State Medicaid Agencies Grapple Case By Case With Costly Gene Therapy For MLD

By Maaisha Osman / April 1, 2024 at 3:03 PM
FDA’s approval of a gene therapy treatment for pediatric patients with a rare disease condition called metachromatic leukodystrophy (MLD) -- now crowned as the nation’s most expensive drug -- has placed the burden on state Medicaid agencies to individually navigate value-based contracts with the manufacturer as CMS sidesteps the state payment issue. The manufacturer of the gene therapy Orchard Therapeutics tells Inside Health Policy that it is actively negotiating with multiple states and expects the Medicaid programs to evaluate...


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