PHP80 - HOW CLASSIFICATION OF CELL AND GENE THERAPIES AS DRUGS, DEVICES OR PROCEDURES COULD HAVE SIGNIFICANT IMPLICATIONS FOR THEIR PRICING AND REIMBURSEMENT AND COMMERCIAL SUCCESS

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Many countries have different pricing and reimbursement (P&R) processes for prescription drugs, hospital drugs, medical devices, and procedures. Newer transformational technology classes such as CAR-T, cell- and gene-based therapies may not be classified as drugs and thus may be subject to distinct P&R processes. Indeed, CADTH have announced that it will assess Kymriah® (the first CAR-T cell therapy with marketing authorization in Canada), through its HTA process for medical devices and clinical interventions. CAR-T, cell- and gene-based therapies are classified as advanced therapy medicinal products (ATMPs) in Europe; this research examines how ATMPs have been reimbursed in major European markets. European Commission (EC)-approved ATMPs were identified alongside their reimbursement status in UK (NICE, SMC), France (HAS), Italy (AIFA), and Germany (G-BA) from relevant websites (to 12/06/2018). Ten ATMPs have received EC-approvals to date. The first four (ChondroCelect®, Glybera®, MACI®, Provenge®) have been withdrawn from the market for commercial reasons. Of the remaining six ATMPs, 5/6 have been reviewed (Holoclar®, Imlygic®, Strimvelis®, Zalmoxis®, Spherox®) by at least one national HTA body in scope, but only two have received more than one positive recommendation (Strimvelis® and Holoclar®). All national HTA bodies in scope have reviewed at least one of these currently marketed ATMPs. In Germany, 1/6 ATMPs (Holoclar®) have been classified as a procedure achieving NUB-Status 4, which translates into reference pricing; two ATMPs withdrawn from the market (ChondroCelect®, MACI®) were also classified as procedures. Reimbursement has clearly been a key challenge for ATMPs to date. ATMPs have been assessed by payers both as drugs and also as devices/procedures with significant implications on their pricing and reimbursement. Similarly, CAR-T cell therapies will also likely need to undertake different reimbursement procedures in some markets. Payer communications and evidence planning will need to be aligned to different payer stakeholders to optimize their commercial success.