The Patient-Centered Outcomes Research Institute plans to spend up to $50 million to fund comparative effectiveness research for hepatitis C treatments just weeks before the third drug in a line of very costly new treatments is expected to be approved.
Two new treatment regimens for patients with hepatitis C were approved this year by the Food and Drug Administration. Both drugs are very expensive, raising questions about which of the millions of potential patients should receive them. About 3 million Americans have hepatitis C, which can lead to cirrhosis or liver cancer.
Gilead Sciences' drug Sovaldi, which the FDA approved a year ago, costs $84,000 for a course of treatment. Another Gilead drug, Harvoni, which received approval in October, costs about $100,000. A new drug from AbbVie is expected to receive regulatory approval this month.
Sovaldi has a cure rate of 95%, higher than the 40% cure rate associated with previous treatments. The Sovaldi treatment regimen is also shorter than some of the older treatments. The drug's arrival on the market last year triggered widespread debate about whether all patients with hepatitis C should be treated and what role cost plays in making care decisions.
The drug regimens that existed before Sovaldi and Harvoni are more complex to administer, have more toxic side effects and have much lower cure rates. But the new treatments have not undergone comparative effectiveness studies, said Dr. Joe Selby, PCORI's executive director.
“There's a lot that can be learned about how we administer these drugs to patients,” he said.
The PCORI funding awards are expected to be announced in February. Areas of focus likely will include comparing patients who start treatment right after a diagnosis to those who undergo active surveillance until the disease begins to manifest; studying whether there are more side effects or adverse events than those reported in clinical trials; and how best to treat patients who are at high risk of transmitting the disease.
There also needs to be better evidence to help clinicians understand how hepatitis C progresses in patients.
“Evidence of the benefits of treating patients with mild disease is lacking, and the new impediment to treatment is now the cost of therapy,” Dr. Marc Ghany, who works in the National Institutes of Health liver diseases branch, wrote last week in an editorial in the Journal of the American Medical Association.
In an interview, Ghany said the new treatments are a major improvement. Still, physicians and researchers want to understand the long-term safety issues. “We need to show that achieving a cure is doable and maintained long term,” Ghany said.