It’s a choice no doctor ever wants to make: which patient will get a chance to live, and which will not.
That is a decision oncologists may soon confront when the first two products in a revolutionary new way to treat cancer reach the U.S. market this year.
Unlike pills or chemotherapy, the therapy known as CAR-T can’t be stockpiled. Because it’s a bespoke treatment that involves re-engineering a patient’s cells in a specialized lab, the logistics of getting CAR-T to the thousands of Americans who will be eligible as soon as this year are uniquely daunting. Some blood cancer patients who face imminent death won’t get it in time, at least initially, forcing doctors to make difficult decisions.
“God, it’s awful,’’ said one of the founding fathers of CAR-T, Carl June from the University of Pennsylvania, tearing up at the memory of children he was unable to save. “I can’t tell you how bad.’’
CAR-T has the potential to become a blockbuster for the manufacturers of the first drugs, Swiss giant Novartis AG and Kite Pharma Inc. of California, with annual sales reaching $1 billion within five years based on analysts’ estimates. In the shorter term, the drugmakers face three major challenges to get their treatments to patients once approved by the U.S. Food and Drug Administration: a weeks-long, complex manufacturing process; potentially lethal side effects; and the limited number of medical centers that will be ready for CAR-T at launch time -- fewer than 50.
Novartis told investors last month that its treatment will target about 49,000 blood cancer patients with the most common form of non-Hodgkin lymphoma, worldwide. The first indication to get approved, in October, will be for an ultra-rare form of relapsed leukemia that develops in about 2,200 children and young adults each year.
Novartis said it has by now produced more than 250 CAR-T cocktails across various clinical studies. While that’s still a fraction of the eligible population, the drugmaker said it’s taking the necessary steps to scale up.
“We are confident we will be able to meet the needs of patients,’’ Novartis said by email.
Kite said its approach will be deliberately prudent, with a “sure and steady’’ introduction of its product. About 7,400 U.S. patients will be eligible for its initial approval in aggressive non-Hodgkin lymphoma, expected in November.
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