[PHP272] Advanced Therapies: Widening The Gap Between Payers And Regulators

[PHP272] Advanced Therapies: Widening The Gap Between Payers And Regulators

2016 Value in Health

Hanna, E. | Rémuzat, C. | Auquier, P. | Toumi, M. | Volume: 19, Issue: 7, Pages: A487,

OBJECTIVES: Advanced Therapy Medicinal Products (ATMPs) are innovative therapies including gene therapies, cell therapies, and tissue engineered products. These therapies are expected to halt or cure many chronic, disabling diseases. While European regulators tend to speed market access of such therapies through accelerated pathways (authorisation under exceptional circumstances, conditional marketing authorisation, accelerated assessment, adaptive pathways and PRIME), health technology assessment (HTA) bodies/payers are increasingly scrutinising the incremental value of these products. The study objective was to identify potential gap in the evaluation of ATMPs between payers and regulators in Europe. METHODS: A search was conducted in European Medicines Agency (EMA) website to identify ATMPs approved in Europe; HTA assessment of these ATMPs was reviewed for France, Germany and United Kingdom through HAS, IQWiG/G-BA, NICE and SMC websites. RESULTS: Seven ATMPs received a marketing authorisation (MA) in Europe until June 2016: Chondrocelect® (2009), Glybera® (2012), MACI® (2013)-MA suspended in 2014, Provenge® (2013)- MA withdrawn in 2015, Holoclar® (2015), Imlygic® (December 2015), Strimvelis® (May 2016). None of these ATMPs has been recommended for reimbursement by HTA bodies in the study scope. Only Chondrocelect® is reimbursed on a case-by-case basis in Germany after negotiation between hospital and appropriate regional health insurance. HAS did not recommend Chondrocelect® and Glybera® due to insufficient actual benefit. NICE considered that Provenge® was not cost-effective and did not meet the criteria for end-of-life consideration. Glybera® and Provenge® were rated as “non-quantifiable added benefit” by IQWiG/G-BA. Holoclar®, Imlygic® and Strimvelis® have not yet been assessed. CONCLUSIONS: EMA is accelerating the regulatory pathways for innovative products whereas HTA bodies tend not to recommend ATMPs for reimbursement mainly because of immature data. Parallel advice may help harmonizing HTA and regulators’ perspectives and provide manufacturers recommendations to achieve market access for ATMPs, while more than 900 ATMPs are in development.

https://www.doi.org/10.1016/j.jval.2016.09.814