Defelippe, Victoria M;
J M W van Thiel, Ghislaine;
Otte, Willem M;
Schutgens, Roger EG;
Stunnenberg, Bas;
Cross, Helen J;
O'Callaghan, Finbar;
... Braun, Kees PJ; + view all
(2023)
Toward responsible clinical n-of-1 strategies for rare diseases.
Drug Discovery Today
, 28
(10)
, Article 103688. 10.1016/j.drudis.2023.103688.
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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.
| Type: | Article |
|---|---|
| Title: | Toward responsible clinical n-of-1 strategies for rare diseases |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.drudis.2023.103688 |
| Publisher version: | https://doi.org/10.1016/j.drudis.2023.103688 |
| Language: | English |
| Additional information: | Copyright 2023 The Authors. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
| Keywords: | single patient trial, methodology, clinical care, rare diseases, personalized medicine, learning healthcare systems |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10175735 |
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