White Paper: Estimating the PMPM of Gene Therapies for Sickle Cell Disease in a National All-Payer Risk Pool


 

AMCP White Paper - Gene Therapy Sickle Cell

Cell & Gene Therapies (CGT) offer potentially curative treatment to patients with chronic and hereditary conditions but carry high price tags, sometimes as high as $3 million per patient. This paper focuses on two of the financial barriers most relevant to payers: the concentration of cost in a single upfront event and the mismatch between who pays and who benefits over time. One proposal put forth to overcome both challenges is to create a national risk pool. To further explore this idea, AMCP conducted preliminary analyses to estimate the PMPM costs of two gene therapies for sickle cell disease under a mandatory all-payer national risk pool model.

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