Personalised Medicine Using N-of-1 Trials: Overcoming Barriers to Delivery
Abstract
:1. Introduction
2. N-of-1 Trial Services
3. Establishing a Framework for Running N-of-1 Trials of Statins to Identify or Rule Out Side Effects
4. Promoting Personalised Care by Establishing N-of-1 Trials Services
- A research pharmacy to encapsulate, number and label two lots of medication: some placebo and some the drug.
- A simple randomisation system for the crossover, with appropriate time periods tailored to the clinical question, and to number the capsule packs.
- A flexible method to collect repeated self-reports of possible beneficial and unwanted effects (such as pain relief or gastrointestinal symptoms).
- Some ‘back-end’ online software to process, analyse and present the data to clinicians and patients.
Author Contributions
Funding
Acknowledgments
Conflicts of Interest
References
- Poisson, D.; et Double, L. Recherches de Statistique sur L’affection Calculeuse, par M. le Docteur Civiale. Comptes Rendus Hebdomadaires des Séances de l’Académie de Sciences (Statistical Research on (Urinary) Stone, by M. Dr Civiale); Bachelier: Paris, France, 1835; pp. 167–177. [Google Scholar]
- Mirza, R.D.; Punja, S.; Vohra, S.; Guyatt, G. The history and development of N of 1 trials. J. R. Soc. Med. 2017, 110, 330–340. [Google Scholar] [CrossRef] [PubMed]
- Guyatt, G.H.; Haynes, R.B.; Jaeschke, R.Z.; Cook, D.J.; Green, L.; Naylor, C.D.; Wilson, M.C.; Richardson, W.S. Users’ guide to the medical literature: XXV. Evidence-based medicine: Principles for applying the users’ guides to patient care. JAMA 2000, 284, 1290–1296. [Google Scholar] [CrossRef] [PubMed]
- Paterson, C. Seeking the patient’s perspective: A qualitative assessment of EuroQoL, COOP-WONCA Charts and MYMOP. Qual. Life Res. 2004, 13, 871–881. [Google Scholar] [CrossRef] [PubMed]
- Guyatt, G.; Sackett, D.; Adachi, J.; Roberts, R.; Chong, J.; Rosenbloom, D.; Keller, J. A clinician’s guide for conducting randomized trials in individual patients. CMAJ 1988, 139, 497–503. [Google Scholar] [PubMed]
- Larson, E.; Ellsworth, A.; Oas, J. Randomized clinical trials in single patients during a 2-year period. JAMA 1993, 270, 2708–2712. [Google Scholar] [CrossRef] [PubMed]
- Nikles, C.; Glasziou, P.P.; Del Mar, C.B.; Duggan, C.M.; Mitchell, G. N of 1 trials. Practical tools for medication management. Aust. Fam. Physician 2000, 29, 1108–1112. [Google Scholar] [PubMed]
- Nikles, C.; Mitchell, G.; Del Mar, C.B.; Clavarino, A.; McNairn, N. An N-of-1 trial service in clinical practice: Testing the effectiveness of stimulants for attention-deficit/hyperactivity disorder. Pediatrics 2006, 6, 117. [Google Scholar] [CrossRef] [PubMed]
- Yelland, M.J.; Nikles, C.J.; McNairn, N.; Del Mar, C.B.; Schluter, P.J.; Brown, R.M. Celecoxib compared with sustained-release paracetamol for osteoarthritis: A series of N-of-1 trials. Rheumatology 2007, 46, 135–140. [Google Scholar] [CrossRef]
- Yelland, M.J.; Poulos, C.J.; Pillans, P.I.; Bashford, G.M.; Nikles, C.J.; Sturtevant, J.M.; Vine, N.; Del Mar, C.B.; Schluter, P.J.; Tan, M.; et al. N-of-1 randomized trials to assess the efficacy of gabapentin for chronic neuropathic pain. Pain Med. 2009, 10, 754–761. [Google Scholar] [CrossRef] [PubMed]
- Vohra, S.; Moher, D. Complementary and alternative medicine in Canadian children: A call for action. Paediatr. Child Health 2005, 10, 154–156. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Chalmers, I. Adverse effects of statins (rapid response). BMJ 2014, 348, g3306. [Google Scholar]
- Chalmers, I. I’ll be On Statins all My Life, so Why Am I Unsure about the Side-Effects? The Guardian Newspaper: London, UK. Available online: https://www.theguardian.com/commentisfree/2015/apr/17/statins-all-my-life-side-effects-information (accessed on 1 November 2019).
- Herrett, E.; Williamson, E.; Beaumont, D.; Prowse, D.; Youssouf, N.; Brack, K.; Armitage, J.; Goldacre, B.; MacDonald, T.; Staa, T.V.; et al. Study protocol for statin web-based investigation of side effects (StatinWISE): A series of randomized controlled N-of-1 trials comparing atorvastatin and placebo in UK primary care. BMJ Open 2017, 7, e016604. [Google Scholar] [CrossRef] [PubMed]
- Van Staa, T.P.; Dyson, L.; McCann, G.; Padmanabhan, S.; Belatri, R.; Goldacre, B.; Cassell, J.; Pirmohamed, M.; Torgerson, D.; Ronaldson, S.; et al. The opportunities and challenges of pragmatic point-of-care randomised trials using routinely collected electronic records: Evaluations of two exemplar trials. Health Technol. Assess. 2014, 18, 1–146. [Google Scholar] [CrossRef] [PubMed]
© 2019 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
Share and Cite
Chalmers, I.; Smeeth, L.; Goldacre, B. Personalised Medicine Using N-of-1 Trials: Overcoming Barriers to Delivery. Healthcare 2019, 7, 134. https://doi.org/10.3390/healthcare7040134
Chalmers I, Smeeth L, Goldacre B. Personalised Medicine Using N-of-1 Trials: Overcoming Barriers to Delivery. Healthcare. 2019; 7(4):134. https://doi.org/10.3390/healthcare7040134
Chicago/Turabian StyleChalmers, Iain, Liam Smeeth, and Ben Goldacre. 2019. "Personalised Medicine Using N-of-1 Trials: Overcoming Barriers to Delivery" Healthcare 7, no. 4: 134. https://doi.org/10.3390/healthcare7040134
APA StyleChalmers, I., Smeeth, L., & Goldacre, B. (2019). Personalised Medicine Using N-of-1 Trials: Overcoming Barriers to Delivery. Healthcare, 7(4), 134. https://doi.org/10.3390/healthcare7040134