“There’s no question that these gene therapies are going to be expensive. The question is: At what level will payers start pushing back and simply refuse to cover them?”
The company said the price was determined with consideration of the “clinical, societal, economic, and innovative value represented by this novel gene therapy.
In its statement to WIRED, CSL Behring said the overall effect on the health care system will be small, since hemophilia B is a rare disease affecting only about 6,000 individuals in the US. Only adults are eligible to receive Hemgenix, making the pool of potential patients even smaller. Gene therapies are made by inactivating viruses and using them as delivery devices to shuttle therapeutic genes to patients’ cells. These viruses are grown in huge stainless steel vats and later purified and tested to make sure they’re safe and function properly. All in all, it can take 10 months or longer to make enough doses for patients in a clinical trial.
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