Delivering Evidence-Based Access in Rare Diseases: The Challenges in SMA


This session educates about the specific challenges of rare disease treatments in an HTA and discusses how Real-World Evidence Collection can address the data gap at the time of access decision. The three expert speakers will discuss the opportunities, challenges and limitations of Real-World Evidence Collection based on their practical experience in the rare disease area of Spinal Muscular Atrophy (SMA), a severe neuromuscular genetic disorder. In this interactive and moderated session you will hear their ideas how to promote a better coordination in Real-World Evidence Collection and listen to their suggestions how to facilitate its acceptance by Patients, Physicians and Payers.

April 5, 2022 | 9:00AM EDT

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