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High-cost sickle cell gene therapies push insurers and Medicaid programs to find new payment models
BLUEbluebird bio(BLUE) CNBC·2025-02-18 14:52

Core Insights - The article discusses the challenges and advancements in the treatment of sickle cell disease, particularly focusing on new gene therapies approved by the FDA, such as Casgevy by Vertex Pharmaceuticals and Lyfgenia by Bluebird Bio [2][6][7]. Treatment and Patient Access - DJ Chow, a 19-year-old patient, has benefited from the new gene therapy, which costs over 2millionperpatient,highlightingthefinancialburdenassociatedwiththesetreatments[2][3].−Thetreatmentprocessforsicklecellgenetherapiesinvolvesmultiplehospitalizationsandchemotherapy,indicatingacomplexandcostlytreatmentregimen[2][6].−Asoftheendof2024,onlyabout100patientshaveundergonetreatmentwiththenewtherapies,reflectingaslowramp−upinpatientaccess[6][7].InsuranceandPaymentModels−Thecoordinationbetweentreatmentcentersandinsurershasimproved,butthehighcostsoftherapiesremainasignificanthurdleforcoverage[9][10].−CVSHealth′sCEOnotedthatwhilethecurrentdemandismanageable,theindustryisexploringnewpaymentmodelstoaccommodatetheexpectedincreaseinpatientsneedingtreatment[10][11].−TheBidenadministrationhasintroducedaCellandGeneTherapypaymentmodeltoassiststateswithfundingfortheseexpensivetreatments,withadeadlineforapplicationssetforFebruary28[13][14].BudgetImplicationsforStates−Stateswithhighconcentrationsofsicklecellpatientsmayfacesignificantbudgetimpacts,withestimatessuggestingameanbudgetimpactof2 million per patient, highlighting the financial burden associated with these treatments [2][3]. - The treatment process for sickle cell gene therapies involves multiple hospitalizations and chemotherapy, indicating a complex and costly treatment regimen [2][6]. - As of the end of 2024, only about 100 patients have undergone treatment with the new therapies, reflecting a slow ramp-up in patient access [6][7]. Insurance and Payment Models - The coordination between treatment centers and insurers has improved, but the high costs of therapies remain a significant hurdle for coverage [9][10]. - CVS Health's CEO noted that while the current demand is manageable, the industry is exploring new payment models to accommodate the expected increase in patients needing treatment [10][11]. - The Biden administration has introduced a Cell and Gene Therapy payment model to assist states with funding for these expensive treatments, with a deadline for applications set for February 28 [13][14]. Budget Implications for States - States with high concentrations of sickle cell patients may face significant budget impacts, with estimates suggesting a mean budget impact of 30 million for the ten states with the largest sickle cell populations [14]. - The financial challenges for Medicaid programs are particularly acute, as over half of sickle cell patients are covered under this federal-state health plan [11][12]. Future Outlook - There is hope that as more patients receive therapy, the costs may decrease, allowing broader access to these treatments [18]. - The ongoing research and development of gene therapies are seen as critical for improving the quality of life for patients suffering from sickle cell disease [17].