public Hearing; Request for Comments, 3005-3008 [2013-00607]
Download as PDF Federal Register / Vol. 78, No. 10 / Tuesday, January 15, 2013 / Notices 3005 TABLE 3—NEW ENTRIES TO THE LIST OF RECOGNIZED STANDARDS—Continued Recognition No. Title of standard 1 Reference No. and date L. Sterility 14–376 ........... 14–377 ........... 1 All Sterilization of health care products—Moist heat—Part 2: Guidance on the application of ANSI/AAMI/ISO 17665–1. Standard Test Method for Using Aerosol Filtration for Measuring the Performance of Porous Packaging Materials as a Surrogate Microbial Barrier. ASTM F2638–12. standard titles in this table conform to the style requirements of the respective organizations. IV. List of Recognized Standards FDA maintains the Agency’s current list of FDA recognized consensus standards in a searchable database that may be accessed directly at FDA’s Internet site at http:// www.accessdata.fda.gov/scripts/cdrh/ cfdocs/cfStandards/search.cfm. FDA will incorporate the modifications and minor revisions described in this notice into the database and, upon publication in the Federal Register, this recognition of consensus standards will be effective. FDA will announce additional modifications and minor revisions to the list of recognized consensus standards, as needed, in the Federal Register once a year, or more often, if necessary. V. Recommendation of Standards for Recognition by FDA Any person may recommend consensus standards as candidates for recognition under section 514 of the FD&C Act by submitting such recommendations, with reasons for the recommendation, to the contact person (See FOR FURTHER INFORMATION CONTACT). To be properly considered, such recommendations should contain, at a minimum, the following information: (1) Title of the standard, (2) any reference number and date, (3) name and address of the national or international standards development organization, (4) a proposed list of devices for which a declaration of conformity to this standard should routinely apply, and (5) a brief identification of the testing or performance or other characteristics of the device(s) that would be addressed by a declaration of conformity. srobinson on DSK4SPTVN1PROD with ANSI/AAMI/ISO TIR 17665–2:2009. VI. Electronic Access You may obtain a copy of ‘‘Guidance on the Recognition and Use of Consensus Standards’’ by using the Internet. The Center for Devices and Radiological Health (CDRH) maintains a site on the Internet for easy access to information including text, graphics, and files that you may download to a personal computer with access to the VerDate Mar<15>2010 17:00 Jan 14, 2013 Jkt 229001 Internet. Updated on a regular basis, the CDRH home page includes the guidance as well as the current list of recognized standards and other standards-related documents. After publication in the Federal Register, this notice announcing ‘‘Modification to the List of Recognized Standards, Recognition List Number: 030’’ will be available on the CDRH home page. You may access the CDRH home page at http://www.fda.gov/ MedicalDevices. You may access ‘‘Guidance on the Recognition and Use of Consensus Standards,’’ and the searchable database for ‘‘FDA Recognized Consensus Standards’’ at http://www.fda.gov/ MedicalDevices/ DeviceRegulationandGuidance/ Standards. This Federal Register document on modifications in FDA’s recognition of consensus standards is available at http://www.fda.gov/MedicalDevices/ DeviceRegulationandGuidance/ Standards/ucm123792.htm. VII. Submission of Comments and Effective Date Interested persons may submit to the contact person (see FOR FURTHER INFORMATION CONTACT) either electronic or written comments regarding this document. It is only necessary to send one set of comments. Comments are to be identified with the docket number found in brackets in the heading of this document. FDA will consider any comments received in determining whether to amend the current listing of modifications to the list of recognized standards, Recognition List Number: 030. These modifications to the list or recognized standards are effective upon publication of this notice in the Federal Register. Dated: January 9, 2013. Leslie Kux, Assistant Commissioner for Policy. [FR Doc. 2013–00605 Filed 1–14–13; 8:45 am] BILLING CODE 4160–01–P PO 00000 Frm 00067 Fmt 4703 Sfmt 4703 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2012–N–1248] Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need; Public Hearing; Request for Comments AGENCY: Food and Drug Administration, HHS. Notice of public hearing; request for comments. ACTION: The Food and Drug Administration (FDA) is announcing a public hearing to obtain input on a potential new pathway to expedite the development of drugs, including biological products, for serious or lifethreatening conditions that would address an unmet medical need. The drug’s safety and effectiveness would be studied in a smaller subpopulation of patients with more serious manifestations of a condition. Such a pathway could involve smaller and more rapid clinical trials than would occur if the drug were studied in a broader group of patients with a wide range of clinical manifestations. The labeling of drugs approved using this pathway would make clear that the drug is narrowly indicated for use in limited, well-defined subpopulations in which the drug’s benefits have been shown to