Stakeholder perspectives on adaptive clinical trials: a scoping review

Area of Science:

• Clinical trial methodology
• Biostatistics
• Drug development

Background:

• Adaptive clinical trials (ACTs) are an innovative approach using accumulating data to inform trial conduct.
• Adaptations include dose-tier comparisons, response-adaptive randomization, and stopping rules.

Purpose of the Study:

• To assess stakeholder attitudes, perspectives, and understanding of adaptive designs (ADs).

Main Methods:

• A scoping review of articles on stakeholder perspectives of adaptive designs.
• Searched four electronic databases and gray literature, screening 172 titles.
• Coded primary findings from 18 selected articles for analysis.

Main Results:

• Perceived advantages: limiting ineffective treatments, improving research efficiency.
• Perceived barriers: insufficient sample size, consent issues, bias risk, type 1 error, cost, unclear rationales, and lack of education.
• Stakeholder perceptions varied, with a notable absence of patient perspectives in the literature.

Conclusions:

• Diverse stakeholder perceptions exist regarding adaptive designs.
• Need for enhanced training, guidelines, and toolkits to address barriers.
• Crucial to educate all stakeholders, including patients, clinicians, and staff, not just principal investigators.