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

Author

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

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.

Suggested Citation

  • Gaasterland, C.M.W. & Jansen-van der Weide, M.C. & Vroom, E. & Leeson-Beevers, K. & Kaatee, M. & Kaczmarek, R. & Bartels, B. & van der Pol, W.L. & Roes, K.C.B. & van der Lee, J.H., 2018. "The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design," Health Policy, Elsevier, vol. 122(12), pages 1287-1294.
    • Handle: RePEc:eee:hepoli:v:122:y:2018:i:12:p:1287-1294
    DOI: 10.1016/j.healthpol.2018.09.011
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    References listed on IDEAS

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    1. Elberse, Janneke Elisabeth & Pittens, Carina Anna Cornelia Maria & de Cock Buning, Tjard & Broerse, Jacqueline Elisabeth Willy, 2012. "Patient involvement in a scientific advisory process: Setting the research agenda for medical products," Health Policy, Elsevier, vol. 107(2), pages 231-242.
    2. Boote, Jonathan & Baird, Wendy & Beecroft, Claire, 2010. "Public involvement at the design stage of primary health research: A narrative review of case examples," Health Policy, Elsevier, vol. 95(1), pages 10-23, April.
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