Cost and Allocation of Scarce CAR-T Therapy for Patients With Multiple Myeloma

Although chimeric antigen receptor T-cell (CAR-T) therapies represent a groundbreaking advance with the potential to improve survival outcomes for patients with relapsed or refractory multiple myeloma (MM) after 4 or more prior lines of therapy, a range of practical and ethical challenges limit the timely and equitable distribution of these treatments.

In a recent study led by Kourelis Taxiarchis, MD, associate professor of medicine in the division of hematology at Mayo Clinic in Rochester, Minnesota, survey responses from MM CAR-T physician leaders at 17 CAR-T treatment centers in the United States reported a median allotment of 1 slot per month, a median of 20 patients on the waitlist, and a median wait time of 6 months prior to leukapheresis.¹ Such shortages and delays may reduce the effectiveness of CAR-T treatment and increase the risk of patient mortality.²

To gain further insights into CAR-T access issues and needed solutions, we interviewed Dr Taxiarchis and 2 other physician scientists who recently published papers on the topic: Benjamin Derman, MD, assistant professor of medicine in the section of hematology/oncology at the University of Chicago Medical Center in Chicago, Illinois, and Edward Cliff, MBBS, MPH, an Australian hematology registrar who is currently a postdoctoral research fellow in the Program On Regulation, Therapeutics, And Law (PORTAL) at Harvard Medical School in Boston, Massachusetts.^3,4

What are the key practical and ethical challenges affecting the allocation of CAR-T therapies for patients with MM? 

Dr Taxiarchis: The major practical limitation, especially at the time that our article was written, was a limited supply of these therapies in comparison to the demand. As a result, each CAR-T center of excellence surveyed in this paper had to develop internal processes to fairly prioritize patients for these treatments.¹

Dr Cliff: Both logistics and high costs represent substantial challenges for patients in accessing CAR T-cells. Only certain centers offer CAR T-cells, and timely referrals are required, often along with the need for bridging therapy to allow for the cells to be manufactured. In addition, as the costs are extremely high, with list prices exceeding $400,000 per patient, among other costs that can total $1 million, insurance companies often create additional obstacles to approval, which can further delay CAR-T therapy.⁴

Even in the highly select population enrolled in the CARTITUDE-4 trial, a number of patients either progressed or died while waiting for CAR T-cells to be manufactured.⁵

Additionally, specifically in myeloma, there have been manufacturing challenges which have limited the supply of available CAR T-cell manufacturing slots, although the companies — BMS and Janssen — have invested substantial amounts in trying to improve this manufacturing capacity, so that will hopefully improve going forward.

This article originally appeared on Hematology Advisor