OBJECTIVES : ATMPs are unique, defined by the European Medicines Agency’s (EMA) as Somatic cell therapy, Tissue engineered or Gene therapy medicinal products. Pre- and post-marketing development is complex, with additional considerations given to manufacture, labelling, efficacy, safety and traceability. This is an overview of ATMP availability within Europe.

METHODS : A list of authorised ATMPs was obtained from the EMA central evaluation for a European Public Assessment Report (EPAR). HTA agency websites for Great Britain, France and Germany were then searched to verify the current ATMP market access status.

RESULTS : Almost three hundred innovative ATMPs are under development within the EU. EPAR status is currently available for sixteen, with a total of eleven authorised for EU use. Four further products have been withdrawn, three at the request of the marketing authorisation holder. One ATMP was refused EU authorisation as benefit of treatment was not deemed sufficiently demonstrated. The National Institute for Health and Care Excellence (NICE) currently recommend seven ATMPs with restrictions; two reference a Patient Access Scheme (PAS) and three within the Cancer Drugs Fund. The Scottish Medicines Consortium report two ATMPs as accepted for use, one of which takes account of PAS. In France, SMR deemed five ATMPs ‘important’, while ASMR was viewed as ‘minor’ or ‘moderate’, with only one product listed with reimbursement (65%). As many ATMPs are orphan drugs, Germany reports a benefit decision for Imlygic only as ‘no additional benefit’. Reasons for the decisions made include absence of long-term benefit, safety or tolerance data, a lack of direct comparison with limited therapeutic options, small patient numbers and insufficient treatment cost in relation to health benefit.

CONCLUSIONS : Despite consideration to support and accelerate ATMP authorisation, patient access remains a challenge. The EMA suggest early HTA and developer engagement to address added therapeutic value and reimbursement.