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Some serious or chronic diseases require so-called life-saving drugs. In Italy, once approved by AIFA, they are covered by the national health system as necessary for the survival of the patient. An example of a lifesaving drug is insulin for diabetics, or self-injectable adrenaline, a worldwide and relatively inexpensive drug. This last feature is not very common for this class of drugs, which are generally characterized by the very high cost.
The case of Zolgensma
It’s about a innovative gene therapy for the treatment – in a single course of administration – of spinal muscular atrophy that we hear about these days. Federico Musciacchio, this is the name of the child from Taranto suffering from severe neuromuscular disease, had received the drug last May thanks to a solidarity contest who had raised the sum necessary to buy it. Approximately two million euros: Zolgesma is considered to be the most expensive drug in the world. The same disease can also be treated with another drug, Spinraza, which costs you from 200 to 300 thousand euros per year for each patient and, unlike the first, must be taken for life.
The second most expensive drug in the world
Approved in the United States in November 2020 and in Europe (only in exceptional circumstances) last May, the drug Zokinvy is used for the treatment of Hutchinson-Gilford or Progeria and, in general, in children suffering from progeroid syndromes, causing ultra-rare genetic diseases premature aging and death. The dosage of the drug is variable and depends on age and body surface area, but for a patient who takes four 50mg capsules per day the estimated cost is approximately 1.08 million dollars per year.
Oncological therapies
Among the best-selling life-saving cancer drugs in the world are Opdivo, Keytruda and Imbruvica – the latter being the most recent – and they cost around 100 thousand euros per patient per year. Among the most expensive innovative cancer therapies there are also the so-called Car-T, which work by genetically modifying certain cells of the patient’s immune system (T lymphocytes). In Europe, the two drugs Kymriah and Yescarta were authorized in 2018 for the treatment of some blood cancers in the event that standard treatments prove ineffective. In this case, the price for a single treatment is about 330 thousand euros per patient, and is often decisive. According to data from the US FDA, the anti-cancer drugs not yet approved in Italy include the drug Danyelza, used to treat high-risk neuroblastomas in the bone and bone marrow in children over one year of age. In the United States, the cost of a vial of Danyelza in an intravenous solution is approximately $ 22,018, which, extended to one year of treatment, could range from $ 1.5 to $ 2 million.
Kalydeco and Orkambi
The two drugs for the treatment of cystic fibrosisthe most widespread serious genetic disease which counts about 200 new cases every year in Italy, have a cost of around 200 thousand euros per patient per year. In Italy, these drugs are also covered by the National Health System.
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