OBJECTIVES: Patient access to ATMPs can be limited due to restrictions set by national or regional health technology assessment (HTA) bodies and/or pricing and reimbursement (P&R decision makers). This research explores variation from regulatory to P&R decisions impacting patient access and time to patients, across Australia, EU4 and UK.

METHODS: HTA and P&R assessments for Australia, France, Germany, Italy, Spain, and the UK were obtained for the ATMPs approved in all these countries (Kymriah, Yescarta, Zolgensma, Spinraza, Luxturna). Information was researched on the websites of the Regulatory Agencies in the reference Countries. Key points investigated: level of restriction between the regulatory label population and the time to patient.

RESULTS: The average time to reimbursement for the 5 drugs was 384 (range 70-1897) days. Especially France, Spain, UK and Australia restricted ATMP’s use to specific conditions within the authorized indications (diagnostic procedure, previous therapies, subpopulations) and, in some cases, they even decided not to reimburse some indications; Italy applied strict restrictions on diagnosis or based on previous treatments, while Germany didn’t apply any restrictions. Most European countries reimbursed these drugs based on a performance-based agreement based on registries or post-marketing studies and on re-evaluation after 2 or 5 years, while Australia identified a ceiling and reimbursed only partially the cost of these drugs. Interestingly, it is possible to observe a significant difference in the identification of the therapeutic added value among the countries.

CONCLUSIONS: The significant differences between regulatory and reimbursement decisions in Australia and in various European countries created different dynamics for the access for ATMPs. There is a high variability in the HTA/P&R decision making factors at national level, and often the reasons for discrepancies are not transparent. The joint clinical assessment (JCA) foreseen by the new EU-HTA Regulation might have a beneficial impact on this topic.