PNS164 Payer Assessments of CELL and GENE Therapies: WHEN Is a Drug NOT a Drug?

Abstract

The objective of this analysis is to identify where reimbursement, HTA, and pricing procedures for cell and gene therapies (CGTs) diverge from those of traditional pharmaceuticals, and to provide recommendations on how manufacturers can best prepare for HTA assessments and pricing negotiations to ensure positive outcomes for these technologies. Policies that several markets (i.e., EU5, CAN, AUS, SWE) have followed for the approval of CGTs were examined in order to identify how, if at all, these procedures diverge from those of traditional pharmaceuticals. Additionally, case studies of the N=5 approved CGTs were conducted to understand how their reimbursement outcomes vary vs. traditional pharmaceuticals (i.e., conditional on short-term re-assessments, real-world evidence (RWE) generation, value-based contracts). While ENG / SWE follow the standard approval procedure and consider CE analysis during CGTs’ HTA assessments, CAN has paved the way and designed a specific CGT assessment process which accounts for how these differ vs. typical pharmaceuticals, and AUS has developed a separate authorization to be applied to gene therapies only. Similarly, DEU is moving toward compassionate use programs for CGTs which will increase the availability of RWE and eventually allow for further data requirements in CGT risk assessments. Additionally, while there is currently no specific CGT HTA / reimbursement assessment pathway, there are some clear deviations vs. traditional pharmaceuticals in some markets such as ESP / ITA (i.e., outcomes-based contracts for tisagenlecleucel / axicabtagene ciloleucel) and FRA (i.e., national CAR T registry required with expected future re-assessment). As payers’ CGT experience increases, manufacturers should prepare for potentially differential assessments vs. typical pharmaceuticals. Manufacturers should plan to generate required data early in the life cycle to maximize the robustness of the evidence package both pre-and post-launch, closely follow CGT pricing agreements, and monitor for policy changes in how CGTs are assessed.