For years, innovative medicines have become more and more expensive. The record price has just been achieved by a new treatment produced by the CSL Behring laboratory. The FDA on Tuesday (November 22) approved hemgenix, a new treatment for hemophilia B, the US equivalent of the marketing authority explains in a communicated. Price of this “gene” therapy which consists of a single dose: 3.4 million dollars (3.264 million euros), making it the most expensive drug in the world. It dethrones Novartis AG’s 2019 FDA-approved Zolgensma for the treatment of spinal muscular atrophy, which was priced at about $2.1 million, and Bluebird Bio Inc.’s zynteglo given for beta-thalassemia, worth of $2.8 million in 2022.
Hemgenix would reduce the risk of serious bleeding in patients with haemophilia B, a genetic bleeding disorder. In fact, patients do not produce a protein necessary for the formation of blood clots that stop bleeding. This disease would affect nearly one in 40,000 people. Until now, patients with this disease are treated for life with regular infusions. The new gene therapy is designed to be administered all at once: it would therefore replace the countless infusions carried out during a patient’s life. According to the two studies carried out, the treatment would free 94% of patients from the expensive infusions currently used to treat the potentially fatal disease.
A very high price compared to the cost of treating these patients for life
“Although the price is a bit higher than expected, I think (this treatment) has a chance of (meeting success) because existing drugs are also very expensive and hemophilia patients live in constant fear of bleeding,” explains Brad Loncar, biotech investor and chief executive officer of Loncar Investments, quoted by Bloomberg on November 23rd. “Today’s approval provides a new treatment option for patients with hemophilia B and represents a significant advance in the development of innovative therapies for those experiencing the heavy disease burden associated with this form of hemophilia,” added Dr. Peter Marks, director of the FDA’s Center for Hemophilia Biologics Evaluation and Research, quoted in the news release.
In a recent analysis of the drug’s cost-effectiveness, which weighs health benefits against compensatory costs, the NGO Institute for Clinical and Economic Review suggested that a fair price for the drug was between 2.93 and 2 96 million dollars, he explains Cnn November 23rd.
How is the price of a drug determined?
How is the price of a drug determined? Various factors are at play such as the cost of research, raw materials, production, promotion and distribution. If the manufacturer is free to fix his price freely, he must negotiate an amount, in France, with the High Authority of Health and the CEPS, the Economic Committee for Health Products, if he wants the latter to be reimbursed by the Security social.
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Before this committee, the manufacturer must demonstrate the benefits and innovation provided by the new treatment. Depending on the degree of medical service rendered, a level of reimbursement is established. This way of working is regularly criticized by associations that fight for greater transparency on the part of drug manufacturers, the latter not being legally obliged to share real data on research costs. In May 2019, WHO passed a resolution calling for more transparency from drugmakers in price negotiations.
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