Inequitable Racial and Ethnic Representation in Duchenne Muscular Dystrophy Clinical Trials.

Area of Science:

• Clinical trial diversity
• Rare disease research
• Health equity

Background:

• Duchenne muscular dystrophy (DMD) disproportionately affects certain populations.
• Clinical trials are crucial for developing effective treatments for rare diseases like DMD.
• Ensuring diverse participation in clinical trials is essential for generalizability of results.

Purpose of the Study:

• To analyze the racial and ethnic distribution of participants in Duchenne muscular dystrophy (DMD) phases II and III clinical trials.
• To assess the completeness of reporting for demographic data in DMD clinical trials.
• To identify potential disparities in clinical trial participation.

Main Methods:

• Systematic review of 36 Duchenne muscular dystrophy (DMD) phases II and III clinical trials registered between 2005 and 2018.
• Analysis of publicly available demographic data from clinicaltrials.gov.
• Examination of trial funding sources and geographic locations.

Main Results:

• White individuals constituted 84% of participants in DMD clinical trials.
• A significant percentage of trials (22%) failed to report racial data, and 44% omitted ethnicity data.
• Industry funding dominated DMD trials (89%), with minimal representation from the National Institutes of Health (3%).

Conclusions:

• Duchenne muscular dystrophy (DMD) clinical trials show a significant underrepresentation of minority populations.
• Inconsistent and insufficient reporting of racial and ethnic data hinders accurate assessment of trial diversity.
• There is an urgent need to implement strategies for enhancing diversity and ensuring equitable representation in future DMD clinical trials.