Novartis considers "pay-for-performance" for CAR-T

Novartis is considering a “pay-for-performance” pricing scheme if its CAR-T therapy gets approved, following trial results published earlier this week.

Results of the phase 2 JULIET study of 51 patients in relapsed or refractory diffuse large B-cell lymphoma (DLBCL) showed 45% of patients saw their cancers respond to the treatment known as CTL019, with 37% showing a complete response where cancer was eradicated after three months.

At three months, all patients in complete response were still disease free at data cutoff, Novartis added.

The Swiss company could adopt an approach where payers fund the potentially very expensive cell therapy, should it produce this kind of response in a “real-life” setting.

It price has been predicted to be as high as $800,000 per patient – but this will hinge on which patients experience a lasting remission in their disease, and which do not.

Chimeric Antigen Receptor T-cell (CAR-T) therapies involve harvesting a patient’s own T-cells and genetically altering them so they attack cancer.

In an interview with Bloomberg, Novartis’ head of drug development, Vas Narasimhan, said the company is open-minded about how to price its drug, which the FDA could approve, or reject, before early November.

“We’re looking at multiple creative options,” Narasimhan told Bloomberg. “We’re still in the forming stages of figuring out what the right approach is going to be.”

The FDA is due to make a decision on Kite’s rival in a different type of blood cancer later in November, and in the longer term there could be competitor CAR-T products should the regulator decide these first two trailblazers are approvable.

But there are also issues such as side effects, which can be extreme, and 57% of patients experienced cytokine release syndrome, a cascade of immune proteins that can be damaging to patients’ bodies.

In JULIET, 26% of patients experiencing more severe grade 3 or grade 4 symptoms, although there were no deaths as a result and symptoms were managed. Three patients did die from disease progression within 30 days of infusion.

All of this means that Novartis may find that payers unwilling to fund the drug in all cases. Research by health economics experts at the University of York has suggested strategies such as payment for patients who achieve remission, or “lifetime leasing” based on monthly payments until a patient dies, could help to make the high costs of treatment more manageable.

However the document points to uncertainty in the evidence produced by CAR-T therapies, adding that payment-by-results was still unlikely to make treatments cost-effective when used with curative intent.

Novartis may have reasons to be optimistic about trial results, but being a pioneer in pricing and reimbursement in this cutting-edge field is also a risky business.