By Barbara D. Buch, M.D.
At FDA, one of our foremost responsibilities is to evaluate and if medical products meets the appropriate standard, to approve or clear drugs, biological products and medical devices. We know that these products are safer and more effective for everyone when they are tested in clinical trials that include diverse populations.
The design and analysis of clinical trials has evolved significantly over the last three decades. FDA now has a variety of statutory, regulatory, and policy-related tools at its disposal that provide a framework for guiding medical product sponsors and FDA review teams in the collection, subset analyses, and communication of these data.
Collecting and analyzing information in clinical trials about sex, age, and race/ethnicity, makes it possible for individuals or groups considering a treatment option to look at the information and ask, “Was there anyone like me in the clinical studies? And if so, how did they do?”
Section 907 of the Food and Drug Administration Safety and Innovation Act (FDASIA) directed FDA to look at these questions on a broader scale: to investigate how well demographic subgroups (sex, age, race and ethnicity) are included in clinical trials; whether they are analyzed for safety and effectiveness by these subgroups; and to improve on making the resulting information available to the public. After systematically reviewing 72 medical product applications, FDA published a report, in August 2013, which concluded that FDA has been doing a good job, but we acknowledged we could do better. In August of last year we came up with a plan to improve our performance. The Action Plan includes 27 action items focused on three priorities:
- Quality: to improve the completeness and quality of demographic subgroup data collection, reporting and analysis;
- Participation: to identify barriers to subgroup enrollment in clinical trials and employ strategies to encourage greater participation;
- Transparency: to improve the public availability of demographic subgroup data.
Since the release of the report, FDA has formed an agency-wide steering committee, which I chair. FDA has made significant progress.
So far, FDA:
- Has launched the Drug Snapshots web page that extracts Demographic Subgroup Data for FDA approved products. The information in a drug trials snapshot is taken from the data submitted in a new drug application or a biologic license application. It includes information on study participants, how the study was designed, the results of the efficacy and safety studies and the differences in side effects and in benefits among sex, race and age groups.
- Is leveraging IT platforms already in place to support electronic submissions that enhance FDA’s systems for collecting, analyzing, and communicating standardized data collection categories by age, racial and ethnic groups in submitted applications. This will facilitate harmonized data collection and analysis of subgroup outcome trends, and diverse clinical information in diverse populations over the total product life cycle in a standard way. These systems are also developed to facilitate industry’s data input and allow for better tracking of these data.
- Has added education/training for reviewers about demographic inclusion, analysis, and communication of clinical data. We have also developed plans to incorporate details of demographic subgroup analyses in review templates.
- Has proposed changes (to the MedWatch adverse event reporting forms to enhance the clarity and utility of the demographic information FDA is able to collect in the post-market setting. These include collecting data about race/ethnicity and age.
- Has launched a study with health care professionals to improve usability and understanding of medical device labeling, including instructions for use.
- Is working with industry to try to establish best practices and ways to help ensure appropriate use of enrollment criteria in clinical trial protocols.
- Has established a joint working group with the National Institutes of Health (NIH) to create a framework for collaborating and exchanging information on inclusion policies, practices and challenges.
- Is participating with NIH in a session at the Society for Clinical Trials annual meeting in May 2015, on approaches to clinical trial study design and analyses that maximize sex-specific data reporting.
We are proud of our progress to date – but we can always do more. That is why in early 2016, FDA will host a public meeting to gain insight and feedback. Watch this space for details, as well as new developments in our quest to integrate more fully the demographics of patient populations into our review of medical products.
Barbara D. Buch, M.D., is the Chair of the 907 Steering committee and the Associate Director for Medicine in FDA’s Center for Biologics Evaluation and Research