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Could cost put Gene Therapy out of reach for millions?

September 16, 2021

In the last decade, genetic engineering technology has made impressive strides toward the ultimate goal of curing disease by changing a cell’s genetic instructions.

The resulting gene therapies will have the ability to treat numerous diseases at DNA level with a single dose, bringing treatments for rare diseases like sickle cell disease, muscular dystrophy, and progeria.

It is even hoped that future variations of the therapies could be vital in treating more common conditions, such as heart disease and chronic pain.

However, the excitement and anticipation for gene therapy are slightly marred by the enormous price tags involved.

Gene therapies are the result of years of research and development costing hundreds of millions to billions of dollars. Sophisticated manufacturing facilities, highly trained personnel and complex biological materials set gene therapies apart from other drugs.

Pharmaceutical companies say recouping costs, especially for drugs with small numbers of potential patients, means higher prices.

The toll of high prices on health care systems will not be trivial.

A gene therapy cure for sickle cell disease, which is expected to be available in the next few years, is estimated to cost $1.85 million per patient.

It is feared that introducing dozens of similar therapies into the market would strain health care systems and create difficult financial decisions for private insurers.

The good news is that solutions are being actively pursued, including new payment methods that entice insurance companies to cover high-cost therapies and distribute risks across patients, insurance companies and drugmakers.

Another model, known as the ‘Netflix model’, would act as a subscription-based service. Under this solution, a state Medicaid program would pay a pharmaceutical company a flat fee for access to unlimited treatments. This would allow a state to provide the treatment to residents who qualify, helping governments balance their budget books while giving drugmakers money upfront.

Such a model has worked well for improving access to hepatitis C drugs in Louisiana.

Improving access to gene therapies requires collaboration and compromise across governments, nonprofits, pharmaceutical companies, and insurers.

Taking proactive steps now to develop innovative payment models and invest in new technologies will help ensure that health care systems are ready to deliver on the promise of gene therapies.

A full version of this article can be found here

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