outweigh its risks. The purpose of the public hearing is to obtain information and comments from the public on the need for and feasibility of this pathway and its potential advantages and disadvantages. SUMMARY: Dates and Time: The public hearing will be held on February 4 and 5, 2013, from 9 a.m. to 4 p.m. The public hearing may be extended or may end early depending on the level of public participation. Attendance, Presentations, and Comments: Individuals who wish to attend or present at the public hearing must register on or before 5 p.m. e.s.t. on January 22, 2013. To register for the DATES: E:\FR\FM\15JAN1.SGM 15JAN1 srobinson on DSK4SPTVN1PROD with 3006 Federal Register / Vol. 78, No. 10 / Tuesday, January 15, 2013 / Notices public hearing, email your registration information to ExpeditedPathwayPublic Mtg@fda.hhs.gov. Section IV of this document provides attendance and registration information. Either electronic or written comments will be accepted after the hearing until March 1, 2013. ADDRESSES: The public hearing will be held at FDA’s White Oak Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the Great Room (Rm. 1503), Silver Spring, MD, 20993– 0002. Entrance for the public meeting participants (non-FDA employees) is through Building 1 where routine security check procedures will be performed. For parking and security information, please refer to http://www. fda.gov/AboutFDA/WorkingatFDA/ BuildingsandFacilities/WhiteOak CampusInformation/ucm241740.htm. Submit electronic comments to http://www.regulations.gov. Submit written comments to the Division of Dockets Management (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. FOR FURTHER INFORMATION CONTACT: Jonas Santiago, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993–0002, 301–796–5346, Fax: 301–847–3529, email: Expedited PathwayPublicMtg@fda.hhs.gov; or Stephen Ripley, Center for Biologics Evaluation and Research (HFM–17), Food and Drug Administration, 1401 Rockville Pike, Suite 200N, Rockville, MD 20852–1448, 301–827–6210. SUPPLEMENTARY INFORMATION: FDA is announcing a public hearing to obtain input on a potential new pathway for approving drugs, including biological products, targeted at serious or lifethreatening conditions and intended to address an unmet medical need. The drug’s safety and effectiveness would be studied in a smaller subpopulation of patients with more serious manifestations of a condition. Such a pathway could involve smaller and more rapid clinical trials than would occur if the drug were studied in a broader group of patients with a wide range of clinical manifestations. The labeling of drugs approved using this pathway would make clear that the drug is narrowly indicated for use in limited, well-defined subpopulations in which the drug’s benefits have been shown to outweigh the risks. I. Background In the last two decades, major advances in molecular and cellular VerDate Mar<15>2010 17:00 Jan 14, 2013 Jkt 229001 biology have greatly expanded our understanding of a broad range of complex disease processes and have led to major advances in the treatment of conditions such as cystic fibrosis, HIV, hepatitis C, and multiple sclerosis. In some cases, however, the resourceintensive programs needed for approval of drugs to treat a broad condition with a wide range of clinical manifestations require very large study populations and can hinder the ability to make promising new drugs available in a timely manner to subpopulations of patients with important unmet medical needs. FDA recognizes its role in fostering the application of scientific advances to the treatment of disease through drug development, including the use of novel approaches that can facilitate development of treatment for unmet needs. Traditional drug development programs are designed to evaluate the benefits and risks of treatment with a high degree of precision for the range of manifestations of a disease or condition. Often this will involve studies that expose a large number of patients to the drug, normally for an extended period of time. In some cases, such as when safety issues have arisen with prior drugs in a class, additional trials are needed to help identify serious but infrequent risks. Typically, these studies are needed when there is an expectation that the drug will be used broadly in patients with less severe manifestations of the condition. Existing processes to expedite drug development and review of important new therapies have worked effectively in many circumstances. For example, more than 100 new therapies and indications have been approved under the accelerated approval process (21 CFR part 314, subpart H; 21 CFR part 601, subpart E).1 In addition, FDA’s existing flexibility in applying the statutory requirements for approval has effectively facilitated development of drugs for conditions where the entire intended patient population has serious unmet medical needs. However, FDA believes that it is important to explore the need for and feasibility of a new process focused on developing drugs for subpopulations of patients with serious or life-threatening conditions, including patients with serious or life-threatening infections caused by antibiotic-resistant bacteria. 