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The most serious problem in medicine is not scientific, but financial. Nusinersen, a drug against spinal muscular atrophy, costs $750,000 for the first year of treatment, and halves after the second. It is not a pill, but rather a series of injections into the cerebrospinal fluid that can only be performed by well-trained personnel, and it also contains a sophisticated biological molecule (antisense RNA in the jargon) that cannot be purchased over the counter. Could. , Medicine seller…
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The most serious problem in medicine is not scientific, but financial. Nusinersen, a drug against spinal muscular atrophy, costs $750,000 for the first year of treatment, and halves after the second. It is not a pill, but rather a series of injections into the cerebrospinal fluid that can only be performed by well-trained personnel, and it also contains a sophisticated biological molecule (antisense RNA in the jargon) that cannot be purchased over the counter. Could. , Medicine shop. Similar arguments apply to elaprase against Hunter syndrome ($660,000 annually), Brineura for neuronal lipofuscinosis ($700,000), carbaglu to reduce ammonia in the blood ($700,000), and onward from there. Current medical progress is based almost entirely on more advanced, more effective, and more expensive treatments. Even Hollywood’s weight-loss drug Ozempic – which also reduces diabetes and heart risk – is cost-prohibitive outside Beverly Hills.
Pharmacology is rapidly progressing in the 21st century, but health managers, insurers and politicians are still happily set in the 19th century, when everything was organized on the basis of aspirin, sawdust and opium, in that order. The pasta people are not equal to their scientists. Not even his patients.
One of the advanced therapies that is proving most useful against some leukemias and other blood cancers is called CAR-T (expanding the acronym doesn’t help understand the concept). It involves extracting lymphocytes (white blood cells) from the patient, modifying their genes so that they can attack the corresponding cancer, growing them en masse and reintroducing them into the body. It doesn’t work for all patients, but when it does, the results save lives and make life worth living. The treatment is used only once – if it works, it cures the original problem – and costs 300,000 euros.
Large Spanish public hospitals are authorized to extract lymphocytes and reproduce them, but the genetic modification is the property of Novartis, a Swiss pharmaceutical company. In Spain, funding is provided for two rare cancers that affect 300 people per year, i.e. 90 million euros. But it is likely that CAR-T, or a derivative technology, will be applied to more types of tumors, and not necessarily to such rare ones. There is promising data for the treatment of brain cancer and autoimmune diseases such as arthritis and childhood diabetes. This figure is about to reach zero, and no one knows how many there will be. Where do you get out of here?
Maybe in Bombay. ImmunoACT, a small Indian biotech company, is producing its own version of CAR-T (called NexCAR19) at a price 10 times lower than Novartis’ original price. From 300,000 euros to 30,000 euros. This still sounds like a lot, but it is enough for poor countries to access these vital treatments that have until now been limited to the rich world. Or part of the rich world, to put it more appropriately, because in such matters it is not very good for the poor of the United States to live in a rich country. Preliminary trials in Bombay show that the leukemia has disappeared in 19 out of 33 patients, and has subsided in the other four. The safety profile of NexCAR19 is even higher than that of the Novartis treatment.
You don’t have to be a prophet to know that medicine will continue to advance through more effective and more expensive procedures in the years to come. Health economists and health ministries have a lot of work to do. An effective treatment can save a lot of costs, but not in a legislature.
(Tags to translate)Opinion
