Sky-high price of $3.5 million FDA-approved hemophilia drug explained

Ushering in a new era of medicine.

Ushering in a new era of medicine.
Photo: Eric Piermont (Getty Images)

There is a unique new treatment for patients with hemophilia B on the market, but it comes at an obscene price.

Patients with hemophilia B have a rare genetic bleeding disorder in which people do not produce the protein needed to form blood clots. It is usually controlled by recurrent prophylactic treatment, where clotting factors are introduced via intravenous infusions. And even with these regular intravenous interventions, patients can face spontaneous bleeding and severe joint pain.

The gene therapy drug Hemgenix could change that. Its manufacturer, CSL Behring, touted the approval as “historic”, explaining that it “reduces the annual bleeding rate, reduces or eliminates the need for prophylactic treatment, and generates high and sustained factor IX levels for years after a single infusion”.

What’s not a bargain, however, is its price. CSL Behring plans to load a mind-blowing $3.5 million for the 10ml single-use vial, making it the most expensive medicine in the world.

However, compared to the possible benefits, including eliminating the lifetime cost of managing hemophilia B, the price seems less exorbitant. “We are confident that this award will generate significant savings for the entire healthcare system and significantly reduce the economic burden of hemophilia B,” the company saidaccording to Reuters.

Quoteable: Hemgenix is ​​a game-changer for patients with hemophilia B

“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advances in the treatment of hemophilia, preventing and treating bleeding episodes can affect a person’s quality of life. Today’s approval provides a new treatment option for patients with hemophilia B and represents significant progress in the development of innovative therapies for people suffering from the high disease burden associated with this form of disease. haemophilia. —Stone Marksdirector of the FDA’s Center for Biologics Evaluation and Research

Hemgenix, by the numbers

1 in 40,000: People with hemophilia B, most of whom are men

Over $20 million: Lifetime costs of managing hemophilia B

$2.93 million to $2.96 million: Fair price for Hemgenix, suggested by the Institute for Clinical and Economic Review

94%: Patients who discontinued factor IX prophylaxis and remained prophylactic-free in CSL studies

≥5%: Patients in the trial experienced some common side effects, including elevations in liver enzymes, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea and feeling sick.

Drugs of interest: Zolgensma, Zynteglo, Skysona

Hemgenix isn’t the only drug with a seven-figure price tag. Others include:

  • Zolgensma, at the price of $2.1 millionis used to treat children under 2 years of age with spinal muscular atrophy.
  • Zynteglo, which costs $2.8 millionis intended to treat beta-thalassemia, a rare disease whose patients often require lifelong blood transfusions
  • Skysona, a drug for a rare neurological disorder that is made by the same company that made Zynteglo, bluebird, cost $3 million

The common factor in all of these drugs is that they are all new and novel life-changing gene therapies, which work by altering genetic material in order to treat a disease.

Will insurance cover gene therapy?

Until now, insurers have mainly relied on stop-loss policies, intended to protect against catastrophic or unforeseeable losses, to cover gene therapy. But as more and more drugs are introduced in this area, in mid-2022, over 2,000 gene therapies were being developed around the world targeting dozens of therapeutic areas including cancer, neurological, blood, immunological, cardiovascular and infectious diseases – they will have to come up with a better plan.

“As the gene therapy market expands, additional challenges related to therapy delivery and complex supply chain management will also need to be addressed,” said the pharmacy benefits manager. . Optum warns. “Because these therapies have been approved based on clinical trials with very small population sizes and no long-term data, plan sponsors will need help to ensure the right patient is getting the right treatment and that the benefits will persist for a significant period of time.”

It is a complicated problem to solve. Expensive therapies place a heavy one-time burden on a single insurer in a city—all at once, writing Phillip Barker, managing director of the healthcare practice at Guy Carpenter Reinsurance Company.

Barker says some of the burden can be eased by manufacturers working with payers, like when Spark proposed discounts based on results for its treatment for hereditary blindness, or by using reinsurers to help manage risk and manage volatility. But there aren’t many precedents for how these contracts would work, so it will take some trial and error to shape them into shape.

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Eleanor C. William