The FDA has approved a new gene therapy for the treatment of hemophilia B, a genetic bleeding disorder.
Drugmaker CSL Behring has set a price of $3.5 million for the one-time treatment.
Hemophilia patients told Insider they were excited about the new drug but worried about the price.
Bryen Lackey liked to bowl, until his blood disease got in the way.
Lackey is learning to live with a severe case of hemophilia B, a rare genetic condition that prevents blood from clotting properly. That illness required surgery on his right elbow, one of his problematic joints, which has kept the 18-year-old out of his bowling league.
As Lackey prepares to start college next spring, he said he’s excited about the potential of a newly approved treatment that could make his life less hemophilia-centric. On Nov. 22, the Food and Drug Administration approved Hemgenix, the first gene therapy for the treatment of hemophilia B. Hematologists hope the one-time treatment could end the weekly infusions patients like Lackey receive to prevent bleeding.
While the treatment leads the way as the first approved gene therapy for hemophilia, its price is also unprecedented. The drug’s seller, Australian pharmaceutical company CSL Behring, has set a list price of $3.5 million for a single treatment, making it the world’s most expensive drug, and it’s unclear whether insurance companies will pay for it.
In interviews with Insider, patients shared mixed reactions to that price point. Lackey, for example, estimates that his insurance company spends $900,000 to $1 million a year on his treatments. This means that the price of gene therapy may not be obscene in the long run if its effects last for years.
“It seems a little expensive,” Lackey told Insider, “but compared to what we’re paying, it doesn’t seem too terrible.”
A ‘good bet’ or an unfair one? Patients and experts are split over a $3.5 million price tag
CSL’s new drug is the latest in a line of new gene therapies, which are drugs that modify a patient’s DNA to correct the genetic mutation that causes their disease. These treatments can be extraordinarily effective, with a single infusion leading to transformative benefits.
But a critical unknown factor in judging CSL’s $3.5 million price tag is the length of treatment. It’s unclear how long the treatment will last, as the effects of other gene therapies diminish over time. A clinical study of 54 haemophilia B patients showed that one-off treatment reduced bleeding by 64% over 18 months, and 98% of treated patients stopped needing regular preventive infusions.
Leonard Valentino, CEO of the nonprofit National Hemophilia Foundation, called the price a “good bet,” as he expects therapy to last six to 10 years or even longer, based on clinical data in humans and animals. dogs. The typical cost of regular preventative treatments is $300,000 to $600,000 a year for a patient with severe hemophilia B, he said, meaning gene therapy would be cost-effective if he kept patients off those treatments for many years. .
Additionally, the number of patients eligible for Hemgenix will be reduced, meaning the drug’s impact on U.S. healthcare costs will be limited. Valentino estimated that 700 to 1,000 adults with hemophilia B in the United States may be interested and eligible to receive this gene therapy.
But not all patients think the $3.5 million price tag is justified. Jerry McMillan Jr., a 47-year-old with severe hemophilia B, told Insider he believes it’s “not a fair price at all” and fears some insurers may not cover the treatment. He is interested in getting gene therapy, but said he is concerned that his Medicare insurance may not cover the costs for him or others. The federal agency has not yet announced whether it will cover the cost.
Hemophilia has loomed large in McMillan’s life. As a child he went to the emergency room 20 to 24 times a month and recently had surgery to replace his left ankle. He is preparing for knee replacement surgery in January – both operations resulting from the cumulative damage of bleeding over his life, he said. He hopes gene therapy can prevent future harm, if he can access and afford it.
“There are going to be a lot of poor people who can’t afford it,” McMillan said.
Robert Lojewski, CSL’s senior vice president and general manager, told Insider that the company will offer co-pay support for people with commercial insurance if insurance companies don’t cover the full cost of treatment.
Hemgenix is the latest in a string of multimillion-dollar gene therapy drugs
Swiss pharmaceutical giant Novartis came out with what was then the world’s most expensive drug in 2019 when it priced a gene therapy for spinal muscular atrophy at $2.1 million. That record has been surpassed three times in recent months, with Bluebird Bio pricing two gene therapies at $2.8 million and $3 million, respectively, ahead of CSL’s $3.5 million price tag.
Lojewski said the $3.5 million price tag wasn’t driven by other gene therapy prices. Instead, he said the drug’s price comes from its value to patients and society and that it could save the US healthcare system money compared to the lifetime cost of hemophilia treatments today. The company also plans to offer commercial health insurers reimbursement on an undisclosed percentage of the cost if the effect of treatment isn’t lasting, he added.
“From a payer’s perspective, whether commercial or government, there are savings being made and the savings are significant,” Lojewski said.
Benjamin Rome, a health policy researcher at Brigham and Women’s Hospital and Harvard Medical School, told Insider that the US health care system needed radical reform to pay for these one-time treatments. The current system is designed around daily pills taken for chronic conditions, not complex one-time treatments that could be curative, he added.
“A one-time fee isn’t the best solution,” Rome said, “but we don’t have a health care system that can think of other ways to do that.”
Read the original article on Business Insider