The company said the price was determined by considering the “medical, social, economic, and technological advantages represented by this new system.” Earlier last month, the Institute for Clinical and Economic Review, a nonprofit research organization based in Boston that evaluates the cost of drugs and other medical services, said Hemgenix would cost more than $2.9 million.
But Michael Gusmano, a professor of health policy at Lehigh University and a research analyst at the Hastings Center, an independent bioethics think tank in Garrison, New York, says the price of Hemgenix and other gene drugs should not be considered a bargain. “It thinks the cost of modern medicine is worth it,” he says. “We have a system where prices are very different around the world,” noting that the US, unlike other developed economies, does not regulate or negotiate the prices of new drugs when they come on the market.
Although most patients do not pay the full cost of the drug out of pocket, people who do not have insurance and those who have low-cost plans may have trouble getting the drug. CSL Behring says it plans to offer discounts, and other gene manufacturers have also launched programs to help patients.
In a statement to WIRED, CSL Behring said the overall health impact would be small, since hemophilia B is a rare disease that affects about 6,000 people in the US. Only adults are eligible to receive Hemgenix, which makes the pool of potential patients very small.
Paulk says gene therapy is very expensive because it is difficult and expensive to make. “This is not a little drug like Tylenol,” he says. “This is very different from your old pills. This type of medicine can often be made by robots, there are usually hundreds of doses made per day, and the process can be passed on to other people when you get the medicine. “
Gene therapy involves creating non-viral viruses and using them as delivery vehicles to deliver therapeutic genes to patient cells. These viruses are grown in large stainless steel tanks and then cleaned and tested to ensure they are safe and work properly. All in all, it may take 10 months or more to develop sufficient doses for patients in clinical trials.
Although these treatments have been in development for decades, gene therapy is still relatively new to the market. Paulk thinks the cost of production will drop—and ultimately, the price of genetically engineered drugs will drop—as companies become more proficient at producing them on a large scale. “We’re going to be very successful,” he says, but it may take five to 10 years for that to happen.
With hundreds of gene therapy drugs in the pipeline and more expected to be approved in the coming years, it could mean a multi-million dollar treatment coming to market. The banking industry has been working on these services for a long time, but there is still uncertainty as to how long the results will last. Clinical trials have followed patients for years, not decades.
If they don’t last as long as expected, Gusmano wonders what that means for prices. And even if they do, he says, million-dollar price tags don’t last long. “There is no doubt that this gene therapy will be expensive,” says Gusmano. “The question is: At what point will payers start paying back and just refuse to cover them?”