Demographic representation in clinical trials for cell-based therapy.

Area of Science:

• Regenerative Medicine
• Clinical Research
• Health Equity

Background:

• Inclusion of diverse populations in clinical research is essential for assessing novel therapy safety and efficacy.
• Pharmacokinetics and pharmacodynamics vary across demographic groups, impacting treatment generalizability.
• Under-representation of women and minorities in clinical trials can lead to impaired generalizability of findings.

Purpose of the Study:

• To analyze voluntary participation rates of different demographic groups in cell-based therapy clinical trials.
• To compare participant demographics against disease prevalence in the patient population.
• To identify challenges in achieving representative inclusion in cell-based therapy research.

Main Methods:

• Retrospective analysis of eight cell-based therapy clinical trials conducted between 2007 and 2017.
• Comparison of participant demographic data with disease prevalence data from the University of Miami Health System.
• Calculation of participation to prevalence ratios (PPR) for different demographic groups.

Main Results:

• Overall, 29.5% of participants were Hispanic and 20% were women.
• Women were under-represented in most trials, except for dilated cardiomyopathy and aging-frailty.
• Hispanic and Black patients were under-represented in several disease-specific trials, while white patients were over-represented in others.

Conclusions:

• Cell-based therapy trials face challenges in achieving representative demographic inclusion.
• Significant under-representation of women and minority groups was observed in several trial categories.
• Novel community engagement strategies are needed to enhance the inclusion of under-represented groups in future research.