Need for academic pathway for cell therapy cancer treatment

Subject: Need for academic pathway for cell therapy cancer treatment

Cell therapy is a promising development in the treatment of cancer, specifically for cancers of high unmet need, such as acute myeloid leukaemia (AML), glioblastoma and melanoma. Throughout the EU, cancer organisations are investing in academic research on cell therapy, since commercially developed cell therapy is unlikely to solve every unmet need.

However, despite recent efforts by the European Medicines Agency (EMA) to reach out to academics (e.g. to offer them access to the PRIME scheme for priority medicines), the European authorisation procedure is geared towards commercial players. The certification procedure of the Committee for Advanced Therapies is open to small and medium-sized enterprises, but not to academic centres. Gaining market access for these innovative treatments is difficult, which hampers patient access and is not in line with the goals set out in the pharmaceutical strategy. A non-commercial, academically driven pathway is therefore needed to ensure effective, safe and affordable treatments for all patients.

1. Does the Commission agree that all European patients deserve the best possible cell therapy cancer treatment?

2. How will the Commission develop a specific authorisation procedure accessible to academics?

3. Is the Commission considering regulatory pilots in the ‘sandbox’ to develop pathways for the academic development, manufacture and administration of cell therapy?

Answer given by Ms Kyriakides on behalf of the European Commission

(9 December 2022)

In the EU, a legal framework is in place for advanced therapy medicinal products (ATMPs) (1), including cell and gene therapies for cancer.

This framework stipulates that ATMPs need to obtain an EU marketing authorisation via the centralized procedure, coordinated by the European Medicines Agency (EMA). Up until now 23 ATMPs were authorised, including nine medicines for various types of cancer.

These therapies are however not accessible to patients in all EU countries due to various factors, such as commercial interests of the companies and/or national pricing and reimbursement policies.

The EU pharma legislation (2) provides an exemption to the central marketing authorisation for ATMPs prepared on a non-routine basis according to specific quality standards and used within a given Member State in a hospital (‘hospital exemption’).

Manufacturing of such products is subject to a national authorisation, and Member States need to assure that national requirements for traceability, pharmacovigilance and quality are equivalent to those for centrally authorised products.

In the Pharmaceutical Strategy for Europe, the Commission committed to support research with particular attention to academia and not-for-profit organisations aiming at better translation of research into product development.

In this regard, the Commission is rolling out initiatives to understand the specific needs of academic developers of ATMPs, also considering as necessary a possible tailored pathway for academic development of ATMPs.

In the context of the revision of the pharmaceutical legislation, the Commission is also considering a sandbox provision in order to allow regulatory pilots for new cutting-edge product development.

⋅1∙ Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending

Directive 2001/83/EC and Regulation (EC) No 726/2004 (Text with EEA relevance). ⋅2∙ Under Article 3 (7) of Directive 2001/83/EC.