Redesign of trials under different enrollment mixes

Area of Science:

• Cardiovascular research
• Clinical trial design
• Health equity

Background:

• Historical heart disease research predominantly featured male participants.
• Exclusion of female participants in past trials raises concerns about generalizability and completeness of findings.
• The mid-to-late 20th century saw several large-scale, multi-center cardiovascular trials focused exclusively on men.

Purpose of the Study:

• To re-evaluate two significant male-only cardiovascular trials from the 1970s and 1980s.
• To analyze the potential impact on statistical power and financial cost if females had been included in these historical trials.
• To highlight the importance of inclusive participant selection in clinical research.

Main Methods:

• Retrospective analysis of two historical male-only cardiovascular trials.
• Simulation of trial designs incorporating female participants.
• Estimation of changes in required sample size and overall study cost.

Main Results:

• Inclusion of females would have necessitated larger sample sizes.
• Increased participant numbers would have led to higher overall study costs.
• Potential for enhanced statistical power and broader applicability of findings.

Conclusions:

• Historical cardiovascular trials that excluded females may have had limitations in scope and applicability.
• Designing trials with inclusive participant criteria is crucial for robust and generalizable scientific evidence.
• Future research should prioritize sex-specific analyses and diverse participant recruitment.