Important takeaways for patients, payers, and providers are covered by expert faculty.
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 discuss the implications of spinal muscular atrophy therapy studies for patients, providers, and payers. While transformational for a previously fatal pediatric disease, the extremely high costs of these innovative therapies (e.g.,nusinersen costs $125,000 per dose) raises societal questions about equitable access and sustainability.
As more gene and cell therapies emerge for other rare diseases with the potential for significant benefits, balancing innovation incentives and cost management across the healthcare ecosystem will be an ongoing challenge. Payers must rapidly get up to speed on unfamiliar diseases and make coverage decisions with limited long-term data.
Key next steps include continued education and collaboration among all stakeholders — patients, providers, payersand policymakers — will be needed to ensure patient access while supporting further progress.
Video synopsis is AI-generated and reviewed by Managed Healthcare Executive® editorial staff.