Optimizing Multidisciplinary Care Approaches for the Future of SMA Management

EP: 1.Describing SMA Progression Over Time

EP: 2.Unmet Needs of Older Patients Living With SMA

EP: 3.Examining the Importance of Early Treatment for SMA

EP: 4.Key Functional Milestones Affected by Disease Progression

EP: 5.The Importance of Supportive Care for Patients Living With SMA

EP: 6.Introduction to Therapies Used to Treat SMA

EP: 7.Splicing Modifiers and Gene Therapy for SMA

EP: 8.Key Clinical Considerations for the Management of SMA

EP: 9.Assessing Different Factors When Selecting a Treatment

EP: 10.Evaluating the Potential of Intrathecal Gene Therapy

EP: 11.Other Investigational Treatment Options for SMA

EP: 12.Insurance Considerations for SMA Therapies

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EP: 13.Optimizing Multidisciplinary Care Approaches for the Future of SMA Management

EP: 14.Final Panel Thoughts on the Management of SMA

Clinical Brief: Stakeholder Collaboration for Equitable SMA Treatment Access

Main Discussion Topics

• Need for collaboration between clinicians, payers, and patient advocates
• Psychosocial impact of performance-based treatment authorization
• Potential approaches to facilitate more equitable treatment access
• Communication strategies to address coverage uncertainties

Key Points for Physicians

• Direct communication between clinicians and payers can improve mutual understanding of treatment value.
• Performance-based authorization creates significant anxiety for patients during assessments.
• State Medicaid engagement has proven beneficial in developing reasonable policies for patients with SMA.
• Multiple stakeholder perspectives are needed to develop balanced approaches to treatment access.

Notable Insights

The panel described the psychological burden on patients when assessments determine treatment continuation, likening it to "going to the Olympics every time" with the pressure to perform well enough to maintain therapy access.

Clinical Significance

Collaborative approaches involving clinicians, payers, and patient advocates can lead to more equitable and sustainable access to SMA treatments while acknowledging resource limitations and the need for evidence-based decision-making.