Before it received a stunning rejection from the Food and Drug Administration, BioMarin Pharmaceutical had hinted that the price tag on its hemophilia A gene therapy could fall in the $2 million to $3 million range, likely setting it up to be the world's most expensive drug. On Wednesday, a watchdog group often critical of high-cost drugs said such a price, under certain conditions, may actually be cost effective for the healthcare system.
The Institute for Clinical and Economic Review noted that its assessment is "highly preliminary," since the FDA decided this month not to approve the gene therapy called Roctavian. It will take roughly another year and a half for BioMarin to collect the data needed to resubmit Roctavian to the agency.
Even so, the cost of currently available hemophilia drugs led ICER to conclude there are several scenarios where Roctavian — which is meant to be a yearslong, if not lifelong treatment — could be cost-effective at price of $2.5 million.
Hemophilia is a rare disease in which patients lack proteins critical for clotting blood. In hemophilia A, for example, the missing protein is called Factor VIII.
It's also a very expensive disease to treat. Therapies that provide replacement clotting protein often carry a six-figure price tag — and don't entirely protect from bleeding. When patients do bleed, that too can be costly. Citing research from 2017, ICER reported the non-drug costs associated with such an event are around $4,600 for patients aged 18 to 45.
Gene therapy has emerged as a potential answer to some of these problems. Clinical tests of Roctavian and other gene therapies have shown that, after a single dose, hemophilia patients can go years without needing their typical anti-bleeding medication. Such a long-lasting option is attractive not only for patients, but for payers as well.
"Even a gene therapy that comes in at a couple million dollars, presuming durability, can be worthwhile financially speaking," Michael Sherman, chief medical officer at Harvard Pilgrim, a New England insurer, said in a recent interview with BioPharma Dive.
Indeed, ICER found "extremely high incremental cost-effectiveness ratios" when looking at two scenarios in which Roctavian has a $2.5 million list price. The first had the healthcare system retain 50% of the modeled cost savings from Roctavian instead of attributing them to the therapy. The second had the healthcare system retain cost savings beyond $150,000.
Notably, ICER's model initially found Roctavian at $2.5 million not be cost effective for the bulk of adult hemophilia A patients at standard cost-effectiveness thresholds. However, that model relied on Factor VIII use in clinical study. When it incorporated doses more representative of how patients in the U.S. use Factor VIII, the model's findings were "completely reversed."
"Overall, the findings illustrate that factor VIII is such an extremely costly treatment, especially at currently used dosages in the US, that new treatments are capable of generating large cost savings in comparison," ICER wrote.
"If prices of factor VIII were to come down from effective competition or other measures, the appropriate pricing of new treatments, as suggested by cost-effectiveness thresholds, would come down significantly as well."
ICER's draft report also evaluated Hemlibra, a hemophilia A drug sold by Swiss pharma giant Roche. Hemlibra has an annual list price of about $450,000.
Again, ICER determined that for the bulk of hemophilia A patients, Hemlibra's cost-effectiveness depends on what it's being compared to. The group found Hemlibra to be "highly cost saving," for example, when considering the higher doses of Factor VIII being used in the U.S., and when considering the larger number of patients now receiving more expensive, longer-lasting preparations.
While Hemlibra has notched hundreds of millions in sales and become a standard of care for certain hemophilia patients, doctors have noted that insurance companies have been an obstacle to access, at least early on. ICER took a look at private payer coverage of Hemlibra and found that, as of April, 12 companies had more restrictive coverage than what's on the drug's label.
Private payer policies for Hemlibra
|Payer||Covered?||More restrictive than label?||Subgroup restrictions?||Step therapy protocol?|
SOURCE: ICER, using data from the Tufts Medical Center Specialty Drug Evidence and Coverage Database. Data as of April 2020.