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
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

103688

Informations de copyright

Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.

Auteurs

Victoria M Defelippe (VM)

Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France. Electronic address: v.m.defelippe-2@umcutrecht.nl.

Ghislaine J M W van Thiel (G)

Department of Medical Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.

Willem M Otte (WM)

Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.

Roger E G Schutgens (REG)

Van Creveldkliniek, Benign Hematology Center, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX Utrecht, the Netherlands; European Reference Network for Oncological and non-oncological Rare Hematological Diseases (EuroBloodNet), Hôpital St Louis / Université Paris 7, 1 Avenue Claude Vellefaux, 75475 Paris, France.

Bas Stunnenberg (B)

Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Donders Center for Medical Neuroscience, Radboud University Medical Center, Thomas van Aquinostraat 4, 6525 GD Nijmegen, the Netherlands.

Helen J Cross (HJ)

European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; University College London (UCL) Great Ormond Street, Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.

Finbar O'Callaghan (F)

European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Paediatric Neuroscience, UCL Great Ormond Street, Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.

Valentina De Giorgis (V)

European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Fondazione Mondino National Institute of Neurology, University of Pavia, Via Mondino 2, 27100 Pavia, Italy.

Floor E Jansen (FE)

Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France.

Emilio Perucca (E)

European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Department of Medicine, University of Melbourne (Austin Health), Heidelberg, VIC 3084, Australia; Department of Neuroscience, Monash University, Melbourne, VIC, Australia.

Eva H Brilstra (EH)

European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Department of Genetics, Center for Molecular Medicine, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.

Kees P J Braun (KPJ)

European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.

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