1 See a list of Center for Drug Evaluation and Research Drug and Biologic Accelerated Approvals as of September 30, 2011, available at http://www. fda.gov/downloads/Drugs/DevelopmentApproval Process/HowDrugsareDevelopedandApproved/ DrugandBiologicApprovalReports/UCM278506.pdf. PO 00000 Frm 00068 Fmt 4703 Sfmt 4703 II. New Pathway FDA is seeking input on a potential new pathway to approve drugs for use in limited, well-defined subpopulations of patients with serious or lifethreatening conditions for whom the benefits of the drug have been shown to outweigh the risks. The pathway could include product labeling with a specific designation to make clear that the drug indication is limited to the narrow subpopulation and the rationale for limiting use to that population. The pathway also might provide for the designation and an appropriate logo to appear on a drug’s container label. This designation could be designed to inform the health care community, including practitioners, payers, and patients, of compelling reasons to carefully manage use of such drugs, limiting use to appropriate patients, as the benefit-risk profile only warrants use in the identified subpopulation. In addition, the potential new pathway could be used to help reduce the development of resistance to important antibacterial drugs by limiting their use to those patients in whom use is appropriate and necessary. This approval of a narrow indication could be broadened if additional data become available which demonstrate the safety and effectiveness of the drug in treating a broader condition or patient population. For example, a drug could be initially approved using this pathway for a narrow subpopulation of patients because of uncertainty about a cardiovascular risk that would not be acceptable in a broad population. If a long-term study subsequently demonstrates that the benefit-risk profile makes the drug appropriate for broader use, the designation could be removed. Alternatively, there may be drugs for which we would not anticipate the possibility of approval in the broader population, such as when there is a known toxicity that, while acceptable in patients with serious manifestations of a condition, would not be appropriate for use in patients with milder manifestations of the condition. The proposed pathway was recommended by the President’s Council of Advisors on Science and Technology (PCAST) in their September 2012 ‘‘Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation,’’ as a way to improve drug evaluation. The PCAST recommendations support the goal of increasing the output of innovative, new medicines for patients with important unmet medical needs, while increasing drug efficacy and safety, through E:\FR\FM\15JAN1.SGM 15JAN1 Federal Register / Vol. 78, No. 10 / Tuesday, January 15, 2013 / Notices industry, academia, and government working together to decrease clinical failure, clinical trial costs, time to market, and regulatory uncertainty.2 srobinson on DSK4SPTVN1PROD with III. Scope of the Public Hearing and Discussion Questions FDA is holding this public hearing to seek input from interested members of the public including patients and consumers, practitioners and other members of the medical community, regulated industry, insurers, and managed care organizations on a potential new pathway to approve drugs shown to be safe and effective in a subpopulation of patients with serious or life-threatening conditions in which an unmet medical need exists. FDA is interested in obtaining information and public comment on the following issues: 1. Considering existing processes to expedite drug development and review of important new therapies (i.e., accelerated approval, fast-track designation), would this new pathway increase the therapeutic options for serious or life-threatening conditions for which an unmet medical need exists? If not, what might be some alternative approaches? 2. Can you identify specific serious or life-threatening conditions for which an unmet medical need exists and for which this approval pathway may benefit subpopulations of patients? 3. What approaches could be undertaken (by FDA or by people or organizations other than FDA) to monitor use of drugs approved under this pathway to determine whether they are being used inconsistent with the terms of approval? What approaches could be undertaken to prevent, manage, or monitor use in a broader population where safety and efficacy has not been demonstrated? For example, if this pathway were adopted to approve new antibacterial drugs when limited use was needed (e.g., to prevent the emergence of further antimicrobial resistance), what other measures (by FDA or by people or organizations other than FDA) might ensure that these products are used appropriately only in the indicated subpopulations? 4. Would this pathway help to address some of the current challenges in antibacterial drug development, particularly for serious or lifethreatening infections for which there is an unmet medical need? 2 For more information on the PCAST Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation, see http://www.whitehouse.gov/sites/default/files/ microsites/ostp/pcast-fda-final.pdf. VerDate Mar<15>2010 17:00 Jan 14, 2013 Jkt 229001 5. This potential pathway could be used to approve drugs for a limited subpopulation based upon smaller clinical trials, when benefit-risk is appropriate for the limited population but safety and efficacy have not been demonstrated for use in a broader population of patients or patients with less severe manifestations of the condition. For the serious or lifethreatening conditions you identified in question 2, what benefit-risk considerations need to be taken into account before and after marketing and how should they be addressed? 