Nearly a year after they hit the market in the US, two CAR-T cell therapies for blood cancers have won approval from European regulators.
US drug maker Gilead Sciences and Switzerland-based Novartis said Monday that they had received approval from the European Medicines Agency for their respective CAR-Ts, Yescarta (axicabtagene ciloleucel) and Kymriah (tisagenlecleucel). Yescarta was approved for relapsed/refractory diffuse large B-cell lymphoma, while Kymriah was approved for DLBCL and also pediatric acute lymphoblastic leukemia. The Food and Drug Administration approved Kymriah for pediatric ALL last August, followed by its approval for Yescarta in DLBCL in October, subsequently expanding Kymriah’s label to include DLBCL earlier this year. Yescarta’s label also includes relapsed/refractory primary mediastinal B-cell lymphoma, or PMBCL, a subtype of DLBCL.
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With EMA approvals in hand, Gilead and Novartis must now secure reimbursement from each of the countries the EMA covers, which will require negotiations with country-level health authorities. The EMA regulates drugs for all the European Union countries, as well as the non-EU European Economic Area nations of Norway, Iceland and Liechtenstein. The agency is currently headquartered in London but will move to Amsterdam next year following the UK’s withdrawal from the EU. Switzerland has its own regulatory agency, Swissmedic.
Gilead hit a snag Tuesday in an important set of negotiations, namely with the UK’s National Institute for Health and Care Excellence, or NICE, an agency that acts as a drug-pricing watchdog and determines whether drugs are cost-effective for use by the National Health Service. NICE determined that the incremental cost-effectiveness ratio for Yescarta was more than £50,000 per quality-adjusted life year gained, and the agency thus said it would not recommend Yescarta for routine use. NICE also pointed out that the trial used to secure regulatory approval, ZUMA-1, did not include a comparator arm despite showing patients achieved improved response rates and overall survival, meaning that the CAR-T’s benefit compared with salvage chemotherapy remains unknown. However, the agency noted that its latest determination was not a final guidance, and there will be another consultation. Kymriah remains under review.
In the US, Novartis had a pay-for-performance arrangement with the Centers for Medicare and Medicaid Services, whereby Medicaid would reimburse Kymriah’s $475,000 list price in pediatric ALL as long as patients responded within 30 days. But CMS pulled out of the arrangement after scrutiny by the agency about alleged company influence, but the drugmaker has similar agreements with private payers. Reimbursement for Kymriah and Yescarta’s $373,000 list price in DLBCL, however, has been a thornier issue. A non-binding vote by a Medicare advisory panel last week could make patient-reported outcomes in clinical trials mandatory for coverage, while an expert suggested in a New England Journal of Medicine paper that price competition or randomized trials could be a way to ensure even coverage.
Photo: Yukiko Matsuoka, Flickr
