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 for FDA-approved human medical products.
In Section 907 of the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA), 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 the 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 website.
To comply with that request, in August 2013, FDA published a report entitled, “Collection, Analysis, and Availability of Demographic Subgroup Data for FDA-Approved Medical Products.” Section 907 of FDASIA also requires the Agency to develop an Action Plan based on the findings of the 907 Report. The Agency will utilize previous comments to the docket, outreach to stakeholders, and input from this public meeting to develop a plan that is pragmatic and public-health focused.
The public hearing will be held on April 1, 2014, from 9:00 a.m. to 3:00 p.m. To attend the meeting in person or via webcast, please register using this site.
There will be an opportunity for attendees to make oral presentations during the meeting. If you are interested, please register as a "Presenter" and send an email to FDASIA907@fda.hhs.gov including the following: 1) your name 2) title 3) business affiliation (if applicable) 4) type of organization you represent (e.g., industry, consumer organization, etc.) and 5) a brief summary of your presentation. Requests to make oral presentations during the meeting will be accepted until close of business on March 21, 2014. Registrants to make oral presentations will receive confirmation once they have been accepted.
There is also an open public docket for this hearing. Electronic comments can be submitted 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 for FDA-Approved Human Medical Products, Public Hearing.’’ Electronic or written comments to the docket will be accepted after the hearing until May 16, 2014.
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?
For more information, refer to the FDA meeting website at http://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/default.htm.