A panel of experts discuss improving collaboration between health systems and managed care organizations to ensure optimal outcomes for patients with rare diseases.
Jessica Nance, MD, MS, and Emma Ciafaloni, MD, FAAN, discuss ethical dilemmas, exploring how patient subgroup restrictions on orphan drugs can impact access and cost control.
Ryan Haumschild, PharmD, MS, MBA, leads a discussion on factors that influence coverage of non-oncology gene therapies, exploring the balance between long-term cost savings and high initial prices, patient selection, and the durability of effect for these therapies.
Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early intervention and comprehensive care.
Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease.