Navigating Undefined Parameters for SMA Treatment Coverage

EP: 1.SHINE Study: Illuminating the Promise of Nusinersen in SMA Treatment

EP: 2.Examining Patient Characteristics from SHINE Study

EP: 3.Highlighting Key Data from the CHERISH and SHINE Studies

EP: 4.Examining Key Findings from NURTURE Study

EP: 5.Relevance of NUTURE Study Findings for Patients, Payers, Providers

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EP: 6.Navigating Undefined Parameters for SMA Treatment Coverage

This is a video synopsis/summary of a Between the Lines featuring Thomas Crawford, M.D.; and Jim Kenney, R.Ph., MBA.

Crawford and Kenney further discuss potential solutions to balance innovation, access and costs for rare disease therapies. Outcomes-based agreements between manufacturers and payers could help link costs to real-world benefits. However, consistent measurement tools valid across patient populations are lacking.

Crawford suggests developing biomarkers and scientific methods to demonstrate molecular changes supporting therapy decisions rather than waiting years to assess functional impacts. Regulatory and economic models adjusting pricing to level of benefit may also help balance value (e.g., dramatically extending young lives versus slowing decline in adults).

In diseases like SMA where maximal gains plateau early, managing expectations around ongoing improvement versus stabilization is an added complexity. Still, any slowing in decline could represent a breakthrough. Resolving these tensions in an equitable and sustainable way remains a pressing challenge requiring ongoing collaboration among stakeholders.

Video synopsis is AI-generated and reviewed by Managed Healthcare Executive® editorial staff.