Breaking Down the High Cost of Life-Saving Medications: Navigating the Financial Challenges of Advanced Medical Treatments

2024-03-23 04:00:00

The most pressing 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 half as much after the second. It is not a pill, but a series of injections into the cerebrospinal fluid that can only be performed by well-trained personnel, and which also consist of a sophisticated biological molecule (antisense RNA, in the jargon) that cannot be bought over the counter. druggist…

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The most pressing 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 half as much after the second. It is not a pill, but a series of injections into the cerebrospinal fluid that can only be performed by well-trained personnel, and which also consist of a sophisticated biological molecule (antisense RNA, in the jargon) that cannot be bought over the counter. drugstore. Similar arguments apply to Elaprase against Hunter syndrome ($660,000 annually), Brineura for neuronal lipofuscinosis (700,000), Carbaglu to lower blood ammonia (700,000) and from there on up. The advance of current medicine is based almost entirely on increasingly 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 of Beverly Hills.

Pharmacology progresses at an exponential rate in the 21st century, but health managers, insurers and politicians are still happily installed in the 19th century, when everything was arranged based on aspirin, saws and opium, in that order. The pasta people are not up to par with their scientists. Not even his patients.

One of the advanced therapies that are proving most useful against some leukemias and other blood cancers is called CAR-T (extending the acronym does not help to understand the concept). It consists of extracting lymphocytes (white blood cells) from the patient, modifying their genes so that they attack the cancer in question, culturing them en masse and reintroducing them into the body. It does not work for all patients, but when it does, its results save lives and make life worth living. The treatment is used only once – if it works, it cures the root problem – and costs 300,000 euros.

Large Spanish public hospitals are authorized to extract lymphocytes and reintroduce 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 a year, that is, 90 million euros. But it is likely that CAR-T, or a derived technique, will be applied to more types of tumors, and not necessarily so rare. There is promising data for the treatment of brain cancer and autoimmune diseases, such as arthritis and childhood diabetes. The figures are going to add up to zeros, and no one knows how many. Where do you get out of here?

Maybe in Bombay. ImmunoACT, A small Indian biotechnology company is producing its own version of CAR-T (called NexCAR19) at a price 10 times lower than Novartis’ original. From 300,000 euros all the way down to 30,000. This still seems like a lot, but it’s enough to poor countries can access to these vital therapies until now restricted to the rich world. Or part of the rich world, to put it more appropriately, because it doesn’t do much good for the poor in the United States to live in a rich country in cases like this. Preliminary trials in Bombay show that the leukemia has disappeared in 19 of 33 patients, and has shrunk in another four. The safety profile of NexCAR19 is even higher than the Novartis treatment.

You don’t have to be a fortune teller to know that medicine will continue to advance in the coming years through increasingly more effective and more expensive procedures. Health economists and health ministries are going to have to do a lot of numbers. An effective treatment can save a lot of costs, but not in one legislature.

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