A significant innovation in cancer therapy is known as CART, which really stands for chimeric antigen receptor T-cells. Let’s just keep calling it CART.
In this treatment, a sample of the patient’s white blood cells (from the T cell line) is removed from the body and treated so that it is more active in recognizing certain types of cancer cells, particularly for blood cancers. The modified cells are then injected back into the patient and the body’s immune system gets to work killing cancer cells with renewed vigor.
It’s a one-time treatment, but as you might suspect, the drug companies want to charge a lot for this service. The FDA has approved the treatments in people who have failed first-line treatments: 1) for children and young adults with B-cell acute lymphoblastic leukemia (BLL) and 2) “aggressive” B-cell non-Hodgkin lymphoma. The price tag? $475,000 and $373,000, respectively, just for the treatment. Furthermore, the jazzed up immune system causes its own set of problems. 77% of patients in one study had adverse effects from the treatment that included seizures and ICU stays. In fact, the 20% – 30% who end up in the ICU can rack up charges greater than the cost of the drug.
The drugs have not been around long nor were the randomized trials large, so no one can say if they cure disease. For some patients, this treatment is just a temporary bridge to a bone marrow transplant. In the phase 2 trials, for indication 1), the event-free response rate was 50% at one year; for 2), it was 39% at two years.
Both CMS/Medicare in the U.S. and the National Health Service in the UK (through the review by NICE) have decided to pay for the treatment. Unfortunately, both are funding these treatments through convoluted mechanisms that involve work-arounds from traditional funding processes. I don’t want to bore you with the details.
A key difference is that Medicare currently has no mechanism to negotiate a lower price or limit treatment to only the patients most likely to benefit. The NHS has a long list of requirements for the BLL that include age < 26, a long list of treatments that were tried and didn’t work, performance scores of > 50% (physical function scores), and many others. They are collecting data on outcomes such as need for stem cell transplant and other significant treatment adverse events. They are prepared to discontinue providing the treatment by 2023 if the economic analysis does not support its use.
How much does CART cost in the UK? I can’t tell you, because the NHS has negotiated a confidential discount with Novartis while the drug is in this pilot phase. NHS estimates that about 30 patients will be eligible for this experiment. Medicare is not allowed to do this. Currently in the UK, this drug is being paid for through a special Cancer Drugs Fund. Eventually, for more widespread adoption of the drug, it must meet NICE cost-effectiveness guidelines like every other treatment.
The managers of the NHS are willing to adopt new technologies, but always with limits. How does a system or a country reduce treatment costs? When it is willing to tell suppliers of exorbitantly-priced drugs, “No. We won’t buy your drug because it costs too much.” Careful observation and consideration is sometimes required that is not amenable to a quick decision. The NHS is once again a model of how to balance the needs of the few with the needs of the many.
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