6. Would the use of a formal designation and logo to reflect approval under this pathway, with clear labeling of clinical information that only supports use in the indicated subpopulation, but without other constraints from FDA be effective in limiting use to the indicated subpopulation? Why or why not? IV. Attendance, Registration, and Requests for Oral Presentations The public hearing is free and seating will be on a first-come, first-served basis. Attendees, including those not presenting, need to register for the public hearing. If you wish to attend or make an oral presentation during the hearing, you must register by submitting either an electronic or written request received on or before January 22, 2013. (See FOR FURTHER INFORMATION CONTACT.) You must provide your name, title, business affiliation (if applicable), address, telephone and fax numbers, email address, and type of organization you represent (e.g., industry, consumer organization). If requesting to present, you also should submit a brief summary of the presentation, including the discussion question(s) that will be addressed and the approximate time requested for your presentation. FDA has included discussion questions in section III of this document. You should identify the question(s) and the number of each question you wish to address in your presentation. We encourage individuals and organizations with common interests to consolidate or coordinate their presentations to allow adequate time for each request for presentation. FDA will do its best to accommodate requests to speak and will determine the amount of time allotted for each oral presentation, and the approximate time that each oral presentation is scheduled to begin. Persons registered to make an oral presentation should submit to FDA an electronic copy of their presentation and an abstract to ExpeditedPathwayPublic PO 00000 Frm 00069 Fmt 4703 Sfmt 4703 3007 Mtg@fda.hhs.gov on or before January 30, 2013. If you need special accommodations because of a disability, please contact Jonas Santiago (see FOR FURTHER INFORMATION CONTACT) at least 7 days before the meeting. V. Notice of Hearing Under 21 CFR Part 15 The Commissioner of Food and Drugs is announcing that the public hearing will be held in accordance with part 15 (21 CFR part 15). The hearing will be conducted by a presiding officer, who will be accompanied by FDA senior management. Under § 15.30(f), the hearing is informal and the rules of evidence do not apply. No participant may interrupt the presentation of another participant. Only the presiding officer and panel members may question any person during or at the conclusion of each presentation. Public hearings under part 15 are subject to FDA’s policy and procedures for electronic media coverage of FDA’s public administrative proceedings (part 10 (21 CFR part 10, subpart C)). Under § 10.205, representatives of the electronic media may be permitted, subject to certain limitations, to videotape, film, or otherwise record FDA’s public administrative proceedings, including presentations by participants. The hearing will be transcribed as stipulated in § 15.30(b) (see section VII of this document). To the extent that the conditions for the hearing, as described in this notice, conflict with any provisions set out in part 15, this notice acts as a waiver of those provisions as specified in § 15.30(h). VI. Comments Interested persons may submit either electronic comments to http:// www.regulations.gov or written comments regarding this document to the Division of Dockets Management (see ADDRESSES). It is only necessary to send one set of comments. Identify comments with the docket number found in brackets in the heading of this document. In addition, when responding to specific questions as discussed in section III of this document, please identify the question you are addressing. Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to the docket at http:// www.regulations.gov. E:\FR\FM\15JAN1.SGM 15JAN1 3008 Federal Register / Vol. 78, No. 10 / Tuesday, January 15, 2013 / Notices VII. Transcripts Transcripts of the public hearing will be available for review at the Division of Dockets Management (see ADDRESSES) and on the Internet at http:// www.regulations.gov approximately 30 days after the public hearing. A transcript will also be made available in either hard copy or on CD–ROM, upon submission of a Freedom of Information request. Written requests are to be sent to the Division of Freedom of Information (ELEM–1029), Food and Drug Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857. Dated: January 9, 2013. Leslie Kux, Assistant Commissioner for Policy. [FR Doc. 2013–00607 Filed 1–14–13; 8:45 am] BILLING CODE 4160–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Health Resources and Services Administration Agency Information Collection Activities: Proposed Collection: Comment Request ACTION: Notice. In compliance with the requirement for opportunity for public comment on proposed data collection projects (section 3506(c)(2)(A) of Title 44, United States Code, as amended by the Paperwork Reduction Act of 1995, Pub. L. 