Use Of Pragmatic Clinical Trials To Support Drug Launch

• Published in: Value in health Vol. 20; no. 9; pp. A660 - A661
• Main Authors: Rémuzat, C, Kloc, K, Toumi, M
• Format: Journal Article
• Language: English
• Published: Lawrenceville Elsevier Science Ltd 01.10.2017
• Subjects: Bias; Clinical research; Clinical trials; Decision makers; Decision making; Drug development; Health care expenditures; Internal validity; Patient-centered care; Pragmatism; Public sector; R&D; Recruitment; Research & development; Uncertainty; Websites; United States > US; Europe
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2017.08.1585

OBJECTIVES: Pragmatic clinical trials (PCTs) are currently an important topic of interest in drug development. Increasing external validity of trials, PCTs have the potential to close the gap between internal validity of explanatory trials and real-life practice. The objective of this study was to assess the current use of PCTs to support drug launch. METHODS: Peer-reviewed articles on PCTs were searched in Medline/ Embase through Ovid. In addition, search of the EUnetHTA and EU Commission websites was performed, supplemented by Google search of grey literature. RESULTS: PCTs are currently on the rise; however, their use to support drug launch is uncommon. PCTs have several limitations, i.e., 1) methodological/analytical issues inherent to the potential risk of bias of more flexible designs, 2) operational issues with coordinating the recruitment of large patient populations from different settings, 3) concerns of regulatory and ethical nature, e.g. securing rights and interests of participants while remaining pragmatically flexible, 4) substantial costs incurred to conduct large PCTs. In the United States, PCTs are currently mainly promoted through public sector funding, while their potential use by regulators and payers is still uncertain. Before PCTs can be used for decision-making, academic experience (e.g. from the National Institutes of Health Health Care Systems Research Collaboratory, the National Patient-Centered Clinical Research Network, the Clinical Trials TYansformation Initiative) with these trials should broaden. In Europe, PCTs are usually considered by decision-makers as complementing (but not replacing) standard randomised clinical trials. Evidence from PCTs seems more acceptable for drugs with a known benefit/risk profiles, while less acceptable for drugs with novel mechanisms of action. CONCLUSIONS: PCTs are currently underused for initial drug regulatory/reimbursement dossier filing. Currently, multi-stakeholder initiatives are undertaken to enhance pragmatism in clinical trials and reduce decisionmaking uncertainty through developing tools, guidelines, and research on new trial designs and statistical methodologies.