18-year-old patient says $3.5 million hemophilia drug he needs seems a ‘little steep’

For as little as $3.5 million, you can get your hands on the latest gene therapy drug to treat hemophilia B: Hemgenix, now the world’s most expensive drug. A fleeting title these days.

Hemophilia is a genetic disorder that prevents blood from clotting when a person’s body cannot produce the protein needed to form blood clots. This causes sufferers to bleed profusely from even the smallest cuts and cuts, and sometimes even bleed spontaneously.

Symptoms are lifelong and, depending on their severity, can drastically affect a person’s livelihood. Many require weekly IV fluids, frequent hospital visits, and face too many emergencies.

Treating it is therefore a top priority, especially for hemophilia B, its more severe and rarer form.

So the maker of Hemgenix, Australian drug company CSL Behring, which is charging a paltry $3.5 million for a single round of therapy, is getting something changeable Responses from hemophilia patients, to say the least.

“It seems a bit steep,” said Bryen Lackey, an 18-year-old living with hemophilia B insider. “But compared to what we pay [currently]it doesn’t seem too terrible.”

A bit euphemistic perhaps, but Lackey’s answer shows just how costly it is to control the disease in general. He estimates his insurance company is already spending up to $1 million a year on his current treatment.

Others were much more outspoken in their criticism. Jerry McMillan Jr., a 47-year-old hemophilia B patient, told the website that the cost of Hemgenix was “not a fair price at all.”

“There will be a lot of poor people who can’t afford it,” he said Insider, Raising concerns about whether insurance companies would even be willing to cover the price of the drug.

Gene therapy drugs like Hemgenix can be life-changing even with just one round of therapy — a fact used to justify their high price. However, their benefits wear off over time.

And how insider It’s reportedly unclear how long the effects of Hemgenix will even last. At least one clinical study — believed to be the largest in hemophilia B to date — has shown that 98 percent of Hemgenix recipients did not require subsequent infusions.

And for that, it could It’s worth it, since other existing treatments that last several years cost up to $600,000 annually, according to National Hemophilia Foundation CEO Leonard Valentino, who called the drug’s asking price a “good bet.” insider.

In connection with how absurdly expensive medicines are, in other words, it could be good value. Who are we kidding – it’s ridiculous, but the problem is more systemic in nature.

“A one-time fee isn’t the best solution, but we don’t have a healthcare system that could think of other avenues,” said Benjamin Rome, an associate physician and health policy researcher at Brigham and Women’s Hospital insider.

Rome authored a research letter published over the summer that pointed to the skyrocketing prices of drugs since 2008, which have risen by nearly 11 percent each year – a worrying trend reflected in the astonishing fact that the title of “world’s most expensive drug” by has been overtaken three times in just the last few months, after insiderreported.

At this point, like Lackey, it’s not unfair to be unfair about Hemgenix’s price. It’s pretty much the norm. And that doesn’t bode well for hemophiliacs — or for patients in the American healthcare system.

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