OBJECTIVES: Modelling actuarial cashflow of Valrox for Hemophilia A from ICER report, December 22, 2022*.

METHODS: An actuarial value chain model that represents cashflow between 7 health entities (insurer, government, manufacturer, wholesaler, pbm, pharmacy, patient) was built in Microsoft-Excel (2019). The model represents 15 years cashflow associated with the introduction of a new gene therapy treatment and benefits for the target Hemophilia A patient population. Inputs and assumptions were taken from the ICER report* including: Age and gender demographics, prevalence, treatment uptake, success rate, treatment wear-off, productivity cost, treatment cost, alternative treatment cost, adverse events, inflation, quality of life. Deterministic scenario-based sensitivity analyses were performed. Treatment uptake scenarios including: 0%, 50%, 100%, 200% of baseline, lifetime model horizon, net income for each stakeholder and QALY impact of drug pricing strategies (e.g. discounts, rebates, risk sharing) assumed a price elasticity of 0.44.

RESULTS: The model projects cashflow statements for 7 entities monthly for 15 years. Outputs include monthly population counts: Healthy, unhealthy and untreated, successfully treated patients, unsuccessfully treated patients. Gross income, costs, and net income for the 7 entities over 15 years are also projected.

CONCLUSIONS: Over a shorter term (15 years vs. lifetime) the benefits of gene therapy will be overestimated as projected Factor VIII levels decrease over time. More scenarios can be tested based on stakeholder feedback and data availability. By combining actuarial and HEOR modelling, new insights can be gained to inform patient access and drug coverage decisions.

ICER Report*: Tice JA, Walton S, Herce-Hagiwara B, Fahim SM, Moradi A, Sarker J, Chu J, Agboola F, Pearson SD, Rind DM. Gene Therapy for Hemophilia B and An Update on Gene Therapy for Hemophilia A: Effectiveness and Value; Evidence Report. Institute for Clinical and Economic Review, December 22, 2022. https://icer.org/assessment/hemophilia-a-and-b-2022