It wasn't until October 2019 that an injection of what was probably the most expensive drug in the world saved the life of Baby John: A dose of the drug Zolgensma, which can be used to stop the often fatal Spinal Muscular Atrophy (SMA), cost about two million dollars. It is a very rare hereditary disease in which the muscles recede and which ensures that affected children often do not get older than 18 months.
Lottery for the world's most expensive drug
Because the drug is so extremely expensive, the Swiss pharmaceutical manufacturer Novartis has now come up with a very controversial campaign: The company is raffling a total of 100 treatments with Zolgensma to affected people worldwide. For this purpose, the patients are to be selected twice a month in an anonymized random process, to whom the drug will probably save the life. If you are not selected, you will automatically take part in the next round.
"Gambling" with sick children?
Some experts criticize the campaign as a "survival lottery", and many medical ethicists also view the concept skeptically. For example, Norbert Paul from Johannes Gutenberg University accuses the pharmaceutical giant of a covert marketing campaign aimed at undermining the approval process that is still ongoing in many countries. The lottery guarantees attention to the drug for those affected. The umbrella organization of statutory health insurance (GKV) calls the raffle a "game of chance" with sick children.
Novartis itself, on the other hand, relies on the difficult background that exists at Zolgensma: So far, the drug has only been approved for production in the USA, which means that only 100 doses can be issued outside America. That is why the lottery was agreed with an ethics council in order to be able to help as quickly as possible worldwide.
Why is Zolgensma so expensive at all?
Price collection procedures for drugs are usually very non-transparent. One criterion could be how many healthy years of life the patient can still expect with the therapy. The Institute for Clinical and Economic Review calculated that the long-term Zolgensma could cost between $ 1.1 million and $ 2.1 million. The fact that Novartis acquired Avexis, the manufacturer of Zolgensma, in 2018 for around $ 8 million should also play a role in the calculation.