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FDA Approves A New Potentially Stunning Breakthrough In The Treatment of Pediatric Cancer

How much would you pay to save a child’s life? And should money alone decide whether a young person lives or dies from a dread disease?

Such uncomfortable questions are being asked in the wake of a potentially stunning breakthrough in the treatment of pediatric cancer.

The Food and Drug Administration (FDA) earlier this month approved the first cancer drug that uses the patient’s own cells to fight the disease. Kymriah, created by pharmaceutical giant Novartis, will be used to treat lymphocytic leukemia, the most common childhood cancer in the United States.

Image: Novartis/Wall Street Journal

Each Kymriah treatment is custom tailored to the patient by removing the child’s disease-fighting white cells (T-cells) and genetically engineering them to battle the lymphoma. One expert said the Kymriah process essentially turns already-aggressive T-cells into serial killers of lymphocytic leukemia. It’s Captain America stuff, isn’t it?

The FDA approved the new therapy after a study of 63 patients found that 83% of those who took the drug went into remission. The first patient who was treated with Kymriah was reportedly near death and has now been in remission for five years. But it’s still unclear whether Kymriah is a true “cure” as some who went into remission eventually relapsed. The new treatment will be used on only those who don’t respond to other methods, probably just a few hundred patients per year.

Novartis is testing Kymriah on adults with large B cell lymphoma, but the remission rates have been less impressive.

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Still, awesome news, right? For sure. Except for one little six-digit hitch. The Kymriah treatment will cost $475,000, according to Novartis. A British research group says it could run as high as $649,000. What’s more, patients’ families may also incur significant travel costs, as Kymriah will only be administered at 32 locations around the country, where the child’s T-cells will be collected and shipped to the Novartis lab.

Patient advocate groups are understandably outraged by the Kymriah price tag. The treatment’s cost, they argue, is proof that drug pricing in the US is broken and out of control.

The critics worry that Kymriah’s price point will open the floodgate to more hyper-pricing of pharmaceuticals.

Novartis counters that it is simply trying to generate a reasonable return on the huge investment it made in developing Kymriah over the past five years in collaboration with the University of Pennsylvania. The announced sticker price, the company says, could have been much higher.

Novartis notes that because the disease effects kids, who are often covered by their parents’ insurance, few will end up paying the full price out of pocket. The company may also provide some financial assistance, such as a travel/hotel allowance, to patients with private insurance.

Medicare is considering paying for the treatment, but only if the patient shows improvement in the first month.

Patients for Affordable Drugs, an advocacy group, points out that Novartis benefited from some $200 million in federal spending on basic research into CAT-T cell therapies, which are the basis for Kymriah.

So, what are we to make of this cost/benefit conundrum? Oncology experts seem in agreement that the success of Kymriah holds the promise of radical improvement in how we treat cancer. I can’t think of any such breakthrough technology – cell phones, computers, for example – that didn’t get cheaper over time. Yes, pharmaceuticals often seem to deny this rule. But I have to believe that the cost of Kymriah-type therapies will come down over time as more companies enter the field and the admittedly significant research costs are amortized.  In the meantime, all parties with the power to help should ensure that every child who needs Kymriah is able to get it.

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