Toward responsible clinical n-of-1 strategies for rare diseases
Publication date
2023-10
Authors
Defelippe, Victoria M
van Thiel, Ghislaine J.M.W.
Otte, Willem M.
Schutgens, Roger E G
Stunnenberg, Bas
Cross, Helen J
O'Callaghan, Finbar
De Giorgis, Valentina
Jansen, Floor E.
Perucca, Emilio
Editors
Advisors
Supervisors
Document Type
Article
Metadata
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License
cc_by
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 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.
Keywords
clinical care, learning healthcare systems, methodology, personalized medicine, rare diseases, single patient trial, Drug Discovery, Pharmacology, Review, Journal Article
Citation
Defelippe, V M, J M W van Thiel, G, Otte, W M, Schutgens, R E G, Stunnenberg, B, Cross, H J, O'Callaghan, F, De Giorgis, V, Jansen, F E, Perucca, E, Brilstra, E H & Braun, K P J 2023, 'Toward responsible clinical n-of-1 strategies for rare diseases', Drug Discovery Today, vol. 28, no. 10, 103688. https://doi.org/10.1016/j.drudis.2023.103688