Diversity in Medical Device Clinical Trials: Do We Know What Works for Which Patients?

Area of Science:

• Medical Device Regulation
• Clinical Trial Diversity
• Health Equity

Background:

• Legislation since 1993 has aimed to increase diversity in NIH-funded research, with a 2012 law encouraging the US Food and Drug Administration (FDA) to analyze medical product safety and effectiveness in diverse patient groups.
• Historically, clinical trials for adult medical devices have underrepresented women, racial and ethnic minorities, and older adults.
• While the FDA reported diversity in pivotal trials and subgroup analyses for high-risk devices in 2013, the report lacked specifics on meaningful sample sizes and inclusion of all key subgroups for safety and effectiveness.

Purpose of the Study:

• To evaluate patient diversity and the availability of subgroup analyses in pivotal trials for high-risk and novel medical devices reviewed by the FDA.
• To determine if the FDA's review process adequately addresses demographic differences in medical device safety and effectiveness.

Main Methods:

• Analysis of publicly available documents for 22 high-risk or novel medical devices approved via the premarket approval pathway between 2014-2017.
• Evaluation of patient demographics and subgroup analyses (gender, race, age) within pivotal clinical trials for these devices.

Main Results:

• Only 14% of reviewed devices provided subgroup analyses for both effectiveness and safety (or sensitivity/selectivity) across gender, race, and age.
• 55% of devices reported such analyses for at least one subgroup, but sample sizes were often too small for meaningful conclusions.
• Subgroup analyses were more frequently presented to FDA Advisory Committees than included in public FDA reviews or labeling.

Conclusions:

• Despite legislative encouragement for diversity and subgroup analyses, few are publicly available for high-risk and novel medical devices.
• The lack of comprehensive, publicly accessible subgroup data hinders clinicians and patients from assessing device safety and effectiveness for specific demographic groups.
• Improved transparency and robust subgroup analyses are crucial for ensuring equitable and informed use of medical devices across diverse populations.