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CAR-T therapies promising but cost questions remain, US price group says

Novartis AG and Gilead Sciences Inc.'s chimeric antigen receptor T cell therapies are costly, and a lot about their long-term effectiveness is still unknown, but their promise in providing a one-time treatment for blood cancers aligns with current prices, nonprofit U.S. price group the Institute for Clinical and Economic Review concluded in a draft document.

Novartis' Kymriah was approved in August 2017 for childhood acute lymphoblastic leukemia, or ALL, and the company is pursuing an indication for adult diffuse large B-cell lymphoma, or DLBCL, next.

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Gilead's Yescarta was approved to enter the market on a DLBCL indication in October, making it the first chimeric antigen receptor T cell, or CAR-T cell, therapy for this particular blood cancer, one of the most common. Still, the world of chimeric antigen receptor T cell therapies — in which patients' own T cells are engineered to target the cancer and then reinfused into their blood — is new enough that even Gilead's long-term data is relatively limited. The Institute for Clinical and Economic Review, or ICER, noted that both Gilead's Zuma trial and Novartis' Juliet trial followed patients for less than a median of one year, which significantly limited its conclusions about long-term impact.

Still, ICER concluded that each therapy showed benefit in its indicated area compared to standard chemotherapies on the market and that both Kymriah's $475,000 and Yescarta's $373,000 price tags seem to be in alignment with clinical benefits over patients' lifetimes.

Too early to analyze as rivals

The price watchdog was careful not to compare the two therapies as there have been no head-to-head trials and both have been used in relatively small patient pools thus far. It noted that while patients in Novartis' Juliet trial had significantly fewer neurotoxic side effects than in Gilead's Zuma, "this may represent real differences in the two CAR-T therapies because of differences in their co-stimulatory domains, selection bias, or chance."

However, ICER did check both companies on presenting patient response rates as a percentage of those that received the therapies, rather than the total that enrolled. In both the Juliet and Zuma trials, a number of patients dropped out before infusion due to manufacturing failures, progression of the disease and sometimes death.

Manufacturing capacity for the complex engineering process has become a critical next step in bringing CAR-T cell therapy to patients. Executives from both Novartis and Gilead have discussed plans to roll out more manufacturing facilities and infusion centers over the next year, and turnaround time has become a focal point: Gilead has its process down to 14 days, while Novartis was able to go from 30 to 22 days in the Juliet trial, a reduction that could be essential for patients with the often aggressive disease.

Outside of manufacturing, much of the treatment's payment process is still being fleshed out. ICER's model factored in this uncertainty, for instance including assumed hospital mark-ups for administering the therapy and caring for the patients. In Kymriah's case for ALL, that estimate came out to the therapy's price plus a 76% hospital mark-up.

The cost burden of hospital stays, or inpatient treatment, is increasingly playing a role in analyst's expectations for the rival therapies. Following presentations at the American Society of Hematology conference on Dec. 11, several analysts pointed out that Kymriah's relatively solid safety profile bodes well for outpatient use, with potential for lower costs and greater appeal for cancer patients.