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

By Maaisha Osman / April 1, 2024 at 3:02 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.


Not a subscriber? Sign up for 30 days free access to exclusive, detailed reporting on drug pricing reforms, Medicaid policy, FDA news and much more.