Message from Margaret A. Hamburg, M.D., Commissioner of Food and Drugs

One of the core tenets of rigorous biomedical research, as well as a guiding principle of the FDA’s goal to meet the health needs of patients across the demographic spectrum, is the importance of encouraging diversity in clinical trials.

When a more diverse population participates in clinical trials, we increase the potential to know more about the extent to which different subgroups—males and females, young and old, people of various racial and ethnic backgrounds, and patients with differing comorbid diseases and conditions—might respond to a medical product. And when subgroup data are analyzed, we have available more information about the product that can be communicated to the public. The result is greater assurance in the safety and effectiveness of the medical products used by a diverse population.

FDA has been addressing these issues since the 1980s, when we first released guidance about the importance of studying the effects of drugs in elderly patients. In the 1990s, we issued a “Guideline for the Study and Evaluation of Gender Differences in the Clinical Evaluation of Drugs” and established the Office of Women’s Health. As a result, women today are far more adequately represented in the majority of clinical trials that are the basis for safety and effectiveness decisions about FDA-approved products. Moreover, analysis for sex differences in clinical drug trials, required by regulation since 1998, is now routinely done in new drug applications. More recently, we’ve been looking at ways to encourage greater inclusion of women in medical device trials. In 2011, we issued draft guidance outlining our expectations for sex-specific data analysis and reporting of medical device clinical study information, as well as recommending strategies for enrollment of women in clinical studies, and we have now issued a final version of that guidance.

Advances in science are also playing an increasingly important role in deepening our understanding of how patients within various subgroups respond to medical products. For example, information from areas such as pharmacogenomics is now being incorporated into product development and regulatory review to further address subgroup characteristics and population needs, helping to overcome the challenges of the “one-size-fits all” model of patient treatment. Ultimately, this is steering us towards the goal of tailoring treatments to individuals or subgroups of patients through personalized medicine—including patients in underserved and underrepresented populations.

But for all the progress we have made, there are still areas of concern, particularly involving underrepresentation of racial and ethnic minorities. That’s why it was important that in 2010, as part of the Affordable Care Act, we were able to establish FDA’s Office of Minority Health to advise FDA on ways to reduce health disparities among racial and ethnic subgroups.

In Section 907 of the 2012 Food and Drug Administration Safety and Innovation Act, Congress directed us to take a closer look at the inclusion and analysis of demographic subgroups in applications for drugs, biologics and devices—including by sex, race and ethnicity, and age—and report on our findings. A cross-agency task force analyzed 72 applications for drugs, biologics and devices approved in 2011. Our report, published in August

2013, found that the agency’s statutes, regulations and policies generally give product sponsors a solid framework for providing data in their applications on the inclusion and analysis of demographic subgroups; in general, sponsors describe the demographic profiles of their clinical trial participants; and the majority of applications submitted to FDA include demographic subset analyses that the FDA shares with the public in a variety of ways.

But in that report, we also concluded that we could do better, and Section 907 gave us a vehicle for considering improvements. It directed that one year after the issuance of the Section 907 report we publish and provide to Congress an action plan outlining “recommendations for improving the completeness and quality of analyses of data on demographic subgroups in summaries of product safety and effectiveness data and in labeling; on the inclusion of such data, or the lack of availability of such data, in labeling; and on improving the public availability of such data to patients, health care providers, and researchers.”

Today, after extensive consultation with stakeholders, FDA is delivering its Action Plan to Enhance the Collection and Availability of Subgroup Data. It includes 27 responsive and pragmatic actions, which are divided into three overarching priorities: improving the completeness and quality of demographic subgroup data collection, reporting and analysis (quality); identifying barriers to subgroup enrollment in clinical trials and employing strategies to encourage greater participation (participation); and making demographic subgroup data more available and transparent (transparency).

Some of the action items can be accomplished quickly while others will take longer to achieve and require additional resources. But once the plan is put fully in effect, I believe it will provide additional information to help health care providers and patients make decisions about the medical products they use.

Addressing the three priorities of quality, participation and transparency will require a multifaceted approach and the active participation of both FDA and our many stakeholders. We look forward to working with them as we implement this action plan.


Margaret A. Hamburg, M.D. Commissioner of Food and Drugs

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