Patrick DeMartino MD Pediatric Hematology and Oncology Fellow Doernbecher Children's Hospital Oregon Health & Science University

Sickle Cell Anemia: Paying For Gene Therapy Presents Unique Challenges for Medicaid Interview with:

Patrick DeMartino MD Pediatric Hematology and Oncology Fellow Doernbecher Children's Hospital Oregon Health & Science University

Dr. DeMartino

Patrick DeMartino MD
Pediatric Hematology and Oncology Fellow
Doernbecher Children’s Hospital
Oregon Health & Science University What is the background for this study?

Response: Dozens of gene therapies are expected to be on the market within a decade or so. Much has been written about the high prices of the therapies currently on the market (exceeding $1 million). However, only a small number of patients are eligible for these existing therapies each year. Gene therapy for sickle cell disease (SCD) appears promising and would potentially apply to a relatively large number of individuals in the U.S. We sought to explore potential affordability challenges associated with a gene therapy for SCD. What are the main findings?

Response: Our model focused upon Medicaid plans with a high prevalence of SCD among enrollees. We project the gene therapy would have a considerable budget impact for these health plans (~$1.90 per member per month increase in spending). An alternative payment models (annuity payment) would help reduce the short-term impact. What should readers take away from your report?

Response: Gene therapy presents unique challenges for payers given the high upfront cost and long-term uncertainty of benefit. When priced over $1 million it does not take much utilization to create affordability challenges for payers who need to balance their short-term budgets. New methods for financing these curative therapies may be needed. Might the same challenges to state programs occur with other potential gene therapies (i.e. for inherited blindness)? 

Response: While the price for any gene therapy may be impressive, the prevalence of the disease among health plan members is more consequential when considering affordability for a given payer. For many Medicaid plans, the introduction of direct acting antivirals (DAA) for Hep C created affordability challenges and their price was trivial compared to typical gene therapy but many more patients were eligible for DAAs. Would some patients consider giving up employment or moving in order to receive state Medicaid coverage? 

Response: Unfortunately we already have a system in which individuals are forced to make personal or professional decisions based a need to obtain affordable and comprehensive health insurance. It is conceivable that differential coverage of a gene therapy across Medicaid plans could incentivize individuals to move in pursuit of a therapy. 

I have no disclosures


DeMartino P, Haag MB, Hersh AR, Caughey AB, Roth JA. A Budget Impact Analysis of Gene Therapy for Sickle Cell DiseaseThe Medicaid PerspectiveJAMA Pediatr. Published online March 22, 2021. doi:10.1001/jamapediatrics.2020.7140

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Last Updated on March 22, 2021 by Marie Benz MD FAAD