Home Top Global NewsHealthcare The Era of One-Shot, Multimillion-Dollar Genetic Cures Is Here

The Era of One-Shot, Multimillion-Dollar Genetic Cures Is Here

by Ozva Admin
The Era of One-Shot, Multimillion-Dollar Genetic Cures Is Here

The company said the price was determined taking into account the “clinical, social, economic and innovative value represented by this new gene therapy.” Early last month, the Institute for Clinical and Economic Review, a Boston-based nonprofit research institute that estimates the value of drugs and other medical services, said Hemgenix would be priced fairly at over $2.9 million.

But Michael Gusmano, a professor of health policy at Lehigh University and a research fellow at the Hastings Center, an independent bioethics research institute in Garrison, New York, says the price tag for Hemgenix and other gene therapies shouldn’t be considered a bargain. . “The price of current treatments is supposed to be adequate,” he says. “We have a system where prices are completely out of line internationally,” noting that the US, unlike other economically developed countries, does not regulate or negotiate the prices of new drugs when they come on the market.

While most patients never pay the full cost of medication out-of-pocket, the uninsured and those with high-deduction plans may have trouble accessing these therapies. CSL Behring says it plans to offer discounts, and other gene therapy manufacturers have also established patient assistance programs.

In its statement to WIRED, CSL Behring said the overall effect on the health care system will be small, as hemophilia B is a rare disease that affects only about 6,000 people in the US. Only adults are eligible for receiving Hemgenix, making the pool of potential patients even smaller.

Paulk says that gene therapies are very expensive because they are complex and expensive to manufacture. “This is not a small molecule drug like Tylenol,” she says. “These are very different from the classic medicines that are taken in the form of pills. Those types of drugs can often be made by robots, often millions of doses per day can be made, and the process can be outsourced once you have that chemical synthesis suite.”

Gene therapies are performed by inactivating viruses and using them as delivery devices to deliver therapeutic genes into the cells of patients. These viruses are grown in huge stainless steel tanks, then purified and tested to ensure they are safe and work properly. In total, it can take 10 months or more to produce enough doses for patients in a clinical trial.

Although these treatments have been in development for several decades, gene therapy is still very new to the market. Paulk believes the cost of manufacturing will drop, and subsequently the price of gene therapy, as companies gain more experience manufacturing them at scale. “We will absolutely get better at this,” she says, but it could take five to 10 years for that to happen.

With hundreds of gene therapies in the pipeline, and some of them likely to be approved in the next few years, that could mean more multibillion-dollar treatments entering the market. Companies are confident that these therapies will work in the long term, but there is still uncertainty about how long their effects will last. Clinical trials have followed patients for years, not decades.

If they don’t last as long as expected, Gusmano wonders what that will mean for the price. And even if they do, he says, the million-dollar prices aren’t sustainable in the long run. “There is no question that these gene therapies are going to be expensive,” says Gusmano. “The question is: At what level will the payers start to pull back and just refuse to cover them?”

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