The Multiple Criteria Qualitative Value-Based Pricing Framework "MARIE" for Novel Cell and Gene Therapy.

When setting the price of cell and gene therapies in Japan, the factors considered include the manufacturing and operating costs of the new drug or a comparator, leading to discrepancies between price and values, in particular those specific to cell and gene therapies. Here, we adapted our previously proposed value-based pricing framework "MARIE" for use in estimating value-based prices for cell and gene therapies. We adapted "MARIE" for use in pricing cell and gene therapies by adding "curative treatment" and "gene therapy" as value elements. The adapted framework determines prices of cell and gene therapies by multiplying the hypothetical daily price (determined from a drug price conversion table) and the efficacy period (rounded down to the nearest 6 months). We collected information, including on value elements, for new cell and gene therapies approved between 2015 and 2024 (n = 11) and compared estimated prices with actual list prices at the time of initial listing in the National Health Insurance Drug Price Standard. Overall, estimated prices tended to be similar to or higher than the list price. Estimated prices were 91.7% to 546.7% of list prices. We adapted our VBP framework "MARIE" for cell and gene therapies by incorporating elements specific to these therapies and estimated the prices using the information available at listing. Because certain factors, especially treatment duration, are uncertain at the time of initial listing, a system is needed that reevaluates and recalculates prices as additional evidence becomes available.