104–13), the Health Resources and Services Administration (HRSA) publishes periodic summaries of proposed projects being developed for submission to the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995. To request more information on the proposed project or to obtain a copy of the data collection plans and draft instruments, email paperwork@hrsa.gov or call the HRSA Reports Clearance Officer at (301) 443– 1984. HRSA especially requests comments on: (1) The necessity and utility of the proposed information collection for the proper performance of the agency’s SUMMARY: functions, (2) the accuracy of the estimated burden, (3) ways to enhance the quality, utility, and clarity of the information to be collected, and (4) the use of automated collection techniques or other forms of information technology to minimize the information collection burden. Information Collection Request Title: Organ Donation/Transplant Life Stories (OMB No. 0915–xxxx)—[New] Abstract HRSA’s Division of Transplantation (DoT) is the primary entity in the Department of Health and Human Services (HHS) responsible for the Organ Transplant Program established under the National Organ Transplant Act (Pub. L. 98–507, codified at sections 371–377D of the Public Health Service (PHS) Act). Section 377A of the PHS Act authorizes the Secretary of HHS to establish a public education program to increase awareness about organ donation and the need to provide for an adequate rate of such donations. In brief, DoT’s responsibilities are twofold: (1) to provide oversight and guidance to the national organ transplant system in the U.S. including monitoring the Organ Procurement and Transplantation Network and the Scientific Registry of Transplant Recipients, and (2) to implement a program of public and professional education and outreach aimed at increasing the number of organ donors in this country. Many preventable deaths occur each year because of a staggering imbalance between the supply and demand for donor organs. As of November 2012, the national transplant waiting list exceeded 116,000. In 2011, the total number of deceased and living organ donors was only 14,145. These donors enabled 28,538 patients to receive a transplant while 6,693 died waiting. Without successful interventions to increase donation, the disparity between need and supply is likely to be substantially exacerbated, resulting in even more unnecessary deaths. Organdonor.gov is DoT’s primary mechanism for providing the public with information about organ donation. Number of respondents srobinson on DSK4SPTVN1PROD with Form name Number of responses per respondent Among the most visited pages on organdonor.gov are the donor and recipient life stories, which in a recent evaluation study were shown to raise interest on the topic, and, more importantly, persuade people to register as organ donors. To expand this component of organdonor.gov, DoT proposes to develop an application to give organ recipients and donor families the opportunity to voluntarily submit their stories to DoT via a standardized online form. The online form will be posted on organdonor.gov and will collect demographic and contact information, the individual’s donation/ transplant story up to 500 words, a highresolution photo, and a signed authorization. The standardized, electronic form will increase HRSA staff’s ability to process those stories more efficiently. In addition to enabling story submission, the online application process will make the donor and recipient life stories posted on the site searchable by the public to enhance public viewing and understanding of the organ donation process. Submission of a story and completion of the form is voluntary. Overall, this application has the potential to strengthen DoT’s outreach efforts and increase organ donation registration in the United States. Burden Statement: Burden in this context means the time expended by persons to generate, maintain, retain, disclose, or provide the information requested. This includes the time needed to review instructions, to develop, acquire, install and utilize technology and systems for the purpose of collecting, validating and verifying information, processing and maintaining information, and disclosing and providing information, to train personnel and to be able to respond to a collection of information, to search data sources, to complete and review the collection of information, and to transmit or otherwise disclose the information. The total annual burden hours estimated for this Information Collection Request are summarized in the table below. The annual estimate of burden is as follows: Total responses Average burden per response (in hours) Total burden hours Donation/Transplantation Life Story Submission Form ....... 100 1 100 0.25 25 Total .............................................................................. 100 1 100 0.25 25 VerDate Mar<15>2010 17:00 Jan 14, 2013 Jkt 229001 PO 00000 Frm 00070 Fmt 4703 Sfmt 4703 E:\FR\FM\15JAN1.SGM 15JAN1
[Pages 3005-3008]
[FR Doc No: 2013-00607]
Creating an Alternative Approval Pathway for Certain Drugs
Intended to Address Unmet Medical Need; Public Hearing; Request for
hearing to obtain input on a potential new pathway to expedite the
development of drugs, including biological products, for serious or
life-threatening conditions that would address an unmet medical need.
