[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]



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: FDASIA907@fda.hhs.gov.


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 

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 
    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 FDASIA907@fda.hhs.gov 
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 
    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]