Replacing RCTs with real world data for regulatory decision making: a self-fulfilling prophecy?
BMJ 2023; 380 doi: https://doi.org/10.1136/bmj-2022-073100 (Published 02 March 2023) Cite this as: BMJ 2023;380:e073100
- Beate Wieseler, head of drug assessment department1,
- Mattias Neyt, senior researcher2,
- Thomas Kaiser, head of drug assessment department1,
- Frank Hulstaert, senior researcher2,
- Jürgen Windeler, director1
- 1Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany
- 2Belgian Health Care Knowledge Centre (KCE), Brussels, Belgium
- Correspondence to: B Wieseler beate.wieseler{at}iqwig.de
Key messages
Enthusiasm is growing for the use of observational real world data as a basis for regulatory, clinical, and health policy decision making
But such study designs are ill suited to measure the treatment effects of new drugs
Promoting the use of observational studies from routine practice data sources might hinder efforts needed to improve the feasibility of randomised controlled trials
To ensure high quality and efficient healthcare, the conduct of RCTs, including those in routine care, should be made easier, faster, and cheaper
Attempts to accelerate the provision of new treatments have led to evidence that is limited in quantity and quality being submitted for regulatory approval in recent years.12 Approvals based on single arm trials, for example, have become more frequent, such as for lisocabtagene maraleucel, a CAR-T cell therapy for patients with B cell lymphoma. Single arm trials are not informative enough to enable us to select the best therapy for a patient among several options. This lack of robust evidence, especially the lack of comparisons with standard care, has implications for decision making in clinical practice and health policy, as the place of new drugs in the treatment landscape remains unclear, and reimbursement and pricing decisions cannot be adequately informed.34
Current discussions of the most suitable study types for regulatory approval and the question of whether observational data instead of data from randomised controlled trials (RCTs) would suffice are relevant beyond regulatory agencies.56 The question is intertwined with the potential use of routine practice data, often referred to as real world data or real world …
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