The drug's safety and effectiveness would be studied in a smaller
subpopulation of patients with more serious manifestations of a
condition. Such a pathway could involve smaller and more rapid clinical
trials than would occur if the drug were studied in a broader group of
patients with a wide range of clinical manifestations. The labeling of
drugs approved using this pathway would make clear that the drug is
narrowly indicated for use in limited, well-defined subpopulations in
which the drug's benefits have been shown to outweigh its risks. The
purpose of the public hearing is to obtain information and comments
from the public on the need for and feasibility of this pathway and its
DATES: Dates and Time: The public hearing will be held on February 4
and 5, 2013, from 9 a.m. to 4 p.m. The public hearing may be extended
or may end early depending on the level of public participation.
Attendance, Presentations, and Comments: Individuals who wish to
attend or present at the public hearing must register on or before 5
p.m. e.s.t. on January 22, 2013. To register for the
[[Page 3006]]
public hearing, email your registration information to
ExpeditedPathwayPublicMtg@fda.hhs.gov. Section IV of this document
provides attendance and registration information. Either electronic or
written comments will be accepted after the hearing until March 1,
ADDRESSES: The public hearing will be held at FDA's White Oak Campus,
10903 New Hampshire Ave., Building 31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD, 20993-0002. Entrance for the public
meeting participants (non-FDA employees) is through Building 1 where
routine security check procedures will be performed. For parking and
security information, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
Jonas Santiago, Center for Drug Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002,
301-796-5346, Fax: 301-847-3529, email:
ExpeditedPathwayPublicMtg@fda.hhs.gov;
SUPPLEMENTARY INFORMATION: FDA is announcing a public hearing to obtain
input on a potential new pathway for approving drugs, including
biological products, targeted at serious or life-threatening conditions
and intended to address an unmet medical need. The drug's safety and
effectiveness would be studied in a smaller subpopulation of patients
with more serious manifestations of a condition. Such a pathway could
involve smaller and more rapid clinical trials than would occur if the
drug were studied in a broader group of patients with a wide range of
clinical manifestations. The labeling of drugs approved using this
pathway would make clear that the drug is narrowly indicated for use in
limited, well-defined subpopulations in which the drug's benefits have
been shown to outweigh the risks.
In the last two decades, major advances in molecular and cellular
biology have greatly expanded our understanding of a broad range of
complex disease processes and have led to major advances in the
treatment of conditions such as cystic fibrosis, HIV, hepatitis C, and
multiple sclerosis. In some cases, however, the resource-intensive
programs needed for approval of drugs to treat a broad condition with a
wide range of clinical manifestations require very large study
populations and can hinder the ability to make promising new drugs
available in a timely manner to subpopulations of patients with
important unmet medical needs. FDA recognizes its role in fostering the
application of scientific advances to the treatment of disease through
drug development, including the use of novel approaches that can
facilitate development of treatment for unmet needs.
Traditional drug development programs are designed to evaluate the
benefits and risks of treatment with a high degree of precision for the
range of manifestations of a disease or condition. Often this will
involve studies that expose a large number of patients to the drug,
normally for an extended period of time. In some cases, such as when
safety issues have arisen with prior drugs in a class, additional
trials are needed to help identify serious but infrequent risks.
