A Spotlight on Haemophilia Therapies
Cell and Gene Therapies
Cell and gene therapies offer substantial therapeutic benefits but are expensive and often have a limited evidence base at launch. There are concerns over affordability and long-term cost-effectiveness, as well as uncertainty as to whether their value is accurately captured by current HTA methods.
Early Experience with Health Technology Assessment of Gene Therapies in the United States: Pricing and Paying for Cures
