The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design

C.M.W. Gaasterland, M.C. Jansen-van der Weide, E. Vroom, K. Leeson-Beevers, M. Kaatee, R. Kaczmarek, B. Bartels, W.L. van der Pol, K.C.B. Roes and J.H. van der Lee

Health Policy, 2018, vol. 122, issue 12, 1287-1294

Abstract: In clinical trials, it is relevant to ask patients and/or their caregivers which aspects concerning their disease they consider important to measure when a new intervention is being investigated. Those aspects, useful as outcome measures in a trial, are of pivotal importance for the result of the trial and the subsequent decision-making. In rare diseases the choice of outcome measures may be even more important, due to the small numbers and heterogeneity of the patients that are included.

Keywords: Patient involvement; Outcomes; Trial design; Rare diseases (search for similar items in EconPapers)
Date: 2018
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Persistent link: https://EconPapers.repec.org/RePEc:eee:hepoli:v:122:y:2018:i:12:p:1287-1294

DOI: 10.1016/j.healthpol.2018.09.011

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