“There are millions being made out there on these kids – it’s a huge business,” said Doris Quon, MD, medical director of the Orthopaedic Hemophilia Treatment Center at the University of California, Los Angeles.
Contributing to the costs is the fact that there is no cure for hemophilia and no cheaper substitute for blood factor. Factor may be prescribed at high doses for a lifetime, even more so when a patient has an injury or complications.
Nationwide, a third of adults and children living with hemophilia are covered by Medicaid. And the Medicaid program’s three most expensive drugs per prescription are for hemophilia, according to an analysis by the Kaiser Family Foundation. (California Healthline is produced by Kaiser Health News, an editorially independent publication of the foundation.)
In 2015 alone, Medicaid paid about $353 million for prescriptions of Advate, the most commonly prescribed blood-clotting medication for hemophilia – a 273% increase from 2011.
Generally speaking, the price of hemophilia drugs rise as rival drugs hit the market. But, in addition, doctors are prescribing ever more clotting factor for prevention of joint-damaging bleeds and for improved long-term health. The increase in the cost of Advate, for example, was nearly all attributed to increased use.
Tab for 145 kids: $195 million
The California boy whose drugs cost $21 million in a single year was an extreme case, and the circumstances of his care have not been disclosed because of confidentiality protections. Still, medications to treat hemophilia on average annual cost more than $270,000 per patient, according to a 2015 Express Scripts report, and they can easily soar past $1 million annually.
In contrast to more common diseases like hepatitis C, hemophilia treatment is not a state “budget buster” per se: about 4,000 patients live in California. About 1,100 of them are covered by Medi-Cal or two other government-funded programs for chronically ill children in California, according to Jennifer Kent, director of the state Department of Health Care Services and author of last year’s tweet. But the amount of money spent per person dwarfs that spent on people with other serious diseases.
One Stanford University study of 34,000 California kids with severe chronic diseases found that the tiny portion of children who needed blood factor accounted for 41% of the state’s outpatient drug spending on this entire patient population. About $195 million was spent on just 145 kids over a 3-year period, although some of that money came back to the state in rebates from drug companies – a portion of the cost that Medicaid can recoup after purchase.
Caitlin Carroll, director of public affairs for PhRMA, the pharmaceutical industry lobbying group, said high development costs and the complicated and lengthy manufacturing process play a role in how hemophilia drugs are priced. She added that federally mandated rebates significantly reduce the cost of blood factor. They amount to 17% of the average manufacturer price per unit.
Manufacturers also note that some newer and more expensive hemophilia drugs last longer and do not need to be administered as frequently, so they can prove less costly to payers overall.
Even so, some patients require a monumental investment to survive.