The EMA recognizes that ATMPs – medicines for human use based on genes, tissues or cells – ‘offer groundbreaking new opportunities for the treatment of disease and injury’. But it also acknowledges the challenges faced by developers in getting new treatments from the lab to patients – including the regulatory complexity.
The pilot will focus on the needs of non-profit academic developers: who are major contributors to the development of ATMPs and diagnostic and delivery devices in Europe but may find navigating regulatory requirements particularly challenging.
The 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.
The EMA will guide the participants through the regulatory process with the wider aim of optimizing development of 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. ARI-0001 is a chimeric antigen receptor (CAR) product based on patients’ own T-cells, that is being 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.
The EMA stresses that no new regulatory tools 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,” says the agency.
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 three to four years.
Interested candidates can contact their national regulatory authority or the EMA via email@example.com to express their interest in participating in the pilot or request more information.