The potential effects on pricing and accessibility of a new gene therapy for Parkinson's diseases, using the cost-effectiveness and budget impact methodologies utilised by NICE and ICER

The thresholds differ greatly between the cost-utility analyses (CUA) and budget impact analyses (BIA) used by NICE in England, and ICER in the US, to assess the value for money and affordability. This project aimed to determine the implications for reimbursed price and volumes for a novel gene therapy for Parkinsons’s disease (PD) following a cross-country comparison of the results of the CUA and BIA.

Country-specific CUAs and BIAs were performed using a Markov model. Aligning with previous analysis comparing US and UK prices for high-cost drugs, the CUA identified that the US ceiling price is ~ 1.8 times greater than that of England. However, setting prices at these levels would result in a net budget impact that would restrict the number of patients who could receive treatment, so as not to surpass the threshold for BIA. To increase patient access without surpassing these thresholds and decreasing payers' uncertainty, performance-based annuity payments can be implemented at launch.

This analysis of the cost-utility of PD treatment indicates a price discrepancy between the US and England that is consistent with the pricing of other high-cost drugs. Additionally, the budget impact threshold operational in England exerts a stronger influence on pricing and/or patient volumes than the corresponding threshold utilized by ICER in the US.

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