Gene Therapies Could Treat Rare Diseases — but Cost Millions

A biotechnology and policy expert shines a light on the looming gene therapies soon to hit the market, and the payment strategies needed to get them to patients.

What to Know:

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

  • By 2025, the US Food and Drug Administration predicts that it will be approving between 10 and 20 gene therapies per year, which likely means that other extremely high-cost therapies will soon hit the market.

  • For example, in the next few years a gene therapy cure for sickle cell disease is expected to become available but may cost about $1.85 million per patient. It could cost a single Medicare program $30 million per year even if just 7% of the eligible population receive the treatment.

  • We need to develop new payment models to alleviate the high costs of these treatments, according to Kevin Doxzen, PhD, a Hoffmann Fellow at both Arizona State University, Tempe, and the World Economic Forum. One way could be by linking the amount an insurance company pays a pharmaceutical company for a particular drug to a patient’s outcomes.

  • The “Netflix” model, on the other hand, would involve a state Medicaid program paying a drug company a flat fee for access to unlimited treatments — a method that has already proved effective for improving access to hepatitis C treatment in Louisiana.

This is a summary of the article “New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many” published by The Conversation on August 31. The full article can be found on

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