Typically, these studies are needed when there is an expectation that
the drug will be used broadly in patients with less severe
manifestations of the condition.
Existing processes to expedite drug development and review of
important new therapies have worked effectively in many circumstances.
For example, more than 100 new therapies and indications have been
approved under the accelerated approval process (21 CFR part 314,
subpart H; 21 CFR part 601, subpart E).\1\ In addition, FDA's existing
flexibility in applying the statutory requirements for approval has
effectively facilitated development of drugs for conditions where the
entire intended patient population has serious unmet medical needs.
However, FDA believes that it is important to explore the need for and
feasibility of a new process focused on developing drugs for
subpopulations of patients with serious or life-threatening conditions,
including patients with serious or life-threatening infections caused
by antibiotic-resistant bacteria.
\1\ See a list of Center for Drug Evaluation and Research Drug
and Biologic Accelerated Approvals as of September 30, 2011,
available at http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/UCM278506.pdf.
FDA is seeking input on a potential new pathway to approve drugs
for use in limited, well-defined subpopulations of patients with
serious or life-threatening conditions for whom the benefits of the
drug have been shown to outweigh the risks. The pathway could include
product labeling with a specific designation to make clear that the
drug indication is limited to the narrow subpopulation and the
rationale for limiting use to that population. The pathway also might
provide for the designation and an appropriate logo to appear on a
drug's container label.
This designation could be designed to inform the health care
community, including practitioners, payers, and patients, of compelling
reasons to carefully manage use of such drugs, limiting use to
appropriate patients, as the benefit-risk profile only warrants use in
the identified subpopulation. In addition, the potential new pathway
could be used to help reduce the development of resistance to important
antibacterial drugs by limiting their use to those patients in whom use
This approval of a narrow indication could be broadened if
additional data become available which demonstrate the safety and
effectiveness of the drug in treating a broader condition or patient
population. For example, a drug could be initially approved using this
pathway for a narrow subpopulation of patients because of uncertainty
about a cardiovascular risk that would not be acceptable in a broad
population. If a long-term study subsequently demonstrates that the
benefit-risk profile makes the drug appropriate for broader use, the
designation could be removed. Alternatively, there may be drugs for
which we would not anticipate the possibility of approval in the
broader population, such as when there is a known toxicity that, while
acceptable in patients with serious manifestations of a condition,
would not be appropriate for use in patients with milder manifestations
The proposed pathway was recommended by the President's Council of
Advisors on Science and Technology (PCAST) in their September 2012
``Report to the President on Propelling Innovation in Drug Discovery,
Development, and Evaluation,'' as a way to improve drug evaluation. The
PCAST recommendations support the goal of increasing the output of
innovative, new medicines for patients with important unmet medical
needs, while increasing drug efficacy and safety, through
industry, academia, and government working together to decrease
clinical failure, clinical trial costs, time to market, and regulatory
uncertainty.\2\
\2\ For more information on the PCAST Report to the President on
Propelling Innovation in Drug Discovery, Development, and
Evaluation, see http://www.whitehouse.gov/sites/default/files/microsites/ostp/pcast-fda-final.pdf.
FDA is holding this public hearing to seek input from interested
members of the public including patients and consumers, practitioners
and other members of the medical community, regulated industry,
insurers, and managed care organizations on a potential new pathway to
approve drugs shown to be safe and effective in a subpopulation of
patients with serious or life-threatening conditions in which an unmet
medical need exists. FDA is interested in obtaining information and
public comment on the following issues:
1. Considering existing processes to expedite drug development and
review of important new therapies (i.e., accelerated approval, fast-
track designation), would this new pathway increase the therapeutic
options for serious or life-threatening conditions for which an unmet
medical need exists? If not, what might be some alternative approaches?
2. Can you identify specific serious or life-threatening conditions
for which an unmet medical need exists and for which this approval
pathway may benefit subpopulations of patients?
