When new drugs receive the Food and Drug Administration’s green light to hit the market, excitement about the medical advancement is often tempered by sticker shock. At $482,000 per patient for the first year and $448,000 for each year after, Roche’s new drug Hemlibra (emicizumab), meant for certain people with the bleeding disorder hemophilia A, delivers on sticker shock. But the pricey medication, which was approved by the FDA last November is, in fact, expected to lower the cost of care, according to an independent draft analysis by the Institute for Clinical and Economic Review (ICER).
The analysis, which will be finalized after a period of public comment, compares the long-term costs of care with Hemlibra to those of the current standard of care. And the initial draft finds that treatment with Hemlibra would actually cut the annual cost of care for eligible patients over age 12 by about $1.85 million per year. Annual savings on patients under age 12 is estimated at $720,000 per patient.
“The ICER report found what most of us thought would be the case, but even more so,” says Margaret Ragni, a hematologist and director of the Hemophilia Center of Western Pennsylvania, who served as a third-party reviewer of the ICER report. “It’s cost-savings by a mile.”
People living with hemophilia A lack sufficient blood clotting protein factor VIII (FVIII). Uncontrolled bleeding in these individuals can cause painful and disabling damage to muscles, joints, and nerves. Rare bleeds in the brain, eyes, throat, or GI tract can be life-threatening. To prevent these bleeds, people with severe hemophilia have traditionally self-administered IV treatment with FVIII two to three times every week. It’s a painful, costly, and burdensome way to manage the disease, says Ragni. Unsurprisingly, by the time they reach adulthood, as many as half of people prescribed this prophylactic treatment stop administering it.
What’s more, within the first 50 days of preventive treatment, as many as one in three people develop inhibitors, or antibodies, that make them resistant to the IV-infused FVIII. Instead, people with inhibitors must take larger quantities of drugs called bypassing agents, which are less effective and thus more costly, says Ragni.
“They are the hardest patients to take care of,” she says. “They have uncontrolled bleeds, [they have] more hospitalizations, they use more factor, which is extremely costly, and they die young. There’s no question in any physician’s mind that there’s got to be something better.”
It’s these complex patients who are now offered treatment with Hemlibra. Taken as a once-weekly self-administered subcutaneous injection, Helimbra prevents bleeding in people who have inhibitors. Like a self-administered insulin shot, it’s significantly less painful, less invasive, and less time-consuming than traditional IV medication. For this reason, healthcare providers expect adherence to the medication to be much higher than to IV drugs. “If you can give a subcutaneous injection versus multiple IV infusions and have something that really works at preventing bleeds, it’s a big deal in my view,” says Kenneth Bauer, a hematologist and professor of medicine at Harvard Medical School.
By taking Hemlibra, patients age 12 and older are expected to avoid an estimated 606 bleeds over their lifetime compared to taking no preventive medication at all. The drug may also help them avoid 114 more bleeding incidents than if they took bypassing agents as a preventive measure, according to the ICER review. And by curbing these bleeds, the drug reduces the need for extra medication, hospitalizations, joint damage, and other consequences of the disease, including early death.
But the drug isn’t without risks. In clinical trials, the most common side effects were injection-site reactions, headache, and joint pain. Researchers also found that patients who took bypassing agents to treat the occasional breakthrough bleed while also on Hemlibra experienced blood clots. “You really need to keep FEIBA [bypassing agent used to treat bleeds in hemophilia A with inhibitors] away from this drug, which means you need a patient who’s reliable, who does what they’re instructed to do,” says Bauer.
Lifetime treatment with prophylactic bypassing agents for patients over age 12 costs about $81.5 million, according to the ICER report. But bypassing agents don’t completely eliminate bleeds, which add another $7.9 million to lifetime costs of care. Weekly treatment with Hemlibra, on the other hand, would cost $14.9 million over a lifetime with an additional $3.8 million to treat the reduced number of bleeds, according to the report.
But the value of the new drug doesn’t lie solely in the savings it brings, Ragni says. Hemophilia A with inhibitors, and its current treatment prevents people from living life fully. The condition impacts every aspect of life from participation in school activities, to the ability to complete higher education, pursue various careers, marriage, children, travel, and overall well-being, Ragni says. “You worry every day about a bleed.”
With Helimbra, Ragni hopes the social exclusion and anxiety some patients experience will decrease because they’ll be able to manage their condition much more effectively.