November 14, 2017

$1M or more: Experts warn of high total costs for first gene therapy drug for cancer

Daily Briefing

    The total costs of Kymriah, the first gene therapy for cancer approved in the United States, could surpass $1 million per patient—more than double its $475,000 list price, Liz Szabo writes for Kaiser Health News.

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    About Kymriah and its controversial price tag

    Kymriah, a leukemia drug that was approved by FDA in August, is a CAR T-cell therapy: a form of gene therapy in which doctors harvest and genetically alter a patient's own immune cells so that they're better at fighting cancer. It is approved for children and young adults who have already tried at least two other cancer treatments.

    While the drug itself carries a $475,000 list price, treatment can grow even more expensive after accounting for potential side effects.

    According to Hagop Kantarjian, a leukemia specialist and a professor at the University of Texas MD Anderson Cancer Center, halting a patient's immune system can lead to life-threatening complications that can require long hospital stays and expensive medications.

    According to Szabo, nearly half of all patients who receive CAR T-cell therapy develop "cytokine storm," a severe condition in which the immune system overreacts and causes fevers and sudden drops in blood pressure. CAR T-cell therapy can also cause stroke-like symptoms or comas.

    Because of these side effects, cancer experts estimate that the total costs of Kymriah could surpass $1 million per patient. As Leonard Saltz, the chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center said, "The sticker price is just the starting point," adding, "Think of the $475,000 as parts, not labor."

    The list price itself has drawn some controversy. Walid Gellad, co-director of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh, called the price "outrageous," arguing that the cost to manufacture Kymriah is fairly low. According to a 2012 presentation from Carl June, who pioneered CAR T-cell treatment at the University of Pennsylvania, the treatment costs around $15,000 to make.

    The drugmaker's response

    Julie Masow, spokesperson for Novartis, which manufactures Kymriah, disputed the estimate that Kymriah costs $15,000 to manufacture. Masow did not, however, specify a production cost.

    In one effort to reduce controversy about the therapy's price, Novartis has announced that it will charge patients for Kymriah only if they enter remission within one month of treatment—an approach Novartis is calling "outcomes-based pricing."

    According to Masow, Novartis is working out the specific details of the approach with CMS, but the company is "committed to making this happen," she said.

    But skeptics argue that many patients who initially enter remission will eventually relapse—about 36% within the first year, according to Vinay Prasad, an assistant professor of medicine at Oregon Health & Science University.

    For that reason, Steve Miller, the CMO at Express Scripts, argued that it would be fairer to judge Kymriah's success after six months, rather than just one.

    The debate on cost-effectiveness continues

    Kymriah's sky-high price tag has led to a robust debate on whether the drug is worth the cost.

    Masow, the Novartis spokesperson, argued, "Kymriah is a one-time treatment that has shown remarkable early, deep, and durable responses in these children who are very sick and often out of options."

    Michelle Hermiston, the director of pediatric immunotherapy at UCSF Benioff Children's Hospital San Francisco, said, "A kid's life is priceless," adding, "Any given kid has the potential to make financial impacts over a lifetime that far outweigh the cost of their cure."

    But others question whether Kymriah actually delivers value to patients. As Saltz put it, "If you've paid half a million dollars for drugs and half a million dollars for care, and a year later your cancer is back, is that a good deal?" (Szabo, Kaiser Health News, 10/17).

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