Medical Technology

Gene Therapy could be used to treat rare diseases, but it will cost millions

A biotechnology and policy expert discusses the imminent introduction of gene therapies, as well as the payment strategies required to get them to patients.

What you should know:

  • The most expensive drug in the world is Zolgensma, which treats spinal muscular atrophy and costs approximately $2.1 million for a one-time treatment.

  • By 2025 by 2025, the US Food and Drug Administration predicts that it will approve between 10 to 20 gene therapies per year, which likely means that other expensive treatments will soon be on the market.

  • A gene therapy treatment for sickle cells will be available in the next couple of decades, but it could cost approximately $1.85 million per patient. It could cost a single Medicare program as much as $30 million annually, even only 7 percent of the population eligible receive the treatment.

  • We need to develop new payment models to alleviate the high cost of these treatments as per Kevin Doxzen, PhD, who is a Hoffmann Fellow at both Arizona State University, Tempe, and the World Economic Forum. One way could be by linking the amount that an insurance company is willing to pay a pharmaceutical firm for a specific drug to the results of a patient’s treatment.

  • The “Netflix” model is, however it would involve the state Medicaid program paying the drug company a flat fee for access to unlimited treatments -an approach that has already proved effective for improving access to the hepatitis C treatment in Louisiana.

This is a summary of the article “New gene therapies could soon be able to treat a myriad of rare diseases, but the million-dollar price tags will put them out of the reach of many” published by The Conversation on August 31. The entire article is available at

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