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

MedicalResearch.com 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

MedicalResearch.com: 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.

MedicalResearch.com: 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.

MedicalResearch.com: 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.

MedicalResearch.com: 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.

MedicalResearch.com: 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

Citation:

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

Mar 22, 2021 @ 5:51 pm

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