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An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom.

Pablo E Bretos-AzconaMatthew WallaceMurvin JootunGuanyi JinIon AgirrezabalAgota Szende
Published in: Clinical drug investigation (2024)
Despite the lack of a strong evidence base in MMA and PA, this model provides a useful tool to estimate the cost-effectiveness, and inform value-based pricing, of new mRNA-based therapies. Our analyses also identified areas for research that will have the greatest value in reducing uncertainty in future health economic evaluations of such treatments.
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