PHP307 - REIMBURSEMENT OF ADVANCED THERAPY MEDICINAL PRODUCTS IN EUROPE

Abstract

Advanced Therapy Medicinal Products (ATMPs) are innovative therapies including gene, cell therapies, and tissue-engineered products. These therapies hold promises to cure diseases with high clinical unmet needs. However, ATMPs are facing critical hurdles in accessing the market. The study objective was to review ATMP assessments by Health Technology Assessment (HTA) bodies in the 5 big European Countries (EU5) to understand main limitations for securing adoption. A search was conducted on 01/06/2018 in European Medicines Agency (EMA) website to identify ATMPs approved in Europe. Available ATMPs HTA reports were extracted from EU5 HTA websites and were reviewed. Ten ATMPs were approved in Europe: Chondrocelect®, Glybera®, MACI®, Provenge®, Holoclar®, Imlygic®, Strimvelis®, Zalmoxis®, Spherox® and Alofisel®. For the first four ATMPs, marketing authorizations were withdrawn due to commercial reasons. Four ATMPs were reimbursed in United Kingdom (Imlygic®, Strimvelis®, Holoclar®, Spherox®) with patient access schemes, only Provenge® was not recommended due to high uncertainty. In Italy, three ATMPs were reimbursed for hospital use with managed entry agreements (Strimvelis®, Holoclar®, Zalmoxis®). Similarly in Germany, these three ATMPs were also reimbursed, while, Glybera® and Provenge® had “non-quantifiable added benefit” due to insufficient data and Imlygic® had “no-added benefit” due to inappropriate comparator use. French health authority recommended only Holoclar® with restrictions and did not recommend Glybera® and Chondrocelect® due to insufficient efficacy evidence. No HTA reports were available in Spain. Uncertainty on efficacy due to lack of robust and long-term evidence was the main limitation in securing reimbursement. Restriction of population was sometimes used to mitigate the uncertainty. European regulators tend to accelerate the approval of ATMPs while HTA bodies are reluctant to reimburse ATMPs with immature data and high prices. Parallel advice may help harmonize HTA and regulators’ perspectives and provides manufacturers with recommendations to reduce the uncertainties to ultimately achieve market access for ATMPs.