Earlier this year, Cure SMA launched an expanded Phase 10 of 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 9 (2024)!
Cure SMA and our partners at Biogen, Novartis, Scholar Rock, 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, with the Phase 10 expansion, the Industry Collaboration is conducting several initiatives to support SMA drug approval and coverage, and drive improvements with treatment delivery.
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 10 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 10 Projects:
- Engagement with Regulatory Authorities to Support SMA Drug Approval
- Creation of a Global Set of Patient Centered Outcomes for 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 10 Projects:
- The 9th Annual Cure SMA Community Update Survey
- Exploration of the Diagnostic Journey of Adult-Onset SMA
- Evaluating Unmet Needs of Adults Living with SMA Across the Healthcare Spectrum
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 10 Projects:
- Evaluation of Healthcare Provider Perspectives on Decision Making and Access Barriers for SMA Treatment
- Evaluating Health Disparities in the SMA Community
- Pan European Survey on Treatment Decision Making
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 10 Projects:
- Maintenance of the Cure SMA Find a Treatment Center Website
- Development and Distribution of a Quarterly Newsletter for Healthcare Providers
- Payer Outreach, Engagement, and Advocacy
- Development of Resources to Support Payor Engagement for SMA Treatment Denials and Appeals
SMA Industry Collaboration Phase 9 (2024) Achievements
During Phase 9, 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 2024 End of Year Report.
The SMA Industry Collaboration’s Phase 9 Accomplishments include:
- Publication of two research articles in peer-reviewed journals
- Development and launch of a series of resources to improve clinical trial awareness, knowledge, and access among the SMA community
- Update of the Cure SMA Clinical Trial Readiness Program Basics of SMA and the Effective Clinical Trial Conduct and Best Practices for Clinical Research Coordinators in SMA toolkits
- Distribution and analysis of a survey for adults living with SMA and caregivers to better understand the barriers that impact the communities’ access to SMA treatment and care
- Distribution of the 8th Annual Cure SMA Community Update Survey to track changes regarding the unmet needs of the SMA community over time
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 Biogen, Novartis, Scholar Rock, Genentech/Roche Pharmaceuticals, argenx, NMD Pharma, and SMA Europe.