Identifying target populations to align with decision-makers' needs

Area of Science:

• Clinical Trials
• Pharmacoepidemiology
• Health Services Research

Background:

• Randomized trials (RCTs) assess average treatment effects in enrolled participants.
• Decision-makers require evidence applicable to their specific target populations, which often differ from RCT populations.

Purpose of the Study:

• To guide researchers in aligning evidence generation with decision-maker needs.
• To identify decision-maker groups and their target populations for specific health interventions.
• To determine when RCTs alone suffice versus when real-world data is needed.

Main Methods:

• Outlined 5 key decision-maker groups: policymakers, payers, purchasers, providers, and patients.
• Specified target populations for beta-blocker effectiveness post-myocardial infarction with preserved ejection fraction.
• Summarized scenarios where RCT results apply and suggested analytic approaches.

Main Results:

• RCT generalizability varies depending on the alignment between trial and target populations.
• Real-world data and complementary analyses are often necessary to inform specific decision-maker needs.
• A structured approach is needed to bridge the gap between trial evidence and policy application.

Conclusions:

• Researchers must proactively identify decision-makers and their target populations early in the research process.
• Tailoring evidence generation to specific decision-maker contexts enhances the utility of research findings.
• Integrating RCTs with real-world data analysis is crucial for informing diverse healthcare decisions.