1:21 PM
 | 
Jul 21, 2017
 |  BioCentury  |  Strategy

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 grow the pool of eligible centers by sevenfold within the first year, which could reduce some of the burdens on patients.

Driving to remission

Tisagenlecleucel-T (CTL019) is the first chimeric antigen receptor T cell (CAR T) therapy to come under FDA review. Novartis is seeking approval to treat patients aged three to 25 with relapsed/refractory B cell acute lymphoblastic leukemia (ALL). The PDUFA date is Oct. 3.

On July 12, FDA’s Oncologic Drugs Advisory Committee voted 10-0 that the product had an acceptable benefit-risk profile for the indication.

Tisagenlecleucel-T showed more than double the response rate of available therapies.

In the open-label, pivotal Phase II BB2202 trial, tisagenlecleucel-T met the primary endpoint with an overall remission rate (ORR) of 82.5% in 63 ALL patients, with 75% still in remission at six months. And while overall survival had not yet been reached, the estimated median OS was 16.6 months. All patients were negative for minimal residual disease (MRD).

Some patients went on to receive a hematopoietic stem cell transplant and were censored from the overall survival analysis. Transplant is considered potentially curative in ALL; however, patients must be in remission and MRD-negative to qualify.

It remains to be seen if tisagenlecleucel-T could also be curative.

“Right now we have just a short-term response for most patients and not a lot of experience with it over longer periods of time. So I think that different parents are going to make different decisions about the potential long-term consequences and how they want to deal with them,” Helen Heslop told BioCentury. She is the director of the Center for Cell and Gene Therapy at Baylor College of Medicine.

There is evidence that for some patients, the product may provide a one-dose cure.

During the open public hearing at the meeting, Thomas Whitehead testified that his daughter Emily is still cancer free after being the first patient to receive the therapy in 2012. Don McMahon also reported that his son Connor has been in remission for a year since receiving tisagenlecleucel-T in the pivotal trial.

Prior to receiving tisagenlecleucel-T, Emily’s physicians had said there were no other options and recommended hospice care.

“Our daughter was going to die, now she leads a normal life. If you want to see what a cure looks like for relapsed ALL, she’s standing right...

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