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Part 15 Public Hearing on Section 907 of FDASIA

Remarks by Margaret Hamburg, MD
Commissioner of Food and Drugs
Part 15 Public Hearing on Section 907 of FDASIA
The Great Room, White Oak Bldg. 31
Silver Spring, MD
April 1, 2014


Thank you, Lesley, for that kind introduction.  I also want to recognize and thank the FDA’s Offices of Minority Health and Women’s Health, for their leadership in bringing together academia, industry, health care providers, patients and advocacy groups on this important issue. And of course, I want to thank all of you for being here, either in person or via Internet and the live streaming of this meeting. 

Let me begin by stating that we share a common belief – that encouraging diversity is vital for biomedical research and for ensuring the health needs of patients across the demographic spectrum. In fact, it is more than a belief, it is a commitment.

FDA is striving to develop a well-informed action plan for improving data collection, analysis and communication about demographic subgroups in clinical trials of FDA-regulated human medical products. And certainly, getting your input is essential if we are to arrive at a robust plan that can have a real and actionable impact.

We recognize that when more diverse populations are included in clinical trials, we increase the potential knowledge about possible differences in sex, age, or racial and ethnic groups that might affect the valve or risks of medical interventions.  This helps us to assure that clinical trial participants are representative of those individuals who ultimately will use these important medicines and medical devices, and that the products will be safe and effective for people in these demographic subgroups.

In fact, FDA has been working hard on many of these issues for over 30 years. Our recognition of the importance of studying specific subpopulations was reflected in the 1980s, when we first issued guidance about the importance of studying the effects of drugs in elderly patients.

Our focus on possible variations in response in different populations sharpened after the GAO released a report in 1992 on the need for analyses of drug effects in women in clinical trials. The FDA responded quickly by issuing a guidance to encourage the study and evaluation of gender differences in drugs. In 1994, FDA established its Office of Women’s Health, with the mission of protecting and advancing the health of women – including as advocates for the inclusion of women in clinical trials and analysis of sex/gender effects. 

 As a result of the work of the collaboration between that office, now under the leadership of director Marsha Henderson, and FDA medical product centers, as well as the work of other stakeholders, women today are far more adequately represented in the majority of clinical trials for safety and effectiveness decisions about FDA-approved products.  Even more importantly, analysis for sex differences in clinical drug trials, required by regulation since 1998, is now almost always done in new drug applications.

And we have broadened these efforts as well. For example, clinical data in New Drug Applications (NDAs) must be analyzed by age, gender, and race, and annual reports for Investigational New Drug Applications (INDs) must display the inclusion of patients by these categories as well.

But underrepresentation of racial and ethnic minorities continues to be a concern. That’s why I was pleased that in 2010, thanks to the Affordable Care Act, we were able to establish FDA’s Office of Minority Health. With Dr. Jonca Bull as its director, we are building and growing this new office that will advance FDA’s regulatory mission while advising the Agency on actions that can help reduce racial and ethnic health disparities and achieve the highest standard of health for all. 

In 2011, we issued a guidance for medical devices 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.  We plan to finalize that guidance in the near future.

FDA also holds periodic workshops and public meetings on demographic subgroup inclusion and analyses. For example, in 2011, FDA cosponsored a conference with the Society for Women’s Health Research, Dialogues on Diversifying Clinical Trials: Successful Strategies for Engaging Women and Minorities. That conference focused on novel methods for improving recruitment and retention of women and minorities, community-based approaches to clinical trial design, and federal perspectives on guidelines and regulations to improve diversity in government- and industry-funded research.

And just this past June, FDA held the Health of Women Public Workshop to discuss how to improve the availability, consistency and communication of sex-specific information to patients and providers for the safe and effective use of medical devices in women, and to brainstorm about effective strategies to address clinical research needs.

 It is also important to recognize that our work in this area is constantly being informed by new developments in science. Indeed, a critical part of FDA’s mission is to promote the science and innovation it takes to produce tomorrow’s breakthrough products…and to ensure that our nation is equipped to address the medical care and public health opportunities and challenge of the 21st Century.

No society can be truly healthy if it suffers from disparities in health care and health outcomes.  To close those gaps, we must encourage adequate and diverse participation in biomedical research and appropriate data collection and analysis about women, minorities, elderly and other subpopulations in clinical trials.

To this end, new areas such as pharmacogenomics are now being incorporated into product development and regulatory review to further address subgroup characteristics and population needs. Scientific tools continue to be developed to overcome the challenges of the “one-size-fits all” model of patient treatment and to steer us into the ultimate goal of tailoring treatments to individuals, or subgroups of patients, through personalized medicine — including those in underserved and underrepresented populations.  

I was pleased that in 2012 Congress asked FDA to take a closer look at the inclusion and analysis of demographic subgroups in applications for drugs, biologics and devices – including by sex, race/ethnicity, and age, and file a report on our findings.

To comply with Sec. 907 of the Food and Drug Administration Safety and Innovation Act, we assembled a cross-agency team to scrutinize the product submissions that were approved by our medical product centers in 2011, and we released our report last August.

Our analysis 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 are describing the demographic profiles of their clinical trial participants, and the majority of applications submitted to FDA include demographic subset analyses. 

The report also revealed that FDA shares this information with the public in a variety of ways including through product labeling, the FDA website, and summaries of safety and effectiveness for all approved medical products.

But we think we can do more.  And I know you think so too, as indicated by the thoughtful comments many of you submitted to the docket about the Sec. 907 report. Our goal is to take those comments, the data we collected for the Sec. 907 report, and your input at this hearing, to help inform the development of an action plan that identifies what more we can do regarding demographic subgroups in clinical trials. We hope to publish the plan in August, and we intend for it to be relevant and practical to implement. And most importantly, we intend for this work will have a meaningful impact on health.

Going forward, we will continue to review and improve our internal processes and procedures to assure that FDA-regulated products are being tested in ways that inform real-world use, representing the diverse populations who will be using the products once on the market. And we will continue to explore and advance ways to improve how FDA communicates about subgroup analysis in a clear and informative manner. 

But we will also expand our work with patients and patient advocates, to learn how products are working for them and for you, and how to engage underrepresented communities in clinical trials to improve therapeutic outcomes.

We will continue to work with health professionals to learn about your experiences in treating women, children, the aged, minorities, and other subgroups with FDA-approved medical products, with industry sponsors to improve data collection and analysis for demographic subgroups participating in their trials, and with members of the scientific community to explore ways to advance regulatory science and areas of personalized medicine. 

And we will work with all of our partners to exchange information and ideas on disease burden among underrepresented groups and how we can help to address it through better research, treatment, and care.

The FDA is eager to work hard and to help lead the way—but, as I noted, we can’t do it alone.  We need each of you.  We need your ideas and input.  And we need your commitment.

Thank you for engagement on this important issue.