BOX 7-3Regulatory Decision Making Regarding Clinical Strategy as Discussed by Schneeweiss

As discussed, the utility of a treatment depends not just on the molecular makeup of the drug and how it impacts a disease, but on a host of usage factors, including access, compliance, dosing schedules, need for monitoring, and patient preferences. Sebastian Schneeweiss, professor of medicine and epidemiology, Harvard Medical School and Brigham & Women's Hospital, asked: “To what extent does the regulator have the discretion to go down the road of regulating clinical strategies versus the molecules very narrowly?” For example, if an oral antipsychotic works better than an injectable antipsychotic on a molecular level (i.e., when patients are perfectly compliant), but the injectable antipsychotic is more effective in practice, could a regulator approve this indication based on this information?

From: 7, How Tightly Should Investigators Attempt to Control or Restrict Treatment Quality in a Pragmatic or Real-World Trial?

Cover of Examining the Impact of Real-World Evidence on Medical Product Development
Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series.
National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Sciences Policy; Forum on Drug Discovery, Development, and Translation; Shore C, Gee AW, Kahn B, et al., editors.
Washington (DC): National Academies Press (US); 2019 Feb 6.
Copyright 2019 by the National Academy of Sciences. All rights reserved.

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