Cell and gene therapies are among the most promising — and most complex — medicines in development. Advanced therapy medicinal products follow a dedicated regulatory path, and access remains a significant hurdle.
Advanced therapy medicinal products (ATMPs) are governed by Regulation (EC) No 1394/2007, which sits alongside the general medicines framework as a lex specialis. They fall into three main categories — gene therapy, somatic cell therapy and tissue-engineered products — plus combined ATMPs incorporating a medical device.
The CAT and centralised authorisation
The Committee for Advanced Therapies (CAT) at the EMA provides scientific classification and prepares draft opinions on ATMP applications. ATMPs follow the mandatory centralised procedure, resulting in a single EU-wide marketing authorisation.
The hospital exemption
Article 28 of the ATMP Regulation allows national authorities to permit a custom-prepared ATMP without a central authorisation, under strict conditions: non-routine preparation, made to prescription for an individual patient, used within the same Member State in a hospital under a doctor’s responsibility, to national quality standards. Divergent national implementation is a recognised issue, and the EU pharma reform proposes tighter harmonisation.
Manufacturing under GMP Part IV
ATMP manufacture follows the dedicated GMP guidelines in EudraLex Volume 4, Part IV — a risk-based framework tailored to small batches, short shelf lives and, for autologous products, starting material derived from the patient.
The access challenge
High one-off costs, uncertain long-term durability data and small patient populations put ATMPs under real strain at the HTA and payer stage. Outcomes-based and managed-entry agreements are common tools. ATMPs are also in scope of the EU Joint Clinical Assessment from January 2025, which may streamline the clinical-assessment step — though pricing and reimbursement remain national, and in Greece are shaped by budget and clawback pressures.
How PQRA helps
PQRA supports ATMP classification, regulatory strategy, and the market-access route through to Greek reimbursement.
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