Advanced Therapy Medicinal Products (ATMPs) face the same regulatory and HTA challenges as every new medicinal product, but exaggerated due to their specific characteristics and limitations. Often there is a high upfront cost per patient based on data from a small number of patients. This increases the uncertainties for cost/effectiveness calculations. For ATMPs, de-risking the development programme is essential, and thus an integrated product development strategy is recommended in order to meet both regulatory and HTA requirements. Developers should also seek early dialogue with regulatory and relevant HTA bodies to discuss data requirements, possible new payment models and thus ensure successful licensing and market access.
