Age, Race, and Ethnicity in Medical Device Studies: New FDA Draft Guidance and Decision Frameworks
Under-representation of participants based on age, race, and ethnic minority groups in clinical research is a complicated issue seeped in physical, communicative, educational, financial, and socio-cultural barriers.1
Further, it is adverse to the Belmont Report and the tenet of justice, as it reflects an unequal distribution of the benefits and burdens of research. The Food and Drug Administration (FDA) has issued new draft guidance, titled Evaluation and Reporting of Age, Race, and Ethnicity Data in Medical Device Clinical Studies, which communicates FDA’s expectations to improve inclusion, analysis, and reporting of age, race, and ethnicity in medical device evaluations in order to increase device safety and effectiveness.2
Evidence of Substantial Need
A 2013 FDA report evaluating demographic subgroup data in a sample of approved medical products found a mere 27 percent of studies provided race and ethnicity analysis for medical devices, though race and ethnicity were correlated to observed differences in several devices.3 Further, information was often unavailable to evaluate the data with respect to age. 4 A separate FDA report cites findings that a mere 5 percent of African Americans and 1 percent of Hispanics comprise clinical trial participants compared to their 12 percent and 16 percent representation in the United States population, respectively.5
FDA emphasizes the importance of stratifying analysis based on demographic characteristics as they may explain seeming differences in clinical outcomes. Advanced age and pediatric populations,6 for example, often present co-morbidities and development considerations likely to influence device performance.
Guidance for Sponsors
Sponsors are encouraged to identify how age, race, and ethnicity influence the targeted disease condition prevalence, diagnosis, treatment, and outcomes—as well as the extent to which relevant subgroups have been included in past studies—in the risk analysis of the initial investigational plan and marketing application and interim and final reports of post-market studies.
The guidance also provides an abundance of strategies—including diversifying investigational sites, alternative recruitment and communication efforts, and cultural competency training—to sponsors and investigators to facilitate enrollment and retention of underrepresented groups. FDA also encourages sponsors to discuss meaningful demographic outcome analysis with the agency to determine if the risk-benefit profile is accurate; plus, FDA provides recommendations for presenting demographic information publicly and decision trees for applicable statistical recommendations based on study design type.
Lastly, the guidance highlights that, generally, a robust study design must adequately assess and account for the statistical heterogeneity across—and within—all demographic subgroups during both the pre- and post-marketing stages. Failing to do so may confound underlying patient characteristics and generate erroneous results.
Significant entry barriers exist for minorities in clinical research,7 and under-representation based on age, race, and ethnicity represents an overall diminished ability to evaluate device safety and effectiveness. Auspiciously, great strides are being made by FDA to identify and address the need to broaden participation and reporting based on demographic characteristics.
Comments on the draft guidance are accepted through September 19, 2016.
 For example, prior to 1993, per FDA policy women of childbearing potential were categorically precluded from participation in phase 1 and 2 clinical trials. See 1977 FDA Guideline General Consideration for the Clinical Evaluation of Drugs Publication No. (FDA) 77-3040; See also FDA Guideline Guideline for the Study and Evaluation of Gender Differences in the Clinical Evaluation of Drugs (Part VI), 58 Fed. Reg. 39406, 39407-8. See also Report to Congress: Food and Drug Administration Amendments Act (FDAAA) of 2007, Public Law No. 110-85 Section 901 of the Federal Food, Drug, and Cosmetic Act: Direct-to-Consumer Advertising’s Ability to Communicate to Subsets of the General Population; Barriers to the Participation of Population Subsets in Clinical Drug Trials (September 2009).
 This includes evaluations of Automatic Class III Designations, humanitarian device exemption (HDE) applications and post-market approval studies. FDA notes the final guidance will become part of their Collection of Race and Ethnicity Data in Clinical Trials (September, 2005) and recommends using this guidance in conjunction with the FDA guidance Evaluation of Sex-Specific Data in Medical Device Clinical Studies (August 22, 2014).
 FDA guidance Premarket Assessment of Pediatric Medical Devices (March 2014) subdivides pediatric participants by the following categories: Newborn/neonate (birth – 1 month), Infant (> 1 month – 2 years), Child (>2 years – 12 years), Adolescent (>12 years – 21 years). FDA also notes guidance relating to medical device application requirements to sufficiently describe pediatric populations impacted by the relevant disease condition under the Pediatric Medical Device Safety and Improvement Act (PMDSIA).
 While the provision of research services is often provided at no cost, corresponding necessary clinical care procedures are often the responsibility of study participants. As exemplar see Allen L. Gifford et al., Participation in Research and Access to Experimental Treatments by HIV-Infected Patients, 346 N. Eng. J. Med. 1373-1382 (2002) (study evaluating use and access to clinical research in HIV populations found minorities less likely to have insurance or receive care or have access to care through a primary care provider).