Please use this identifier to cite or link to this item: https://ahro.austin.org.au/austinjspui/handle/1/33176
Title: Toward responsible clinical n-of-1 strategies for rare diseases.
Austin Authors: 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
Affiliation: UMCU Brain Center, Department of Neurology and Neurosurgery, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands
Department of Medical Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.
University College London (UCL) Great Ormond Street, Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.
Van Creveldkliniek, Benign Hematology Center, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX Utrecht, the Netherlands
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.
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.
Fondazione Mondino National Institute of Neurology, University of Pavia, Via Mondino 2, 27100, Pavia, Italy.
Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.
Medicine (University of Melbourne)
Department of Genetics, Center for Molecular Medicine, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.
Department of Neuroscience, Monash University, Melbourne, VIC, Australia.
Issue Date: Oct-2023
Date: 2023
Publication information: Drug Discovery Today 2023-10; 28(10)
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 sit 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. Teaser: N-of-1 strategies are considered a hybrid between research and care; therefore, which regulations n-of-1 strategies must abide by remains a topic of debate. We set the standards for responsible n-of-1 approaches as optimized care.
URI: https://ahro.austin.org.au/austinjspui/handle/1/33176
DOI: 10.1016/j.drudis.2023.103688
ORCID: 
Journal: Drug Discovery Today
Start page: 103688
PubMed URL: 37356616
ISSN: 1878-5832
Type: Journal Article
Subjects: clinical care
methodology
personalized medicine, learning healthcare systems
rare diseases
single patient trial
Appears in Collections:Journal articles

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