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Sickle Cell Gene Therapy Uptake May Lag, But Still Shock State Medicaid Budgets

Executive Summary

Medicaid state programs, expected to be the majority payers for the new gene therapies, lack certain flexibilities to help them quickly adapt to high-priced treatments, particularly given the huge jump from current sickle cell treatment costs to the just-approved breakthroughs.

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Sickle Cell Gene Therapy In Medicaid: CMMI Model As ‘Bandwidth Extender’ For Less Resourced States

Outcomes-based contract that federal government will negotiate with manufacturers on behalf of Medicaid programs might not be appealing to large states. Bluebird and Vertex can apply to the demonstration project this spring and negotiations would follow between May and November.

Gene Therapy: US FDA Labeling For Vertex’s Casgevy, Bluebird’s Lyfgenia Reflect Different Risks

Agency grants same-day approval to the first two gene therapies for sickle cell disease; bluebird’s lentiviral-based lovo-cel carries a boxed label warning on hematologic malignancies, while Vertex’s CRISPR-Cas9 exa-cel carries a warning and precaution about potential off-target effects. Only the Vertex product qualified for a rare pediatric disease priority review voucher.

Medicaid Proposal Basing Best Price On ‘Stacked’ Discounts At Odds With Law, US Legislators Say

Republicans in Congress also amplify biopharma concerns with provisions in a Medicaid proposed rule that would require cell and gene therapy developers to justify pricing through an agency-conducted audit and that could subject drugs that are bundled with services to Medicaid rebate requirements.

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