[Federal Register Volume 79, Number 42 (Tuesday, March 4, 2014)]
[Proposed Rules]
[Pages 12134-12135]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-04625]
=======================================================================
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
21 CFR Part 15
[Docket No. FDA-2013-N-0745]
Action Plan for the Collection, Analysis, and Availability of
Demographic Subgroup Data in Applications for Approval of Food and Drug
Administration-Regulated Medical Products; Notice of Public Hearing;
Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notification of public hearing; request for comments.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA) is announcing a public
hearing to obtain input on the issues and challenges associated with
the collection, analysis, and availability of demographic subgroup data
in applications for approval of FDA-regulated human medical products.
DATES: The public hearing will be held on April 1, 2014, from 9 a.m. to
3 p.m. Submit electronic or written requests to make oral presentations
at the hearing by March 21, 2014. Electronic or written comments will
be accepted after the hearing until May 16, 2014.
ADDRESSES: The public hearing will be held at FDA's White Oak Campus,
10903 New Hampshire Ave., Bldg. 31, Conference Center, the Great Room
(Rm. 1503A), Silver Spring, MD 20993. Entrance for the public hearing
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.
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. All comments should be identified with the corresponding docket
number for the public meeting as follows: ``Docket No. FDA-2013-N-0745,
Action Plan for the Collection, Analysis, and Availability of
Demographic Subgroup Data in Applications for Approval of FDA-Regulated
Human Medical Products, Public Hearing.''
FOR FURTHER INFORMATION CONTACT: Brenda Evelyn, Office of the
Commissioner, Office of Minority Health, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 32, Rm. 2303, Silver Spring, MD 20993
240-402-4201, email: [email protected].
SUPPLEMENTARY INFORMATION:
I. Background
In section 907 of the Food and Drug Administration Safety and
Innovation Act (FDASIA) (Pub. L. 112-144), the U.S. Congress directed
FDA to produce a report that addressed the extent to which clinical
trial participation and the inclusion of safety and effectiveness data
by demographic subgroups, including sex, age, race, and ethnicity, is
included in applications submitted to FDA. Specifically, Congress asked
FDA to consider four key topic areas: (1) A description of existing
tools to ensure submission of demographic information along with how
information about differences in safety and effectiveness of medical
products according to demographic subgroup is made available to health
care providers, researchers, and patients; (2) an analysis of the
extent to which demographic data subset analyses are presented in
applications; (3) an analysis of demographic subgroup representation in
clinical trials submitted to FDA in support of product applications;
and (4) an analysis of the extent to which a summary of product safety
and effectiveness data by demographic subgroup is made available to the
public in product labeling or on FDA's Web site.
To comply with that request, in August 2013, FDA published a report
``Collection, Analysis, and Availability of Demographic Subgroup Data
for FDA-Approved Medical Products.'' \1\ The report describes the
Agency's evaluation of 72 applications approved during 2011 for new
molecular entity drug products, original biologics, and class III
devices (premarket approval).
---------------------------------------------------------------------------
\1\ FDA, ``Collection, Analysis, and Availability of Demographic
Subgroup Data for FDA-Approved Medical Products,'' August 2012,
available at http://www.fda.gov/downloads/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/fdasia/ucm365544.pdf.
---------------------------------------------------------------------------
Regarding collection of data, although there was variation by
product area, the evaluation found FDA's statutory and regulatory
requirements, guidances, policies, and procedures generally informed
sponsors about including tabulations of the demographic data on
clinical trial participants and demographic subset analyses in their
medical product applications.
Similarly, tools (e.g., application review templates and FDA
standard operating policies and procedures) guide regulatory review
staff in the assessment of marketing applications to ensure that
demographic data and subset analyses are included in the information
FDA uses in its review and approval processes.
However, the extent to which demographic subset data were analyzed
varied across medical product types (drugs, biologics, and devices).
Applications for drugs and biologics uniformly addressed subset
analyses by sex, race, and age--that is, the applications mentioned
demographic subsets in some way. The report noted that FDA's new drug
application regulations (21 CFR part 314; specifically Sec. 314.50)
call for demographic analysis in all applications in the integrated
summaries of safety and effectiveness. Guidance and standard operating
procedures for drugs and biologics also emphasize the importance of
such analyses. There are no regulations requiring demographic analysis
for device applications. Nonetheless, the majority of the device
applications contained a subset analysis for age and sex, with a lower
percentage of applications containing a subset analysis for race and
ethnicity. Inclusion did not necessarily mean that the data on patient
subgroups was sufficient for meaningful analysis or to detect relevant
subgroup effects.
