Use of pragmatic clinical trials in reimbursement decisions

Abstract

Abstract Randomized controlled trials (RCTs) have long been considered the highest level of evidence for clinical efficacy. However, these studies are conducted under highly controlled conditions and in carefully selected populations. Pragmatic clinical trials (pRCTs) aim to answer questions about products with broader patient populations, active comparators, or both. Other traits of pRCTs may include outcomes of interest to patients, prescribers, and payers and usual medical care per routine clinical practice. Decision-makers usually assemble several types of evidence and then synthesize the collective information to make their payer decisions about drug reimbursement and coverage. Currently, pRCTs are not frequently used by payers in their evidence reviews, but use of pRCTs in evidence-based payer decision-making may increase in the future if they are being conducted for regulatory purposes or for coverage with evidence development.