Big Pharma advances cancer, heart care–but with $475,000 price tags for consumers?

CAR-T-image-300x274Drug makers have just shown not only their verve in pursuing new ways to treat cancer and heart disease but also their nerve in pricing these novel therapies as if sick patients had the wealth of mega lottery winners. Just look at what Novartis is doing with the medications Kymirah and canakinumab, a drug now marketed under the brand name Ilaris.

One the one hand, it’s hard not to admire the medical science behind both, notably first Kymirah. The drug has been newly approved by the federal Food and Drug Administration to treat children and young adults for B-cell acute lymphoblastic leukemia, a “devastating and deadly” form of the blood cancer that has resisted standard treatment and often resulted in disheartening relapses.

But Kymirah, regulators agreed, offers a treatment “milestone” because it “genetically alters a patient’s own cells to fight cancer,” converting them into a “living drug,” and training them “to recognize and attack the disease.” This Chimeric Antigen Receptor (CAR) T-Cell Therapy (see illustration) “is part of the rapidly growing field of immunotherapy that bolsters the immune system through drugs and other therapies and has, in some cases, led to long remissions and possibly even cures,” as the New York Times has reported.

Meantime, canakinumab or Ilaris has just undergone a major research study (funded by Novartis) that indicates that its inflammation-fighting properties can reduce the risk of heart attacks and strokes in patients who have already had one heart attack and are at high risk for another. It also may have a key role in attacking lung cancer.

As the New York Times reported of Ilaris:

The drug works differently from the cholesterol-lowering statin medicines that have become mainstays in treating and preventing heart disease. Unlike statins, it has no effect on cholesterol. Instead, it reduces inflammation — the response by the immune system to injury or infection — which researchers have long suspected of playing a role in cardiovascular disease and cancer. About half of people who have heart attacks have normal cholesterol levels, and researchers think that in some of them, inflammation may contribute to heart and artery disease.

So Ilaris and Kymirah, especially, have excited medical researchers sufficiently so that no less than FDA chief Scott Gottlieb has greeted the cancer-fighting drug’s approval by proclaiming his agency is “committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”

Sound great?

Here’s some of what also needs to be said: Kymirah must be tailored to each patient, for whom a single dose may cost a whopping $475,000, plus the additional costs of extensive hospital care.

That’s because, as the New York Times has reported, “Kymriah can have life-threatening side effects, including dangerous drops in blood pressure.” These are so worrisome that “the FDA is requiring that hospitals and doctors be specially trained and certified to administer it, and that they stock a certain drug needed to quell severe reactions.”

As for Ilaris, it now is FDA approved only for juvenile arthritis care—treatments that can run $200,000 annually. Novartis has not said what it might charge if the drug were used more widely for heart disease. It, too, has some startling negatives.

As Stat, the health information news site, has reported: “Anti-inflammatory drugs have predictable and dangerous side effects, which showed up” in the Ilaris trial, “in which some patients wound up becoming more susceptible to serious infections, such as the bacterial skin infection cellulitis, the deadly blood infection sepsis, and even tuberculosis. That’s because the body relies on inflammation to trigger the immune system to fight such invaders. Tamp it down too much and the immune system may not leap to your defense.”

In its defense of the high cost of Kymirah, Novartis has argued that the existing therapy for the 600 or so young people who might be candidates for its drug might be bone marrow transplants. Those can have good outcomes but can cost $540,000 to $800,000.

Novartis has pledged to offer financial assistance to patients who need it to afford Kymirah, and has said it will not charge them if they do not respond within a month to the drug’s initial dosing.

As, a health information watchdog site, has pointed out, the drug maker’s pricing comments were disingenuous: Novartis claimed that Kymirah was a “bargain” compared with marrow transplants but did not include in its medication’s pricing estimates the cost of its necessary, collateral care, including hospitalization. Its refund or payback pledge also fails to reckon with the reality that, even its own limited trials (63 patients who had an 83 percent remission rate in three months), patients received more than one dose and one month of Kymirah treatment.

It’s also something of an outrage that Novartis and others in Big Pharma typically defend their sky-high drug prices by arguing they are simply recovering medications’ huge research and development expenses — such as the three-year, 10,000-patient study Novartis conducted and bona fide researchers published in the New England Journal of Medicine and the Lancet on Ilaris as a potential heart disease and lung cancer therapy. But as underscores, with Kymirah, Novartis isn’t acknowledging that the federal government—that’s taxpayers like you and me—has sunk at least $200 million to pioneer the whole CAR-T approach.

In my practice, I see not only the major harms patients suffer while seeking medical services but also the significant damages that dangerous drugs can cause—and the heartbreaking hardships that astronomic drug costs can inflict. Drug companies are businesses, and, as such, they must offer shareholders returns on their R&D investments, which can be big and long-running. But there’s a huge difference between getting a fair, reasonable ROI and just plain sticking it to your patient-customers.

Patients with both persistent heart disease and cancer confront not only life threatening and changing illness and debilitation but they and their families also can be staggered by the costs of medical services. The dazzling new immunotherapies and other cancer treatments have, in fact, acquired a new clinical description: Their costs have become so formidable that doctors, patients, families, and hospitals are describing them as a “toxic” side-effect, leading many to make risky decisions to skip or skimp on pricey meds or to forego care because it pauperizes them and their families.

When and how will Big Pharma be brought back to reality, with caregivers, patients, families, policy experts, and lawmakers finding a way both to encourage important innovations while ensuring these don’t bankrupt us all in their application?

Patrick Malone & Associates, P.C. listed in Best Lawyers Rated by Super Lawyers Patrick A. Malone
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