Dataset Information

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.

SUBMITTER: Peterson I

PROVIDER: S-EPMC7564894 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

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

Peterson Ilse I Cruz Rosángel R Sarr Fatou F Stanley Ann Marie AM Jarecki Jill J

Orphanet journal of rare diseases 20200522 1

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 con ...[more]

PMID: 32443972

Similar Datasets

Project description:Background/aimsRecruitment and retention of research participants are challenging and critical components of successful clinical trials and natural history studies. Infants with spinal muscular atrophy (SMA) have been a particularly challenging population to study due to their fragile and complex medical issues, poor prognosis and, until 2016, a lack of effective therapies. Recruitment of healthy infants into clinical trials and natural history studies is also challenging and sometimes assumed to not be feasible.MethodsIn 2011, our group initiated a two-year, longitudinal natural history study of infants with SMA and healthy infant controls to provide data to assist in the analysis and interpretation of planned clinical trials in infants with SMA. The recruitment goal was to enroll 27 infants less than 6 months of age with SMA and 27 age-matched healthy infants within the two-year enrollment period. A detailed recruitment and retention plan was developed for this purpose. In addition, a survey was administered to participant families to understand the determinants of participation in the study.ResultsAll healthy infants were recruited within the study's first year and 26 SMA infants were recruited within the two-year recruitment period. Thirty-eight participant families responded to the recruitment determinants survey. Nearly half of respondents (18/38, 48%) reported that they first heard of the study from their physician or neurologist. The most common reason to decide to enroll their infant (22/38, 58%) and to remain in the study (28/38, 74%) was their understanding of the importance of the study. Thematic recruitment tools such as a study brochure, video on social media, and presentations at advocacy meetings were reported to positively influence the decision to enroll.ConclusionsA proactive, thematic and inclusive recruitment and retention plan that effectively communicates the rationale of a clinical study and partners with patients, advocacy groups and the local communities can effectively recruit participants in vulnerable populations. Recommendations for the proactive integration of recruitment and retention plans into clinical trial protocol development are provided.

Project description:BackgroundDigital health has the potential to support care delivery for chronic illness. Despite positive evidence from localized implementations, new technologies have proven slow to become accepted, integrated, and routinized at scale.ObjectiveThe aim of our study was to examine barriers and facilitators to implementation of digital health at scale through the evaluation of a £37m national digital health program: ?Delivering Assisted Living Lifestyles at Scale" (dallas) from 2012-2015.MethodsThe study was a longitudinal qualitative, multi-stakeholder, implementation study. The methods included interviews (n=125) with key implementers, focus groups with consumers and patients (n=7), project meetings (n=12), field work or observation in the communities (n=16), health professional survey responses (n=48), and cross program documentary evidence on implementation (n=215). We used a sociological theory called normalization process theory (NPT) and a longitudinal (3 years) qualitative framework analysis approach. This work did not study a single intervention or population. Instead, we evaluated the processes (of designing and delivering digital health), and our outcomes were the identified barriers and facilitators to delivering and mainstreaming services and products within the mixed sector digital health ecosystem.ResultsWe identified three main levels of issues influencing readiness for digital health: macro (market, infrastructure, policy), meso (organizational), and micro (professional or public). Factors hindering implementation included: lack of information technology (IT) infrastructure, uncertainty around information governance, lack of incentives to prioritize interoperability, lack of precedence on accountability within the commercial sector, and a market perceived as difficult to navigate. Factors enabling implementation were: clinical endorsement, champions who promoted digital health, and public and professional willingness.ConclusionsAlthough there is receptiveness to digital health, barriers to mainstreaming remain. Our findings suggest greater investment in national and local infrastructure, implementation of guidelines for the safe and transparent use and assessment of digital health, incentivization of interoperability, and investment in upskilling of professionals and the public would help support the normalization of digital health. These findings will enable researchers, health care practitioners, and policy makers to understand the current landscape and the actions required in order to prepare the market and accelerate uptake, and use of digital health and wellness services in context and at scale.

Dataset Information

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

Publications

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

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets