The Food and Drug Administration on Wednesday approved a futuristic new approach to treating cancer, clearing a Novartis therapy that has produced unprecedented results in patients with a rare and deadly cancer. The price tag: $475,000 for a course of treatment.
That sounds staggering to many patients — but it’s far less than analysts expected.
The therapy, called a CAR-T, is made by harvesting patients’ white blood cells and rewiring them to home in on tumors. Novartis’s product is the first CAR-T therapy to come before the FDA, leading a pack of novel treatments that promise to change the standard of care for certain aggressive blood cancers.
Novartis’s therapy is approved to treat children and young adults with relapsed acute lymphoblastic leukemia. It will be marketed as Kymriah.
The generic name of this stuff is “Tisagenlecleucel.” Now, about that price tag:
Novartis picked the $475,000 price tag in an effort to balance patient access to Kymriah while giving the company a return on its investment, said Bruno Strigini, Novartis’s head of oncology, in a conference call Wednesday. The cost is below Wall Street analyst expectations, which reached as high as $750,000 for a dose. And it’s considerably cheaper than the roughly $700,000 price tag that U.K. regulators said would be fair considering Kymriah’s potential benefits.
For one dose?
In a clinical trial, a single dose of Kymriah left 83 percent of participants cancer-free after three months, results oncologists have hailed as a major advance for patients with few other options. The most frequent side effect was an inflammatory storm called cytokine release syndrome, a reaction to CAR-T that can prove fatal in some patients but is commonly controlled with immunosuppressant drugs.
Deaths due to cytokine release syndrome with OKT3 (muromonab-CD3) have been reported, and it can cause life-threatening pulmonary edema if the patient is fluid overloaded. However, if treated appropriately it is usually not dangerous, just extremely unpleasant for the patient.
I bet it is. And it’s probably unpleasant for one’s insurance carrier as well.
(Via Joanna Blackhart.)