Gene Therapy

The researchers focused on coverage trends across an array of disease states, including lymphoma, vision loss, and spinal muscular atrophy, finding discrepancies in coverage across 16 states and Medicaid Care Organizations.

The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.

Many gene therapies promise life-changing effects, but without long-term data it remains to be seen how long the benefits last.

Experts evaluated the future of cell and gene therapies in the United States and offered insights on potential value-based payment models for these treatments.

With more than 3000 gene therapies in development, payers will have to grapple with the challenges of paying for these innovative but expensive therapies.