Advanced therapy medicinal products (ATMPs) — gene therapies, somatic-cell therapies and tissue-engineered products — are among the most complex products to bring to the European market. Governed by Regulation (EC) No 1394/2007, they follow a centralised route with a dedicated committee, specialised requirements and a narrow national exemption that is often misunderstood.
What is an ATMP?
Regulation (EC) No 1394/2007 defines three categories of ATMP: gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products. A fourth type, combined ATMPs, incorporates one or more medical devices. Because the boundaries between these categories — and with other product types — can be blurred, the classification of a product is itself a formal regulatory step.
The Committee for Advanced Therapies
The Regulation established the Committee for Advanced Therapies (CAT) at the EMA. The CAT is the centre of expertise for ATMPs and:
- issues scientific recommendations on whether a product qualifies as an ATMP (ATMP classification);
- prepares a draft opinion on the quality, safety and efficacy of each ATMP for the CHMP;
- advises on borderline and combined products.
Because ATMPs must be authorised through the centralised procedure, a single marketing authorisation valid across the EU is the only route to market — there is no national or mutual-recognition alternative.
The hospital exemption
Article 28 of the Regulation created the so-called hospital exemption. It allows an ATMP to be prepared and used for an individual patient, on a non-routine basis, under the responsibility of a physician, within a single member state and without a full marketing authorisation. The exemption is implemented nationally, so requirements differ considerably between member states, and it is not a substitute for a development programme aimed at EU-wide authorisation. Sponsors relying on it need to understand exactly how their target market applies it.
Practical development considerations
- Classification and early scientific advice should be sought before the pivotal programme is designed.
- ATMPs benefit from specific regulatory support, including certification of quality and non-clinical data for SMEs and access to the PRIME scheme for eligible products.
- Manufacturing and comparability are frequently the critical path; GMP for ATMPs has its own dedicated guidelines.
- Long-term follow-up and robust risk-management planning are expected given the durable nature of many of these therapies.
How PQRA helps
PQRA guides developers of advanced therapies through the EU framework — from ATMP classification and scientific advice, through centralised marketing-authorisation strategy, to the quality, pharmacovigilance and risk-management requirements these products demand. We help you judge when the hospital exemption is relevant and when a full authorisation is the only viable route.
Contact PQRA to discuss your advanced therapy programme.
No responses yet