Toward responsible clinical n-of-1 strategies for rare diseases.
clinical care
learning healthcare systems
methodology
personalized medicine
rare diseases
single patient trial
Journal
Drug discovery today
ISSN: 1878-5832
Titre abrégé: Drug Discov Today
Pays: England
ID NLM: 9604391
Informations de publication
Date de publication:
10 2023
10 2023
Historique:
received:
17
01
2023
revised:
08
06
2023
accepted:
20
06
2023
medline:
2
10
2023
pubmed:
26
6
2023
entrez:
25
6
2023
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 37356616
pii: S1359-6446(23)00204-0
doi: 10.1016/j.drudis.2023.103688
pii:
doi:
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
103688Informations de copyright
Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.