OBJECTIVES:

Although an increasing number of ATMPs are entering the market, the driving factors for successful commercialisation are unclear. This research aims to evaluate the eight ATMPs approved by the European Medicines Agency (EMA) to date and identify key success factors and barriers that impact market access of ATMPs in Europe.

METHODS:

We analysed health technology assessment (HTA) reports to understand reimbursement decisions of ATMPs in France, Italy, Germany, and the UK. We reviewed academic and news articles for updates and interviewed payers where clarification was needed.

RESULTS:

Eight ATMPs have been approved by the EMA. The first four ATMPs, ChondroCelect, Glybera, Provenge and MACI, have already withdrawn from market due to commercial reasons. One key hurdle was country-level HTA, as the clinical benefits of the four ATMPS were not recognised despite being innovative technologies. Without recognition of the clinical value, it is challenging to justify the high price tags (e.g. Glybera received a non-quantifiable added clinical benefit rating in Germany, and at approximately €1 million per patient, its uptake has been low).

In contrast, the four ATMPs approved by the EMA more recently (between 2015-2016) show more promising futures. The clinical benefits of Holoclar, Imlygic and Strimvelis have been recognised and reimbursement granted in at least one country in Europe. One key contributing success factor was the use of managed entry agreements to mitigate financial/outcome risks for payers and better support value for money. Other factors include the adoption of a private-public partnership model to effectively engage different stakeholders and the abilities to build infrastructure necessary for delivery of ATMPs.

CONCLUSIONS:

ATMPs approved by the EMA in the last two years have been more successful at gaining reimbursement in Europe. Success is driven by a combination of demonstrating clinical benefits and addressing payer clinical/financial concerns, as well as maturity in environment/infrastructure.