Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need; Public Hearing; Request for Comments, 3005-3008 [2013-00607]
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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 https://
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
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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 https://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 https://www.fda.gov/
MedicalDevices/
DeviceRegulationandGuidance/
Standards.
This Federal Register document on
modifications in FDA’s recognition of
consensus standards is available at
https://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]
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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:
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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 https://www.
fda.gov/AboutFDA/WorkingatFDA/
BuildingsandFacilities/WhiteOak
CampusInformation/ucm241740.htm.
Submit electronic comments to
https://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
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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 https://www.
fda.gov/downloads/Drugs/DevelopmentApproval
Process/HowDrugsareDevelopedandApproved/
DrugandBiologicApprovalReports/UCM278506.pdf.
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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
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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
https://www.whitehouse.gov/sites/default/files/
microsites/ostp/pcast-fda-final.pdf.
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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
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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 https://
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 https://
www.regulations.gov.
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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 https://
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
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]]>Agencies
[Federal Register Volume 78, Number 10 (Tuesday, January 15, 2013)]
[Notices]
[Pages 3005-3008]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-00607]
-----------------------------------------------------------------------
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.
ACTION: Notice of public hearing; request for comments.
-----------------------------------------------------------------------
SUMMARY: 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
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
potential advantages and disadvantages.
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,
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 https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
Submit electronic comments to https://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:
ExpeditedPathwayPublicMtg@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 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.
I. Background
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.
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\1\ See a list of Center for Drug Evaluation and Research Drug
and Biologic Accelerated Approvals as of September 30, 2011,
available at https://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/UCM278506.pdf.
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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 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
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
[[Page 3007]]
industry, academia, and government working together to decrease
clinical failure, clinical trial costs, time to market, and regulatory
uncertainty.\2\
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\2\ For more information on the PCAST Report to the President on
Propelling Innovation in Drug Discovery, Development, and
Evaluation, see https://www.whitehouse.gov/sites/default/files/microsites/ostp/pcast-fda-final.pdf.
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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 life-threatening infections for which there is an unmet medical
need?
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?
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 ExpeditedPathwayPublicMtg@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 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).
VI. Comments
Interested persons may submit either electronic comments to https://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 https://www.regulations.gov.
[[Page 3008]]
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 https://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
