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.