Accessing miracles

How access could be an issue for Novartis’ tisagenlecleucel CAR T

With unanimous backing of an FDA advisory committee, overwhelming efficacy and a robust REMS, all signs suggest Novartis AG’s tisagenlecleucel-T is headed to the market for children and young adults with a rare leukemia. The question is how many patients will be able to access it, and not necessarily -- or at least not only -- because of the expected price for the CAR T therapy.

Novartis has not disclosed pricing plans; however, at least one analysis suggests that it would be cost effective at $500,000-$750,000. The pharma has said it is considering outcomes-based arrangements similar to those it used to secure reimbursement for its heart failure drug Entresto sacubitril/valsartan.

Price aside, the ways Novartis proposes to mitigate the risks of serious adverse events could limit access and be burdensome on patients and their caregivers.

At least initially, Novartis will distribute tisagenlecleucel-T to a limited number of infusion centers in the U.S. For families who must travel great distances to receive treatment, this could create both economic and logistical challenges. It also would mean transferring care to new physicians and caregivers.

In addition, the pharma will instruct patients to stay near the treatment facility for up to one month after treatment to ensure that side effects such as cytokine release syndrome (CRS) can be quickly managed if they occur. This adds the need to find and pay for extended lodging, and could mean extended time spent away from work and the family’s support network. It’s unclear whether payers will foot the bill for these non-hospital services.

Novartis said it is developing support programs and informational materials to assist patients and their caregivers with the logistics and ancillary expenses associated with getting to the treatment centers.

“It is hard to argue with this unprecedented clinical success in this group of patients who have no options.”

Grzegorz Nowakowski, Mayo Clinic

Bone marrow transplant (BMT) could serve as a model. In this setting, private commercial plans have been the most generous, reimbursing for travel and lodging. Medicare and Medicaid often do not provide coverage. But not-for-profits provide both financial and in-kind support for many of these patients, including complimentary lodging.

How Novartis implements the launch could provide a road map for the next CAR Ts waiting in the wings, including a therapy from Kite Pharma Inc. that is under FDA review. The biotech likewise is planning a limited distribution at launch but expects to progressively

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