Toward responsible clinical n-of-1 strategies for rare diseases
Defelippe, Victoria M; J M W van Thiel, Ghislaine; Otte, Willem M; Schutgens, Roger E G; Stunnenberg, Bas; Cross, Helen J; O'Callaghan, Finbar; De Giorgis, Valentina; Jansen, Floor E; Perucca, Emilio; Brilstra, Eva H; Braun, Kees P J
(2023) Drug Discovery Today, volume 28, issue 10
(Article)
Abstract
N-of-1 strategies can provide high-quality evidence of treatment efficacy at the individual level and optimize evidence-based selection of off-label treatments for patients with rare diseases. Given their design characteristics, n-of-1 strategies are considered to lay at the intersection between medical research and clinical care. Therefore, whether n-of-1 strategies should be
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governed by research or care regulations remains a debated issue. Here, we delineate differences between medical research and optimized clinical care, and distinguish the regulations which apply to either. We also set standards for responsible optimized clinical n-of-1 strategies with (off-label) treatments for rare diseases. Implementing clinical n-of-1 strategies as defined here could aid in optimized treatment selection for such diseases.
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Keywords: clinical care, learning healthcare systems, methodology, personalized medicine, rare diseases, single patient trial, Drug Discovery, Pharmacology, Review, Journal Article
ISSN: 1359-6446
Publisher: Elsevier Limited
Note: Funding Information: Research Centre at Great Ormond Street Hospital. She holds an endowed chair at UCL Great Ormond Street Institute of Child Health; she holds grants from NIHR, EPSRC, GOSH Charity, ERUK, and the Waterloo Foundation. Funding Information: H.J.C. has acted as an investigator for studies with GW Pharma, Zogenix, Vitaflo, and Marinius. She has been a speaker and on advisory boards for GW Pharma, Zogenix, and Nutricia; all remuneration has been paid to her department. Her research is supported by the NIHR Biomedical. Funding Information: The authors would like to thank members of the ERN EpiCARE and the European Patient Advocacy Groups who have expressed their insights on the use of n-of-1 strategies to improve treatment selection. The figure and box were made using BioRender. This work was supported by Health-Holland [under grant no. LSHM21053-SGF] and MING fonds. Publisher Copyright: © 2023 The Authors
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