In 2018, the European Medicines Agency (EMA) approved the first two chimeric antigen receptor (CAR)-T cell therapies (Kymriah® and Yescarta®) for marketing authorisation for the treatment of certain rare and severe cancers.
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2019 was milestone year as Novartis and Gilead managed to obtain reimbursement for these therapies in many key countries. The Health Economics and Market Access team at the Cell and Gene Therapy Catapult provides an overview of the reimbursement and funding schemes used for Kymriah® and Yescarta® in the five major European markets (France, Germany, Italy, Spain and the United Kingdom, i.e. EU5), as per the final quarter of 2019.
Kymriah® and Yescarta® launched with relatively uniform list prices across the EU5 (see table below). The reimbursement schemes used for these therapies appear to show an increased willingness both on the part of manufacturers and European healthcare system decision-makers to move beyond the more traditional reimbursement approaches (e.g. simple discounts) to payments conditional on a treatment's success. There are interesting differences between countries in terms of the types of schemes applied, and how data uncertainty is managed. In France and the UK, the therapies are reimbursed on the condition of collecting additional data at the cohort level from a combination of follow-up from the pivotal trials, as well as data from use in clinical practice in either country, which form the basis for future reassessments. In Germany, groups of health insurers (covering around 50% of the population in total) are reimbursing CAR-T therapies through outcomes-based reimbursement (OBR) agreements, whereby the manufacturers have to pay back a certain amount for patients who die after treatment. Italy and Spain provide arguably the most novel examples of OBR where reimbursement is broken down into staged payments (in three and two installments respectively) tied to patient outcomes, including survival. To our knowledge, these are the first examples of national reimbursement schemes involving outcomes-based staged payments in the EU5.
Across the EU5, early engagement with Health Technology Assessment (HTA) bodies and National Health Service (NHS) stakeholders is key, not only for securing reimbursement, but also for preparing the institutional readiness to adopt these therapies. Both Kymriah® and Yescarta® rely on highly complex requirements in terms of the care pathway, clinical infrastructure and skill of the clinical staff in order to be delivered successfully to patients. Meeting these requirements is challenging for hospitals, and manufacturers should work closely with healthcare providers to ensure the appropriate clinical infrastructure and processes are in place in preparation for the successful and timely adoption of complex therapies like CAR-Ts and to ensure the best possible health outcomes for patients treated in the real-world setting.