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 theconversation.com.
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Cite this: Gene Therapies Could Treat Rare Diseases — but Cost Millions - Medscape - Dec 29, 2021.