Dr. Julie Kanter, director of the Adult Sickle Clinic at the University of Alabama at Birmingham, recognizes Michael Goodwin’s reluctance to pursue gene therapy for sickle cell, a sentiment shared by many patients. Despite the approval of these therapies in December, Kanter estimates that only 10% of those affected would opt for the treatment even if it were universally accessible. With over 100,000 Americans having sickle cell disease, of whom 50-60% are covered by Medicaid, challenges in expanding treatment capacity and establishing facilities across the country persist.
As state and federal officials navigate the high costs of these therapies for Medicaid-covered patients, a University of Washington analysis suggests a lower price, around $1 million, would enhance accessibility. The Biden administration is initiating negotiations with Vertex and Bluebird Bio for discounts on state Medicaid plans, integrating payments with patient health outcomes through the Cell and Gene Therapy Access Model.
Despite ongoing lawsuits from pharmaceutical giants against the Biden administration over Medicare price negotiations, Vertex expresses confidence in the negotiation process. Discussions aim to address inequities in sickle cell care and ensure wide access.
Private employer health plans also grapple with covering these high-priced treatments, potentially leading to exclusions from benefit packages. Morgan Health explores risk-sharing payment models to aid smaller businesses in managing rising treatment costs. For individuals like Goodwin, covered by his wife’s employer health insurance, the decision to pursue gene therapy hinges on its guarantee of a favorable outcome.
Amidst discussions over payments, Vertex and Bluebird Bio actively engage in educating doctors and patient communities about the advantages of their therapies. Vertex anticipates its first commercial patient starting treatment soon, while Bluebird Bio expects its first Lyfgenia patient in the first quarter.
