The article reviews international experience in market access, health technology assessment (HTA), and reimbursement models for advanced therapy medicinal products (ATMPs). This diverse group of therapies, including gene, cell, and tissue-engineering products, represents some of the most innovative medicines, but at the same time poses significant challenges to regulatory, health economic, and financial systems.
Access to ATMPs is shaped by the interaction between three main systems: regulatory approval, health technology assessment, and payment or reimbursement mechanisms. The specificities of these therapies – high clinical value but often limited evidence base, small number of patients in clinical trials, and use of surrogate endpoints – complicate the evaluation process. In addition, the cost of the therapies is very high and the expenditure is usually concentrated in a short period, as it is often a single treatment.
The article analyzes various regulatory mechanisms aimed at accelerating access to innovative therapies for serious diseases and unmet medical needs. These include accelerated assessment procedures, conditional use authorisations, early access programmes, and special regulatory statuses that support the development of new therapies. These tools allow earlier patient access to treatment, while requiring additional clinical data generation after the product is authorised.
Special attention is paid to practices in the European Union, where the European Medicines Agency uses mechanisms such as accelerated assessment and conditional marketing authorisation. Despite the reduction in the time to regulatory decision, access to therapies remains highly dependent on subsequent national decisions on pricing, reimbursement, and organisational requirements for implementation.
In conclusion, it is highlighted that the successful implementation of ATMPs requires a coordinated approach between regulators, HTA authorities, and payers, as well as the development of new payment models and uncertainty management that ensure sustainable patient access to these high-tech therapies.
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