Cure SMA is pleased to announce the launch of Phase 11 initiates for the SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration, we fund projects to ensure that effective, safe treatments can progress through clinical trials quickly and gain approval from the U.S. Food and Drug Administration (FDA) and international regulators. Our work also ensures that these treatments address the unmet needs of the SMA community, and that the community’s priorities and goals are incorporated into the development, review, and approval of therapies. Click here to see what the SMA Industry Collaboration achieved during Phase 10 (2025)!
Cure SMA and our partners at Novartis, Scholar Rock, Biogen, Genentech/Roche, argenx, NMD Pharma, and SMA Europe are building upon previous achievements to further refine outcome measures, improve clinical trial awareness, and to identify the SMA community’s unmet needs and treatment goals. Additionally, the Industry Collaboration is conducting several initiatives to support SMA drug approval and coverage, and drive improvements with treatment decision making.
The SMA Industry Collaboration is sub-divided into “Topic Groups” that each have a particular focus and specific goals. Every year, each Topic Group outlines the projects it will complete to achieve those goals. Below is a description of ongoing Phase 11 priorities for each SMA Industry Collaboration Topic Group.
Topic Group 1: Approaches to Enabling Drug Development and Clinical Trials
Goals:
- To engage regulatory authorities and deepen their understanding of SMA and the perspectives of SMA-affected individuals on which treatment outcomes are clinically meaningful.
- To enhance SMA clinical trials by developing healthcare provider training resources and improving treatment outcome measures.
- To support initiatives to facilitate equitable clinical trial access for all members of the SMA community.
Phase 11 Projects:
- Engagement with regulatory authorities to support SMA drug development and approval through sharing of SMA community data on topics such as risk/benefit and unmet needs.
- Development of a Global Set of Patient Centered Outcomes for SMA in partnership with the International Consortium for Health Outcomes Measurement (ICHOM).
- Development of a patient-centered value framework to identify outcomes of meaningful benefit to individuals living with SMA.
- Continued support of the SMA Registry and Clinical Trial Finder.
Topic Group 2: Cure SMA Patient and Caregiver-Reported Data
Goal:
- To develop tools to collect patient and caregiver-reported data that will help us better understand the natural history of SMA, and the evolution of the SMA disease experience as new therapies are developed.
Phase 11 Projects:
- Distribution of the 10th Annual Cure SMA Community Update Survey.
- Publication of the 2025 State of SMA Report to highlight important trends and outcomes observed from the Cure SMA community.
- Exploration of healthcare utilization, preventative screenings, and associated barriers among adults.
Topic Group 3: Real-World Healthcare Provider Data on SMA Treatment and Supportive Care
Goal:
- To develop survey measures to capture the perspectives of healthcare providers on current and potential barriers to SMA treatment delivery and decision making.
Phase 11 Projects:
- Continued exploration of healthcare provider perspectives on decision-making and access barriers for SMA treatment.
- Assessment of current SMA treatment data gaps from the perspectives of healthcare providers in the United States and Europe.
- Comparative assessment of measures of treatment effectiveness and shared treatment decision making as reported by adults living with SMA and adult clinicians.
- Evaluation of motor function assessment trajectories in treated teens and adults living with SMA.
- Development and of the 2026 – 2027 European Patient Experience Survey on SMA (EUPESMA).
Topic Group 4: Education and Engagement on SMA Treatment and Supportive Care
Goal:
- To utilize SMA patient data and patient voice to drive healthcare provider and payor engagement and develop community resources to enhance treatment access.
Phase 11 Projects:
- Development of grand rounds presentation to enhance provider awareness on the latest updates in SMA treatment and care.
- Hosting a webinar to educate the SMA community on the insurance denials and appeals processes.
- Translation of the newly revised Health Insurance Roadmap to enable broad access and usability for all members of the SMA community.
SMA Industry Collaboration Phase 10 (2025) Achievements
During Phase 10, the SMA Industry Collaboration spearheaded research efforts that increased SMA treatment awareness and addressed the unmet needs of the SMA community. A summary of these accomplishments may be found in the 2025 End of Year Report.
The SMA Industry Collaboration’s Phase 10 Accomplishments include:
- Publication of three research articles in peer-reviewed journals
- SMA Community Priorities for Future Drug Therapies: Cross-Sectional Survey Findings Representing 410 Adults with SMA
- Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts
- A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophy
- Engagement with regulatory authorities to support SMA drug approval
- Distribution of the 9th Annual Cure SMA Community Update Survey to track changes regarding the unmet needs of the SMA community over time
- Distribution of a survey to evaluate unmet needs of adults living with SMA across the healthcare spectrum
- Distribution of a survey to assess the diagnostic journey and treatment utilization of individuals living with SMA Type 4
- Update of the Cure SMA Health Insurance Roadmap to better support the SMA patient and healthcare provider community when navigating payer denials and appeals
- Development and distribution of a Quarterly Newsletter for healthcare providers
- Payer outreach, engagement, and advocacy to support SMA treatment coverage
- Continued maintenance of the Cure SMA Find a Treatment Center Tool – a searchable online directory
About the Cure SMA Industry Collaboration
The Cure SMA Industry Collaboration was established in 2016 to leverage the experience, expertise, and resources of pharmaceutical, biotechnology companies, and other nonprofit organizations involved in the development of spinal muscular atrophy (SMA) therapeutics to more effectively address a range of scientific, clinical, and regulatory challenges. It is currently comprised of our partners at Novartis, Scholar Rock, Biogen, Genentech/Roche, argenx, NMD Pharma, and SMA Europe.