THE PRICE OF A CURE? ESTABLISHING THE VALUE BASED PRICE OF CAR-T CELL THERAPY FOR ACUTE LYMPHOBLASTIC LEUKAEMIA

Purpose: Regenerative medicines and cell therapies have been widely lauded for their ‘curative’ potential in a wide range of diseases, many of which currently have poor prognoses.  One of the most promising cancer therapies emerging involves equipping T cells with chimeric antigen receptors (CARs). Initial studies have shown unprecedented results in haematological malignancies, generating significant public interest and major industry investment intent on rapid commercialisation.

However, the level of personalisation and manufacturing challenges has led to concerns regarding high manufacturing costs and potential health system affordability. We sought to establish a benchmark price for CAR-T cell therapy based on their potential clinical value as opposed to manufacturing cost.

Method(s): We developed two decision models for CAR-T for treating relapsed/refractory B-cell acute lymphoblastic leukaemia in children and adolescents. The two models assessed different contexts in which the technology might be licensed: (i) as a ‘bridge to stem cell transplantation’ where the goal is to induce short-term remission to facilitate subsequent stem-cell transplant; and (ii) ‘curative intent’ where the goal is long-term remission or ‘cure’ without transplantation. We synthesised clinical evidence from existing phase II CAR-T studies, information from ongoing phase III licensing studies (sample size, endpoints and follow-up) and other epidemiological sources and estimated lifetime costs and QALYs.   The value based price for CAR-T was established for a range of cost-effectiveness thresholds (£30,000-£50,000 per QALY) to take account of different reimbursement considerations (e.g. end of life/orphan designation).

Result(s): The mean QALY gains estimated across the ‘Bridging’ and ‘Curative Intent’ models ranged from between 7.5 to 10.1 QALYs respectively, supporting the significant therapeutic advance this technology appears to provide. At thresholds of £30,000-£50,000 per QALY, the value based price of CAR-T cell therapy was estimated to be £206,800 to £356,100 (Bridging) and £327,300 to £528,600 (Curative Intent).

Conclusion(s): This is the first study to provide a benchmark price of CAR-T for ALL. These results can support manufacturers and reimbursement bodies in determining potential commercial and health system value.  The significant clinical gains provide support for significantly higher value based prices than current technologies (approx. £43,000 for clofarabine and £90,000 for stem-cell transplant).  However, the potential high upfront costs of these technologies may present additional challenges to health system financing and more innovative payment schemes and /or performance-based reimbursement may be required.