Cure SMA Announces Expanded Phase 7 of SMA Industry Collaboration

Cure SMA is pleased to announce the launch of an expanded Phase 7 of our 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 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. The Collaboration consists of three main topic groups, which are below.

As the SMA Industry Collaboration embarks on its seventh year, Cure SMA and our existing partners at Biogen, Genentech, Scholar Rock, Novartis Gene Therapies, and SMA Europe, welcome two new partners – Epirium Bio and Biohaven Pharmaceuticals. During Phase 7, we will build upon previous achievements to further refine outcome measures and enhance knowledge of the SMA patient experience. Additionally, we will work to identify and address disparities that limit patient access to clinical research, treatment, and care.

Phase 7 Goals

The objective of Topic Group 1, Approaches to Enabling Drug Development and Clinical Trials is to engage regulatory authorities to deepen understanding of SMA and the perspective of patients on what outcome measures are clinically meaningful. This year, the topic group will draft and update the 2017 Voice of the Patient Report. The report will discuss the shifting state of the field, and the unmet needs of affected individuals with the advent of new FDA approved therapeutics. It will also include findings from a benefit risk study to be conducted this year to assess the specific risks our community would be willing to accept in exchange for certain treatment benefits. The report will be shared with the FDA to help inform future drug approvals in SMA.

During Phase 7, the topic group will also continue efforts to develop and improve outcome measures for adults with SMA. The group is now working to develop and validate the ATEND, a wheelchair-based motor function scale, bulbar assessments of voice, speech, and swallow, and two patient reported outcome measures (PROMs)—the SMA-PRO and SMA Caregiver Reported Health Index (SMACR-HI). The goal of the SMA-PRO project is to broaden the scope of the PEDI-CAT to better meet the needs of SMA affected individuals across ages and phenotypes. Meanwhile, the SMACR-HI will facilitate tracking of small but clinically meaningful changes in disease burden in pediatric populations. Goals will be achieved through continued collaboration with the Pediatric Neuromuscular Clinical Research Network (PNCRN).

Topic Group 2, Education and Awareness to Promote Broad and Inclusive Research, Treatment, and Care of SMA Patients, seeks to engage individuals and families affected by SMA to understand and identify disparities in SMA care, treatment, and clinical trial research. To facilitate our goal, the topic group will generate a standardized process for demographic data collection that will be implemented within future surveys to capture a range of demographic and socio-economic determinants of health. Additionally, Cure SMA will conduct listening sessions with community members to better understand perceived gaps in SMA management and treatment, and gain insight into the day-to-day experiences of affected individuals and families. Key findings will be leveraged to create future Industry Collaboration activities to encourage equitable access to care and clinical research for all members of the SMA community. Lastly, using data collected by Cure SMA’s annual Community Update Survey, the topic group will develop evidence-based awareness materials to educate stakeholders across the healthcare continuum – including caregivers/families, HCPs, regulators, and payers – about the interplay between copy number and disease status at time of treatment and future outcomes.

As in previous years, Topic Group 3, Cure SMA Patient-Reported Data seeks to provide a forum for informal discussions of developing patient tools to collect patient-reported data to better understand the natural history of SMA, the evolving phenotypes as new therapies are developed, and the continued burden of SMA and changes over time. Since the initial launch in 2017, the Cure SMA Community Update Survey has collected data and information on the SMA community’s experiences and daily challenges. The 6th Annual Community Update Survey will be distributed in spring 2022.  During Phase 7, the topic group will conduct a longitudinal analyses of previously captured Community Update Survey data to illustrate the changing SMA disease trajectory and further educate patients, families, clinicians, and researchers on the changing phenotype. Additionally, the topic group will also compare patient-reported data within the Cure SMA membership database to data collected within the Cure SMA clinical data registry to determine the concordance between patient-reported and clinical data in the SMA population.

Phase 6 Achievements

During Phase 6, the 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 2021 End of Year report. These include:

  • Three published manuscripts
  • Updating Cure SMA Clinical Trial Readiness Program toolkits
  • Development of a competency-based formal education program for SMA physical therapists (STEP-IN SMA)
  • Distribution and analysis of a survey designed to evaluate the unmet medical and supportive needs of individuals affected by SMA during the transition from pediatric to adult care
  • Development of outcome measures designed for teens and adults (SMA-PRO, ATEND, SMACR-HI)
  • Surveying SMA caregivers to assess quality of life within the caregiver community
  • Surveying SMA treatment site providers to assess unmet needs and opportunities to support care
  • Publishing a toolkit in collaboration with SMA Europe on SMA pathology, diagnosis, clinical presentation, therapeutic strategies, and treatment

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, Genentech/Roche Pharmaceuticals, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, and SMA Europe.


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