Evidence-Based Strategies to Improve Access to Therapies for Patients with Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is both a rare disease and the most common fatal genetic disease of infancy. The approval of the first disease-modifying therapy (DMT) for SMA in 2016 revolutionized management, and treatment options have expanded with two additional DMTs approved more recently. However, because SMA is a rare disease, many clinicians and payers have limited knowledge of SMA and the available DMTs, which can contribute to delays in diagnosis and treatment initiation. Evidence-based managed care decision-making is critical in order to enable earlier intervention and optimize patient outcomes in SMA.

In this CMEOCast podcast, expert faculty will help clinicians and payers identify the genetic and clinical assessments used to diagnose SMA and monitor patients over time, apply the latest evidence for efficacy, safety, and cost-effectiveness of SMA therapies, and recognize the importance of the individual patient- and parent/caregiver-related factors in guiding clinical decision-making when managing patients with SMA.

- Learning Objectives -

At the end of this CE activity, participants should be able to:
- Identify the genetic and clinical assessments for diagnosing SMA and outcome measures for monitoring disease activity and treatment response.
- Apply the latest clinical evidence to evaluate the efficacy, safety, and long-term cost-effectiveness of approved and emerging therapies for SMA.
- Recognize the individual patient- and parent/caregiver-related factors that guide clinical decision-making in the management of SMA.

The following learning objectives pertain only to those requesting CNE or CPE credit:
- Summarize the genetic and clinical assessments for diagnosing SMA and outcome measures for monitoring disease activity and treatment response.
- Describe the latest clinical evidence to evaluate the efficacy, safety, and long-term cost-effectiveness of approved and emerging therapies for SMA.
- Recognize the individual patient- and parent/caregiver-related factors that guide clinical decision-making in the management of SMA.