OBJECTIVES: Advanced therapy medicinal products (ATMPs) are kind of experimental treatments in which genes, cells, or tissue-engineered products are used to treat diseases. In the last two decades, enormous progress has been made in this research area. The current review was conducted to understand the existing trends, challenges and to provide future directions for ATMPs in Europe.

METHODS: European regulatory website (EMA), HTA bodies (NICE, SMC, IqWIG, HAS and AIFA) and clinical trial websites (clinicaltrials.gov and EudraCT) were reviewed. A grey literature search was conducted to understand the challenges faced during marketing authorization and launch and identify the potential solutions.

RESULTS: Even though ~800 ATMPs, including ~50% in oncology, are currently in-development in Europe, only 10 ATMPs have yet been approved by EMA. This low rate of regulatory approvals can be attributed to quality or scale-up aspects, non-clinical aspects and absence of long-term efficacy and safety data.

Additionally, evidence from HTA bodies suggested that six ATMPs have been approved by NICE, three each by HAS, IqWIG and AIFA, and one by SMC. The reason of encountering substantial hurdles in market access and reimbursements can be smaller target population, high costs, lack of long-term efficacy and safety data and complexities associated with administration, dosing and monitoring.

These challenges are likely to intensify with marketing of more ATMPs and expansion to cover wider population. To overcome these challenges, it is recommended that manufacturers’ should collect long-term safety and efficacy data and consider new clinical trial methodologies, while payers should refine/restructure value demonstration and pricing strategies.

CONCLUSIONS: Higher efficacy, increasing approvals and "one-time treatment" feature of ATMPs clearly demonstrate the shifting paradigm of healthcare. However, uncertainty of launch approval can augment burden on payers during decision-making. Thus, ATMPs may be the next source of major impact on payers' drug budgets.