Comments on FDA Safety and Innovation Act Action Plan

Division of Dockets Management

Food and Drug Administration

5630 Fishers Lane, Room 1061 (HFA-305)

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Comments of Members of the Patient, Consumer, and Public Health Coalition on

“Guidance: Food and Drug Administration Safety and Innovation Act Action Plan”

Docket No. FDA-2013-N-0745-0053

As Members of the Patient, Consumer, and Public Health Coalition we appreciate the opportunity to provide comments on the Food and Drug Administration Safety and Innovation Act (FDASIA) Action Plan. We strongly support separate analyses of safety and effectiveness for demographic subgroups in clinical trials for drugs and devices. All patients deserve to be assured that the FDA has required that drugs and devices be proven safe and effective on people like them.  The data must be methodologically sound and publicly available.

Several members of our Coalition previously provided written and oral comments on the FDA’s 907 report.  We focused our comments on what the FDA can do to ensure greater diversity in the clinical trials submitted to the agency, and to make sure that subgroup analyses are conducted to determine safety and effectiveness for those different demographic subgroups. We are surprised and disappointed that the FDA’s Action Plan does not reflect those concerns.

The Action Plan is often vague and does not adequately focus on the need for meaningful subgroup analyses.  It also passes the buck on these issues to stakeholders and other federal agencies rather than recognizing the FDA’s failure to use its regulatory power to finally make sure that drugs and devices are adequately tested on men and women, major racial/ethnic groups, and older patients as well as younger ones.

Action Plan’s Priority #1: Quality–Improve the completeness and quality of demographic subgroup data collection, reporting and analysis.

Guidance Documents Needed for Medical Products

We agree with the Action Plan’s inclusion of 3 guidance documents.  However, the FDA has been encouraging greater diversity and subgroup analyses at CDER and CBER for years, with limited success, and there is no evidence that more encouragement will improve the outcome.  If the FDA believes it cannot require such analyses, then they should specify that approval for a general population (e.g. not just White males under 65, or White adults 18-65) requires that the company evaluate the major demographic subgroups to determine if the drug or biologic is safe and effective for those various groups.

A guidance for diversity in clinical trials for the Center for Devices and Radiological Health is long overdue.  However, this section of the Action Plan uses wording that is too vague and weak.

Currently, clinical studies for most medical devices are so small that it would be impossible to conduct meaningful subgroup analyses.  Since the only clinical trials for devices are for the highest risk, often life-saving devices, it is unacceptable that so many devices are studied on so few Blacks. The statistics are only slightly better for women (not counting devices specifically or primarily for women) or people over 65.


  • The FDA should require subgroup analyses that analyze all safety and effectiveness outcomes for each major demographic subgroup.  
  • The FDA should make it clear that the agency will not approve medical products for all populations if the product was not tested for safety and effectiveness on all major demographic subgroups with meaningful subgroup analysis.


We agree that the FDA should train new clinical trial reviewers, and “offer additional education and training courses for experienced reviewers to better clarify FDA’s expectations for data collection and analysis related to demographic subgroups.”


  • The training and education of FDA reviewers and Advisory Committee members should be consistent and substantially and immediately improved across CDER, CBER, and CDRH.


We support the Office of Women’s Health (OWH) plans to target OWH funding to projects that answer specific regulatory research questions and emerging priorities from the product review centers. We support the Office of Minority Health (OMH) plans to develop research projects to better understand medical product clinical outcomes in racial and ethnic demographic subgroups.  We also support the OMH plans to collaborate with National Institute of Health’s (NIH) National Human Genome Research Institute in research into the role of genetics and genomics in health disparities.


The Action Plan should spell out that OWH and OMH will conduct research to better understand subgroup differences in safety or effectiveness based on subgroup analyses of major demographic subgroups that the companies conduct as part of the approval process.  For example, if subgroup analyses indicate a difference in safety for women compared to men on sleeping pills, as was recently the case, the OWH would conduct research to better understand those disparities.   The same would be true for the OMH following up on subgroup analyses showing differences between Blacks and Whites taking hypertension medications.

Action Plan’s Priority #2: Participation–Identify barriers to subgroup enrollment in clinical trials and employ strategies to encourage greater participation.

We support the FDA collaborating with NIH, other Department of Health and Human Services (HHS) agencies, industry and other stakeholders to broaden diverse participation in clinical research.  We also support FDA’s OMH collaborating with agencies of the HHS, NIH, Institute of Medicine and the Human Service’s Office of Minority Health to convene a meeting in 2015 to better understand the barriers to participation in clinical trials by communities of color; we add that this should include older patients.  We also add that this meeting should focus on the incentives and disincentives that currently exist, particularly on those that the companies could improve.


  • The FDA should facilitate the sharing of strategies from companies that are achieving diversity in their trials with companies that are not accomplishing this goal.
  • The FDA should work with individual drug and device companies throughout the approval process to ensure the inclusion of major demographic subgroups (gender, race/ethnicity, and age) in clinical trials.  

Action Plan’s Priority #3: Transparency–Make demographic subgroup data more available and transparent.

We are concerned about the action items in section 3.1. We agree that it is vital to focus on transparency.  However, this states that the FDA will post “demographic composition and analysis by subgroup in pivotal clinical studies for FDA-approved medical products.” It is unclear what this means.  We want to make sure that each major subgroup will be analyzed to determine safety and effectiveness for that group.


  • The FDA should clarify that subgroup analyses should be conducted to determine safety and effectiveness for major demographic subgroups, such as men and women; Whites, Blacks, and Hispanics; adults under 65 and adults over 65.

Section 3.2

Section 3.2 is too vague. It states that the FDA intends “to work with industry, advocacy groups, risk communicators (including FDA’s Risk Communication Advisory Committee) and other stakeholders to explore potential methods for communicating meaningful information on demographic analyses to the public.” It does not identify specific work that FDA will do and it does not define “meaningful information.”


  • Inclusion of demographic subgroup analyses should be required, standardized, and easy-to-understand on the label, so that patients and doctors can quickly find this information.  If it does not exist, this should be clearly stated and the reasons given.


The FDA’s Action Plan is too vague and it is unclear whether the proposed changes would have a meaningful impact.  The Action Plan does not state that the FDA will require diversity in clinical trials or subgroup analyses.  The FDA should clearly state that they will not approve drugs or devices for the general population unless the safety and effectiveness of the product has been separately analyzed for major demographic subgroups.

American Medical Women’s Association

Annie Appleseed Project

Center for Medical Consumers

Connecticut Center for Patient Safety


Mothers Against Medical Error

Our Bodies Ourselves

The TMJ Association

Center for Science and Democracy, Union of Concerned Scientists



The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at (202)223-4000 or

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