- The Food and Drug Administration's approval of Novartis AG's cancer therapy Kymriah was a major milestone for the rapidly maturing field of CAR-T, yet the treatment's $475,000 price tag could be a landmark of a different sort for oncology.
- Despite coming in below expectations of an even higher price, Kymriah's cost easily makes it one of the most expensive drugs on the market, sparking worries from payers and patients about affordability and also about stress on a system already burdened by rising costs.
- Already, Express Scripts Holding Co., a large U.S. pharmacy benefit manager, has voiced concerns about Kymriah's "dramatically higher" cost. In a blog post published last week, the PBM's chief medical officer said the arrival of personalized, one-time treatments like Kyrmiah will require the development of new payment models.
Kymriah (tisagenlecleucel) is the first CAR-T therapy ever approved, blazing the path to market for a crop of cell therapies designed to engineer a patient's immune cells to seek out and destroy cancers.
Such treatments promise to transform the standard-of-care for patients with hard-to-treat blood cancers who have run out of other options. Novartis' Kymriah, for example, led to complete remissions in 83% of pediatric patients suffering from a rare type of leukemia within three months of the rewired cells' infusion back into the body.
A second CAR-T approval could be around the corner, too. Kite Pharma Inc.'s axicabtagene ciloleucel is currently under review by the FDA for a more common type of lymphoma. And CAR-T therapies from other companies are advancing through development.
Although the drugs currently have only been shown to work well in blood cancers, and come with significant side effects, all signs point to CAR-T becoming a larger part of cancer treatment in the near future.
That worries Steve Miller, Express Scripts' chief medical officer, who will have to figure out whether paying for these at-times transformative treatments makes economic sense. CAR-T, considered gene therapy by some, is customized for each patient and is only administered once. As with other gene therapies currently in late-stage development, CAR-T treatments are aimed first for narrow patient populations — limiting the ability of drugmakers to recoup development costs.
"The health care system isn’t set up for this type of economic model," wrote Miller. "Ultimately, Express Scripts believes gene therapies will require payment and patient care systems which are as novel as the medications themselves."
Novartis has taken a stab at experimenting with a new approach to payment. The Swiss phama plans to work with the Centers for Medicare and Medicaid Services to provide Kymriah under a scheme that would allow for payment only if patients respond to treatment by the end of the first month.
Novartis is also likely to price Kyrmiah differently in future indications it may win approval in down the road, such as in diffuse large b-cell lymphoma.
Even so, if Kymriah works as well as it did in clinical trials, Novartis would be receiving payment for roughly eight out of every ten patients treated — meaning the challenge of budgeting for CAR-T treatment might not be solved through outcomes pricing alone.
Miller mentioned other options, such as paying for a treatment over time, establishing risk pools for insurers or financing one-time payments, as other solutions to address the challenge of paying for CAR-T and other gene therapies.
Solving the problem is in the interest of drugmakers, too. The first commercial gene therapy approved in Europe, Uniqure N.V.'s Glybera, cost more than $1 million and was unceremoniously pulled from market last year. The second approved gene therapy, GlaxoSmithKline plc's Strimvelis, has also struggled to find a market.
While both of those drugs were designed for diseases that affect just dozens of patients, there is still a lesson for gene therapy developers.
Both Novartis and Kite Pharma, which hopes to win approval for its CAR-T treatment this fall, have expressed confidence in finding a balance between earning a reward for their innovation and ensuring patient access. Yet as more gene therapies near market, that might require yet more experimentation.