OBJECTIVES : In August 2018, the first chimeric antigen receptor (CAR) T-cell therapies - Novartis’s Kymriah (tisagenlecleucel-T) and Gilead’s Yescarta (axicabtagene ciloleucel) - secured European approval for diffuse large b-cell lymphoma (DLBCL). Although a paradigm shift in treatment, potential weaknesses – including price, uncertainty in the data, and infrastructure obstacles – threatened to hinder uptake. This research explored the evolving reimbursement and prescribing landscape for the approved CAR T-cell therapies in the EU5 (France, Germany, Italy, Spain, United Kingdom).

METHODS : In October 2018, 253 hematologist-oncologists across the EU5 were surveyed regarding their opinions on and expected prescribing patterns for CAR T-cell therapies and 10 payers who influence pricing and reimbursement nationally or regionally were interviewed. Secondary research focused on national and regional HTA decisions in the EU5.

RESULTS : Although payers highlighted concerns about uncertainty in the data and in the logistics of administrating CAR T-cell therapies, payers had overall positive impressions of CAR T-cell therapies. Furthermore, the CAR T-cell therapies have successfully navigated HTA, pricing and reimbursement in the four of the countries under study. The majority of surveyed physicians agreed or strongly agreed the CAR T-cell therapies will fulfill an important unmet need (70-88%) and that they have the potential to replace the standard of care in select patients (66-92%). However, physicians anticipate multiple factors will limit the uptake of CAR T-cells for DLBCL, owing to clinical and payer-imposed hurdles. Approximately half of surveyed physicians (47-59%) expect the budgetary impact of these therapies to be the main factor limiting prescribing.

CONCLUSIONS : Payers and prescribers indicated that early stakeholder engagement and addressing uncertainty through managed entry agreements are crucial to overcoming uncertainty and driving the successful uptake of CAR T-cell therapies in Europe. Payers and physicians indicated that the innovation and efficacy of CAR T-cells allowed for an advantage in HTA, pricing and reimbursement negotiations and prescribing.