- Two newly approved CAR-T cell therapies could be cost-effective even with list prices that put both treatments among the most expensive medicines currently on the market, according to a preliminary report from the Institute of Clinical and Economic Review (ICER).
- ICER, a research organization known for its work assessing the value of new drugs, found Novartis AG's Kymriah and Gilead Sciences Inc.'s Yescarta delivered a net health benefit substantial enough for each therapy to generally meet the group's threshold of cost-effectiveness.
- CAR-T therapy promises to deliver durable remissions for some patients with certain types of leukemia and lymphoma and who have largely run out of other treatment options. But the treatments' cost — $475,000 for Kymriah and $373,000 for Yescarta — have raised questions about affordability and access.
ICER's report, while a draft, looks like good news for Novartis and Gilead. With both Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) now approved, the challenge for both companies has shifted to questions of reimbursement and commercialization.
CAR-T's cost figures to be one potential roadblock to broad uptake. A report from Bloomberg, for example, found that only five patients have received Gilead's Yescarta since its approval in mid-October. Securing reimbursement — especially from Medicare and Medicaid — appears to have held up use of the treatment in hospitals, the report said.
Given CAR-T's complexity and novelty, some hurdles are to be expected. Both companies remain confident in their ability to get treatment reimbursed by government and commercial payers.
ICER's analysis could prove helpful, at least in regards to helping prove treatment's value.
"The findings of our analysis suggest that the CAR-T therapies of focus for this review provide gains in quality-adjusted and overall survival over alternative chemotherapies," ICER concluded in the report. "With the evidence available at this time, these therapies seem to be priced in alignment with clinical benefits over a lifetime time horizon."
The report issued Dec. 19 is a draft, and ICER will collect public comment over the next four weeks. A public meeting is also scheduled for March 2, when an independent council will vote on issues raised by ICER in its report.
Novartis' Kymriah won U.S. approval first, securing a regulatory OK for use in relapsed/refractory acute lymphoblastic leukemia. Gilead's Yescarta quickly followed, winning a nod from the Food and Drug Administration for relapsed/refractory diffuse large B-cell lymphoma.
Kymriah is also under review by the Food and Drug Administration as a treatment for DLBCL.
According to ICER's base case scenario, use of Kymriah would result in an incremental cost-effectiveness ratio of $57,093 per quality-adjusted life year (QALY) versus clofarabine — well below the group's commonly-used threshold of $150,000 per QALY.
Yescarta nudged close to that threshold, checking in at $145,158 per QALY versus salvage chemotherapy in ICER's base case scenario.
"Novartis is confident in the value of Kymriah, and our ability to determine a cost-effective price for this potentially definitive, one-time treatment in its approved indication and potential future indications," the Swiss pharma said in an emailed statement, indicating it remained committed to "innovative pricing arrangements" that could include indication-based pricing.
Key questions still remain about both Kymriah's and Yescarta's clinical benefit in their respective indications. As ICER notes, studies supporting the approval of therapies were uncontrolled and in a relatively small number of patients. Follow-up on participant responses to treatment remain relatively short at six months to a year, resulting in wide confidence intervals for the benefits estimated by ICER in the report.
Still, recently presented data has offered further evidence that patients who initially experience complete responses to treatment are likely to remain in response later on.
In a study of Gilead's Yescarta, for example, 42% of patients with DLBCL remained in response at a median follow-up of 15.2 months, according to results presented at the annual meeting of the American Society of Hematology earlier this month.
More follow-up is needed for both therapies, but the clinical picture to date is suggestive of CAR-T being a one-time treatment for a subset of patients.