Innovating to generate value: managing the Evrysdi(R) drug in the Marche Region

The case study examines the introduction of Evrysdi(R), a drug for treating Spinal Muscular Atrophy (SMA), in the Marche Region of Italy. It explores the governance model, decision-making processes, and operational procedures involved in managing this therapeutic innovation within the public healthcare system. The study highlights the collaboration among regional stakeholders, including clinicians, pharmacists, and policymakers, in managing Evrysdi(R) through designated prescribing centers and the centralized preparation at Carlo Urbani Facility Hospital in Jesi. The case also discusses challenges, such as transitioning from compassionate use to routine care, managing patient expectations, and safeguarding the cold chain during drug delivery. Finally, the study addresses future implications, including the impact of regional healthcare reorganization and the ongoing development of a Diagnostic-Therapeutic Care Pathway for SMA, emphasizing the importance of coordination and patient-centered care in rare disease management.