EMA is launching a pilot to support the translation of basic research developments into medicines that could make a difference in patients’ lives in the European Economic Area (EEA). The pilot is open to academic sponsors and non-profit organisations who are developing advanced therapy medicinal products (ATMPs). These medicines for human use are based on genes, tissues or cells and might offer ground-breaking treatment options to patients.
The pilot will focus on the needs of non-profit academic developers. They are a major contributor to the development of ATMPs and diagnostic and delivery devices, but experience has shown that navigating regulatory requirements can be challenging.
During the pilot, EMA will provide enhanced regulatory support for up to five selected ATMPs that address unmet clinical needs and are solely developed by academic and non-profit developers in Europe. EMA will guide the participants through the regulatory process with the aim to optimise the development of the ATMPs, starting from best practice principles for manufacturing to planning clinical development that meets regulatory standards.
The pilot’s first participant has already been selected. This ATMP is ARI-0001, a chimeric antigen receptor (CAR) product based on patients’ own T-cells, that is developed by the Hospital Clínic Barcelona. In December 2021, the product was granted eligibility to PRIME, EMA’s scheme to support the development of medicines that target an unmet medical need.
Importantly, no new regulatory tool will be introduced as part of this pilot. However, the aim is to assess what further support or regulatory tool may be provided to enhance the number of ATMPs reaching patients in the EEA. In the process, EMA is keen to learn how to better interact with and support academic developers.
The pilot participants will benefit from all the available regulatory flexibilities and development support measures, such as fee reductions and waivers. The progress will be closely monitored, and initial results of the pilot are expected to be available in 3-4 years. Upon completion, a report will be published and a workshop with relevant stakeholders may be organised to discuss the learnings.
Potential developer candidates can contact their national competent authority or EMA via advancedtherapies@ema.europa.eu to express their interest in participating in the pilot or to receive more information.
