The critical need to accelerate cerebral palsy research with consumer engagement, global networks, and adaptive designs.

Journal: Journal of pediatric rehabilitation medicine

Volume: 17

Issue: 1

Year of Publication: 2024

Affiliated Institutions:  H. Ben Taub Department of Physical Medicine and Rehabilitation and Departments of Neurosurgery and Pediatrics, Baylor College of Medicine, Houston, TX, USA. Faculty of Medicine and Health, The University of Sydney, Sydney, Australia. Elsass Foundation, Charlottenlund, Denmark. Cerebral Palsy Research Network, Greensville, SC, USA.

Abstract summary 

The prevalence of cerebral palsy (CP) varies globally, with higher rates and burden of disease in low- and middle-income countries. CP is a lifelong condition with no cure, presenting diverse challenges such as motor impairment, epilepsy, and mental health disorders. Research progress has been made but more is needed, especially given consumer demands for faster advancements and improvements in the scientific evidence base for interventions. This paper explores three strategies to accelerate CP research: consumer engagement, global clinical trial networks, and adaptive designs. Consumer engagement involving individuals with lived experience enhances research outcomes. Global clinical trial networks provide efficiency through larger and more diverse participant pools. Adaptive designs, unlike traditional randomized controlled trials, allow real-time modifications based on interim analyses, potentially answering complex questions more efficiently. The establishment of a CP Global Clinical Trials Network, integrating consumer engagement, global collaboration, and adaptive designs, marks a paradigm shift. The Network aims to address consumer-set research priorities. While challenges like ethical considerations and capacity building exist, the potential benefits for consumers, clinicians, researchers, and funding bodies are substantial. This paper underscores the urgency of transforming CP research methodologies for quicker translation of novel treatments into clinical practice to improve quality of life for those with CP.

Authors & Co-authors:  Thomas Novak Ritterband-Rosenbaum Lind Webb Gross McNamara

Study Outcome 

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Statistics
Citations :  McIntyre S, Goldsmith S, Webb A, et al.. Global prevalence of cerebral palsy: A systematic analysis. Dev Med Child Neurol. 2022;64(12):1494–506. doi: 10.1111/dmcn.15346
Authors :  8
Identifiers
Doi : 10.3233/PRM-240014
SSN : 1875-8894
Study Population
Male,Female
Mesh Terms
Humans
Other Terms
Cerebral palsy (CP);adaptive designs;clinical trials;consumer engagement;global research networks
Study Design
Study Approach
Country of Study
Publication Country
Netherlands