Advanced therapy medicinal products (ATMPs) are a specific group of biological medicines. They may include genetically modified cells derived from patients (such as CAR-T cell therapy), modified viruses, or complexes of biomolecules that enable precise gene editing. Typically, ATMPs receive a centralized marketing authorization that is valid throughout the European Union.
However, there is an alternative route: an ATMPs can be prepared and used within a single Member State under a hospital exemption. This option is solely under the responsibility of a treating physician and for an individual patient. These products do not go through the standard marketing authorization process, are not marketed commercially, and are known as hospital exemption ATMPs.
Requirements for hospital exemptions were established in Estonia in 2022. Initially, hospital exemption was considered a preliminary stage in the development of an authorized ATMP, and the related rules were relatively strict. However, the situation across Europe has evolved, and hospital exemption has increasingly become a viable route for accessing ATMPs. With the updated rules, Estonia has also made conditions for hospital exemption more flexible.
Key changes include:
- Patient number: Previously, a hospital exemption authorisation was valid for the treatment of up to ten patients Now, there is no upper limit for patient number, provided that the treatment's benefit-risk balance is favorable.
- Duration of the hospital exemption: The validity of the hospital exemption authorization was previously limited to two years, with the possibility of a two-year extension under certain conditions. The initial authorization still lasts for two years but can now be extended for up to five years—and repeatedly—if the benefit-risk profile remains favorable.
These changes aim to improve access to treatment for patients
The State Agency of Medicines issues hospital exemption licenses.
