OBJECTIVES: To determine the number of Advanced Therapy Medicinal Products (ATMPs) either recently approved or likely to be approved in Europe until 2024 and to estimate the number of patients that could be eligible for treatment with each product in the EU27+UK.

METHODS: We reviewed ATMPs clinical trials registered in EudraCT database and systematically selected products approved or likely to be approved in Europe between 2020 and 2024. Subsequently, we estimated the size of the target population for each product. We used publicly available data sources to estimate the probability of the launch of each product and the epidemiology of each condition.

RESULTS: Our study shows that in an ideal scenario where every diagnosed patient was to be treated with one of the ATMPs recently approved or with potential to be approved until 2024, about 20 million people in the EU27+UK would have been treated by 2030. In a hypothetical scenario where the ATMPs will penetrate the markets with estimated product-specific market shares, the number of potentially treated patients is reduced by half.

CONCLUSIONS: The large pipeline of ATMPs suggests that many patients could theoretically benefit from the treatment with these innovative therapies. However, the uncertainty on the value, high cost, and one-shot nature of many ATMPs challenge the classical pricing and reimbursement (P&R) process. Variations in the ATMP therapies reimbursement between countries, limited eligibility for extra funds for managing ATMPs in hospitals, and commercially driven withdrawals of some ATMPs fuel uncertainties in ATMP access in Europe. This study confirms the concerns raised in the recent years about the affordability of these therapies for European healthcare systems, but more importantly, highlights the urgent need for the implementation of adequate infrastructure and payment schemes adapted to the unique value offered by this new type of medicines.