Beyond current research practice: Methodological considerations in MS rehabilitation research (is designing the perfect rehabilitation trial the Holy Grail or a Gordian knot?)

das Nair, Roshan; de Groot, Vincent; Freeman, Jennifer

Abstract

Rehabilitation is an essential aspect of symptomatic and supportive treatment for people with multiple sclerosis (MS). MS rehabilitation research has grown considerably, but the volume of robust evidence to guide effective clinical practice in the form of high quality randomised controlled trials (RCTs) is low 1. Such robust research evidence is important because it is a key driver for optimising patient outcomes and service delivery 2 , and influencing health policy decision making. This discussion paper explores some of the challenges of undertaking robust clinical trials within this field. We acknowledge the importance of using a variety of research methods to fully understand whether and how rehabilitation interventions work, but focus on RCTs since they are arguably regarded as a superior research design to determine whether a treatment is beneficial 3. However, RCTs have largely been modelled on pharmacological trials where the precise dose of the intervention, double blind procedures, and safety reporting mechanisms can be strictly adhered to. Such issues become much more complex in rehabilitation trials. Therefore, additional guidelines for conducting and reporting non-pharmacological trials have been published 4. Despite these advances, reporting has not yet significantly improved 5 and specific challenges persist. We describe some key methodological issues faced in rehabilitation trials, and draw upon examples from both within and outside the MS field, where these dilemmas have been (at least partially) tackled. We make some suggestions as to how future research can be scientifically robust whilst remaining relevant and "fit for purpose" in generating new knowledge, which can meaningfully shape rehabilitation practice and improve patient outcomes. Trial designs-efficacy vs. effectiveness trials All RCT designs share some common features (e.g., randomisation), but they also differ based on the general aims of the RCT. Efficacy (or explanatory) trials, which are often highly controlled, aim to answer whether an intervention produces benefits for patients under ideal (often laboratory) conditions, and are typically used to demonstrate 'proof-of-principle' for newer interventions. An example of an efficacy rehabilitation trial is Dalgas et al. 5 .

Citation

das Nair, R., de Groot, V., & Freeman, J. (2019). Beyond current research practice: Methodological considerations in MS rehabilitation research (is designing the perfect rehabilitation trial the Holy Grail or a Gordian knot?). Multiple Sclerosis, 25(10), 1337-1347. https://doi.org/10.1177/1352458519858271

• Journal Article Type: Article
• Acceptance Date: May 9, 2019
• Online Publication Date: Aug 30, 2019
• Publication Date: 2019-09
• Deposit Date: May 13, 2019
• Publicly Available Date: May 13, 2019
• Journal: Multiple Sclerosis Journal
• Print ISSN: 1352-4585
• Electronic ISSN: 1477-0970
• Publisher: SAGE Publications
• Peer Reviewed: Peer Reviewed
• Volume: 25
• Issue: 10
• Pages: 1337-1347
• DOI: https://doi.org/10.1177/1352458519858271
• Keywords: Rehabilitation, randomised controlled trials, complex interventions, mixed-methods, methodology
• Public URL: https://nottingham-repository.worktribe.com/output/2042973
• Publisher URL: https://journals.sagepub.com/doi/full/10.1177/1352458519858271