Pragmatic clinical trials: U.S. payers' views on their value

Area of Science:

• Health economics
• Clinical trial design
• Pharmaceutical policy

Background:

• Randomized controlled trials (RCTs) traditionally inform regulatory decisions.
• Pragmatic clinical trials (PCTs) are emerging as valuable tools for post-regulatory decision-making.
• PCTs utilize head-to-head comparisons, diverse populations, and patient-centered outcomes.

Purpose of the Study:

• To understand U.S. insurers' and public payers' perceptions of pragmatic clinical trials (PCTs).
• To assess the value of PCTs in the evaluation of new prescription drugs by payers.

Main Methods:

• Qualitative evidence gathered through in-depth interviews.
• Interviews conducted with formulary decision-makers from 15 major U.S. payer organizations.
• Study design informed by prior literature and exploratory interviews.

Main Results:

• Payers generally view PCTs favorably, particularly their use of diverse patient populations and active comparator drugs.
• Payers will incorporate PCT results into their evidence synthesis but do not expect them to replace internal data analyses.
• Concerns exist regarding industry sponsorship of PCTs, which can reduce perceived value, though payers will still carefully review results.

Conclusions:

• Trial designers should tailor PCTs to specific diseases and incorporate head-to-head comparisons in later phases (Phase IIIb).
• Phase IV PCTs should be designed to include broader patient populations.
• Payers' perspectives offer valuable insights for optimizing PCT design and utility in drug assessment.