The report stated that all biologics, drugs, and the majority of
the medical
[[Page 12135]]
device applications reviewed provided the composition of clinical study
participants by age, race, and sex. Participants' sex was the most
consistently reported in the medical product applications. For approved
drugs and biologics, the extent to which patients were represented in
clinical trials by age and sex tended to reflect the disease indication
studied. For devices, patient participation by age and sex varied by
product area. Whites represented a high percentage of clinical trial
study participants for biologic, drug, and medical device applications,
and in many cases, other racial subgroups were underrepresented.
FDA's internal policies, procedures, and regulations encourage
demographic subgroup information be included in marketing applications.
Moreover, following medical product approval, FDA communicates
available information to the public both on the demographic profile of
the study participants and on the demographic data subset analyses
using a variety of mechanisms: Initially with product labeling and
publicly posted clinical reviews, and later, once a product is on the
market, with consumer updates, safety alerts, labeling changes, and
other mechanisms, as needed.
As is required by section 907 of FDASIA, in response to the
findings in the report, FDA is developing an action plan to address
improving the completeness and quality of analyses of data on
demographic subgroups in labeling, the inclusion of such data in
labeling, and improving the public availability of information on
demographic subgroups to patients, health care providers, and
researchers.
II. Purpose and Scope of the Public Hearing
As part of FDA's process in the development of the required action
plan, the Agency has decided to hold a public hearing to obtain
information and viewpoints from key stakeholders and expert members of
the public on the following questions:
A. Demographic Subgroup Representation in Clinical Trials
1. What approaches might be used to encourage enrollment of
representative proportions of subgroup participants in clinical trials
consistent with disease prevalence in the underlying population being
studied?
2. What sources could be used to define disease prevalence among
subgroups? Are there priority areas for study in terms of disease/
condition, or in terms of demographic subgroup?
3. What are best practices and considerations for developing
inclusion and exclusion criteria for clinical trials generally and for
the early stages of research?
4. What approaches should FDA use to standardize the capture of
race and ethnicity information, including for studies conducted outside
the United States?
B. Analysis of Demographic Subgroup Data
1. What are the statistical challenges in analyzing clinical trial
data to evaluate subgroup differences?
2. Given that it is not feasible to power most studies to detect
subpopulation differences, what approaches should be used to analyze
subgroups to explore clinically relevant information?
3. How might additional clinically relevant information about
subgroups be obtained in the postmarket setting?
C. Communication of Demographic Subgroup Information to the Public
1. What information regarding demographic subgroups is helpful to
health care professionals to make informed decisions about the use of
medical products? To consumers/patients? To researchers?
2. What is the best way for FDA to communicate and make accessible
such information to health care professionals? To consumers/patients?
To researchers?
III. Attendance and Registration
If you wish to attend the hearing or make an oral presentation
during the hearing, you must register by submitting either an
electronic request (see the Web address listed at the end of this
paragraph) or written request (see FOR FURTHER INFORMATION CONTACT) by
close of business on March 21, 2014. You must provide your name, title,
business affiliation (if applicable), address, email address, and type
of organization you represent (e.g., industry, consumer organization),
and a brief summary of your presentation, if applicable (including the
discussion topic(s) that will be addressed), to http://www.eventbrite.com/e/fda-public-hearing-fdasia-section-907-tickets-10678512719 by March 21, 2014.
FDA will notify registered presenters of their scheduled
presentation times. Persons registered to make an oral presentation
should check in before the hearing and are encouraged to arrive early
to ensure the designated order of presentation times. We will try to
accommodate all persons who wish to present; however, the duration of
each speaker's testimony may be limited by time constraints. Questions
about the meeting may also be also submitted to [email protected]
prior to the April 1, 2014, meeting date.
The hearing is free and seating will be on a first-come, first-
served basis. Early registration is recommended because seating is
limited. FDA may limit the numbers of participants from individual
organizations as well as total number of attendees based on space
limitations.
Registrants will receive confirmation once they have been accepted
to attend the hearing. For those who cannot attend in person,
information regarding viewing a live Web cast of the public hearing
will be located on FDA's Web site.
If you need special accommodations due to a disability, contact
Brenda Evelyn at 240-402-4021.
IV. Comments
Interested persons may submit either electronic comments regarding
this document to http://www.regulations.gov or written comments 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. To ensure
consideration, submit comments by (see DATES). 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.
V. Transcripts
Please be advised that as soon as a transcript is available, it
will be accessible at http://www.regulations.gov. It may be viewed at
the Division of Dockets Management (see ADDRESSES). A transcript will
also be available in either hardcopy or on CD-ROM, after 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: February 26, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-04625 Filed 3-3-14; 8:45 am]
BILLING CODE 4160-01-P