CAR-T therapies represent a significant clinical advance for patients with certain types of advanced lymphoma. In clinical testing, Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) induced remission in half or more of the patients treated, all of whom had seen their disease advance following chemotherapy.
The cell-based therapies are expensive and complex, however. Their creation requires patients enter healthcare facilities for removal of their white blood cells, which are then engineered outside the body to recognize diseased tumor cells, and then re-infused into patients.
Treatment also requires extensive monitoring of patients to guard against common side effects, adding provider costs onto what is already an expensive therapy at $373,000 per dose.
A big issue, then, in expanding sales of Kymriah and Yescarta has been reimbursement. Medicare, which covers patients age 65 and older, has addressed this new technology via a hospital reimbursement category used for stem cell and bone marrow transplants, and bumps payment higher with a 50% “new technology add-on payment” (NTAP). CMS has proposed increasing that to 65%, which has the practical effect of bumping that payment from $186,500 to $242,450.
Hospitals providing CAR-T also receive a payment under the billing code currently used for the treatments, but that would likely still leave hospitals with a loss.
Sales of CAR-T treatments have been modest, if growing steadily. Novartis announced Kymriah sales of $45 million in the first quarter of 2019. For all of 2018, sales were $76 million.
Gilead reported $264 million in Yescarta sales in 2018 and has yet to release first quarter 2019 sales. For Gilead, rapid uptick in sales is essential to justify its $11.9 billion acquisition of Kite Pharma, Yescarta’s original developer.
CMS’ new proposal also discusses creating a separate reimbursement category for CAR-T therapy administration, which would more accurately measure hospital costs for purposes of calculating reimbursement. However, Verma told reporters that the process could take up to three years, which she acknowledged is slow as biopharma innovation accelerates.
“Our technology is moving faster than government policies,” she said.
This lag could prove significant as rival biopharma companies try to develop simpler, off-the-shelf approaches to activating the immune system. For example, German biotech MorphoSys has advanced MOR208 into potentially registrational Phase 2 trials in the same diffuse large B cell lymphoma population in which Kymriah and Yescarta are approved. This monoclonal antibody binds to the same tumor cell target as the two CAR-T agents to stimulate immune response.
Meanwhile, so-called bispecific antibodies, which can activate immune response by binding both to white blood cells and tumor cells simultaneously, have been touted as an alternative to CAR-Ts.