3. What approaches could be undertaken (by FDA or by people or
organizations other than FDA) to monitor use of drugs approved under
this pathway to determine whether they are being used inconsistent with
the terms of approval? What approaches could be undertaken to prevent,
manage, or monitor use in a broader population where safety and
efficacy has not been demonstrated? For example, if this pathway were
adopted to approve new antibacterial drugs when limited use was needed
(e.g., to prevent the emergence of further antimicrobial resistance),
what other measures (by FDA or by people or organizations other than
FDA) might ensure that these products are used appropriately only in
the indicated subpopulations?
4. Would this pathway help to address some of the current
challenges in antibacterial drug development, particularly for serious
or life-threatening infections for which there is an unmet medical
5. This potential pathway could be used to approve drugs for a
limited subpopulation based upon smaller clinical trials, when benefit-
risk is appropriate for the limited population but safety and efficacy
have not been demonstrated for use in a broader population of patients
or patients with less severe manifestations of the condition. For the
serious or life-threatening conditions you identified in question 2,
what benefit-risk considerations need to be taken into account before
and after marketing and how should they be addressed?
6. Would the use of a formal designation and logo to reflect
approval under this pathway, with clear labeling of clinical
information that only supports use in the indicated subpopulation, but
without other constraints from FDA be effective in limiting use to the
indicated subpopulation? Why or why not?
The public hearing is free and seating will be on a first-come,
first-served basis. Attendees, including those not presenting, need to
register for the public hearing.
If you wish to attend or make an oral presentation during the
hearing, you must register by submitting either an electronic or
written request received on or before January 22, 2013. (See FOR
FURTHER INFORMATION CONTACT.) You must provide your name, title,
numbers, email address, and type of organization you represent (e.g.,
industry, consumer organization). If requesting to present, you also
should submit a brief summary of the presentation, including the
discussion question(s) that will be addressed and the approximate time
requested for your presentation. FDA has included discussion questions
in section III of this document. You should identify the question(s)
and the number of each question you wish to address in your
presentation. We encourage individuals and organizations with common
interests to consolidate or coordinate their presentations to allow
adequate time for each request for presentation. FDA will do its best
to accommodate requests to speak and will determine the amount of time
allotted for each oral presentation, and the approximate time that each
oral presentation is scheduled to begin. Persons registered to make an
oral presentation should submit to FDA an electronic copy of their
presentation and an abstract to ExpeditedPathwayPublicMtg@fda.hhs.gov
on or before January 30, 2013.
contact Jonas Santiago (see FOR FURTHER INFORMATION CONTACT) at least 7
The Commissioner of Food and Drugs is announcing that the public
hearing will be held in accordance with part 15 (21 CFR part 15). The
hearing will be conducted by a presiding officer, who will be
accompanied by FDA senior management.
Under Sec.  15.30(f), the hearing is informal and the rules of
evidence do not apply. No participant may interrupt the presentation of
another participant. Only the presiding officer and panel members may
question any person during or at the conclusion of each presentation.
Public hearings under part 15 are subject to FDA's policy and
procedures for electronic media coverage of FDA's public administrative
proceedings (part 10 (21 CFR part 10, subpart C)). Under Sec.  10.205,
representatives of the electronic media may be permitted, subject to
certain limitations, to videotape, film, or otherwise record FDA's
public administrative proceedings, including presentations by
participants. The hearing will be transcribed as stipulated in Sec.
15.30(b) (see section VII of this document).
To the extent that the conditions for the hearing, as described in
this notice, conflict with any provisions set out in part 15, this
notice acts as a waiver of those provisions as specified in Sec.
15.30(h).
found in brackets in the heading of this document. In addition, when
responding to specific questions as discussed in section III of this
document, please identify the question you are addressing. Received
a.m. and 4 p.m., Monday through Friday, and will be posted to the
docket at http://www.regulations.gov.
Transcripts of the public hearing will be available for review at
the Division of Dockets Management (see ADDRESSES) and on the Internet
at http://www.regulations.gov approximately 30 days after the public
hearing. A transcript will also be made available in either hard copy
or on CD-ROM, upon submission of a Freedom of Information request.
Written requests are to be sent to the Division of Freedom of
Information (ELEM-1029), Food and Drug Administration, 12420 Parklawn
Dr., Element Bldg., Rockville, MD 20857.