OBJECTIVES: ATMPs and orphan drugs pose a number of challenges. Their innovative character coupled with their high curative potential are often associated with high costs which renders them challenging therapies for market access, yet they remain vital for patients. Although these therapies gain marketing authorization, negotiating effective Managed Entry Agreements with national reimbursement bodies could be an option to enable patients access to ATMPs and orphan drugs. The objective of this research is to review whether this approach has been adopted by manufacturers to date and to identify the key criteria enabling successful market access of such therapies.

METHODS: Therapies with the ATMPs and orphan drug designations have been extracted from the European Medicines Agency (EMA) website. The websites for HAS (Haute Autorité de Santé, France), AIFA (Italian Medicines Agency, Italy), NICE (National Institute for Health and Care Excellence, UK), IQWiG (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Germany) and MSSSI (Ministerio de Sanidad, Servicios Sociales e Igualdad, Spain) were all accessed in February 2018 to capture the market status and managed entry agreements in place for ATMPs and orphan drugs in the EU5 countries (France, Germany, Italy, Spain, UK).

RESULTS: The EMA approved 45 orphan drugs between 2015 and 2018, amongst which 17 benefited from a managed entry agreement in one of the EU5. The EMA granted ATMP designation to 141 drugs between 2015 and 2018 amongst which only 4 still have a marketing authorization. Managed Entry Agreements are in place for only 3 ATMPs to date in at least one of the EU5.

CONCLUSIONS: The implementation of Managed Entry Agreements for ATMPs and orphan drugs is not widespread in the EU5 countries.