Is the gene therapeutic drug worth 2 million dollars?

In March 2020, the European Medicines Agency recommended granting a conditional marketing authorisation in the European Union for a virus vector-based gene therapeutic drug Zolgensma (ema, 2020). The drug have been used before in United States and Japan for the treatment of pediatric patients younger than two with spinal muscular atrophy (SMA), a group of neuromuscular disorders that are often fatal and result in  muscle weakness and progressive muscle wasting. With a US list price of 2.125 million dollars for a single dose, Zolgensma becomes the most expensive drug in the world ever.

Zolgensma is the world's most expensive drug (Credit: KARE)

In spite of the extremely high price, the drug is still considered as a groundbreaking gene therapeutic milestone. To begin with, Zolgensma is a lifesaving treatment. About one child in every 8000 live births is born with SMA. Most of the patients do not survive early childhood, even if they do, they are growing up with a respirator and disability of walking on their own (dailymed,2019). Moreover, previous medicine named Spinraza requires spinal injections 3 times per year for a patient’s entire life with a price of 125,000 dollars per injection. In the long run, one-shot treatment of Zolgensma is actually not that expensive comparing to a lifetime of Spinraza injections.

A price tag should never be put on a kid’s life but a possible cure comes with one. The eye-popping cost of the drug has also raised ethical dilemmas about what a cure for kids less than 2 years of age is worth. For parents of a SMA patient, they only get two years at most to raise the money before their kid becomes too old to receive the medication. When young patients from a poor family blow out the candles on their second birthday, they are actually extinguishing their hopes of a cure. As a result of high pricing of Zolgensma, Novartis, the company producing it, has received fierce criticism from patient group. Thus, a “lottery programme” has been launched by the company to give away 50 free treatments to randomly chosen patients. However, the effort was still not appreciated by everyone.

 “It proves no sincere commitment to patients and only increases the distress of the families concerned. They are given false hope. If one equals the fate of a patient to a lottery ballot, human dignity and moral values get out of sight. Lotteries are by their nature a form of gambling, and this is absolutely the wrong model to bring to healthcare.” The health ministers of Belgium, the Netherlands, Luxembourg, Austria, and Ireland jointly condemned the programme (Owen Dyer, 2020).

Yes, the company invested a huge amounts of money to develop the drug and it is indeed a fabulous product, but is the gene therapeutic treatment really worth 2 million dollars? Share your opinions in the comment section below.

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1 Reply

We cannot deny it to these kids despite the obscene price tag - so the company has us getting the prior auths and pleading for it for free. This is unspeakable for the lottery losers. Some of them participated in trials and raised funds and now the dream is a nightmare. Also it's even worse - the one baby who looks like they have a near normal neuro exam got their dose in the first few months of life. The ones getting it closer to age two seem to still need to the RNA based therapies too. I hope as more gene therapies are done for more kids we see the prices come down out of the stratosphere.

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