The SMA Clinical Trial Readiness Program: creation and evaluation of a program to enhance SMA trial readiness in the United States

Abstract

Spinal muscular atrophy (SMA) is a rare neuromuscular disease with a rapidly evolving treatment landscape. To better meet the needs of trial sponsors and the patient community in the United States (US) in this evolving context, Cure SMA established a clinical trial readiness program for new and prospective SMA clinical trial sites. Program development was informed by a review of the SMA clinical trial landscape, successful NMD trial and care networks, and factors important to effective trial conduct in SMA. The program was piloted in 2018 with a virtual site readiness evaluation, a trial readiness toolkit, and a readiness program for physical therapists and clinical evaluators. Nine US research hospitals participated in the pilot. Cure SMA evaluated the pilot program and resources through feedback surveys, which supported the program’s relevance and value. Since 2018, the program has been expanded with additional sites, new best practices toolkits, and workshops. In partnership with Cure SMA, SMA Europe is also extending programming to European countries. The program is significant as an example of a patient advocacy group working successfully with pharmaceutical companies, other patient advocacy organizations, and research hospitals to promote trial readiness, and may serve as a model for organizations in other regions and diseases.

Lessons learned implementing and managing the DIVERT-CARE trial: practice recommendations for a community-based chronic disease self-management model

“…We recommend conducting a robust readiness assessment of sites. Other implementation experts have found utility in assessing sites for readiness including understanding capacity and process measure data [ 35 – 37 ].…”

Challenges and opportunities for improving the landscape for Lewy body dementia clinical trials

“…Over the years, partnerships have developed among academic medical experts, patient advocacy groups, pharmaceutical industries, and government bodies for neurological and other diseases to address clinical and research challenges. These collaborations have increased awareness and diagnosis of the condition, improved access to expert care, fostered interest for drug development, and promoted clinical trial site readiness, all critically important for increasing the likelihood of recruitment and completion of clinical trials [ 7 , 8 ]. These partnerships may help reduce barriers in clinical research such as shortening the long latency between drug development and Food and Drug Administration (FDA) approval, reducing the frequency of failed drug trials, and potentially lessening the economic investment for drug development [ 9 ].…”

The Cure SMA Clinical Trial Experience Survey: A Study of Trial Participant Perspectives on Clinical Trial Management and Patient-Centric Management Practices

“…The Cure SMA Clinical Trial Experience Survey—conducted in conjunction with the Cure SMA Clinical Trial Readiness Program [ 22 ]—sought to identify methods for optimizing trial management in SMA, to ultimately improve participants’ experiences. This survey evaluated motivations for trial participation, participant and caregiver expectations and experiences, and how trial management and logistics, communication, and stressors impact expectations and experiences.…”