A not-for-profit organization created and funded by the healthcare reform law plans to spend up to $50 million to pay for comparative effectiveness research for hepatitis C treatments just weeks before the third drug in a new breed of treatment regimens is expected to be approved.
The Patient-Centered Outcomes Research Institute, established under the Patient Protection and Affordable Care Act to fund and promote research into how conditions and diseases should be diagnosed and treated, announced plans to award grants totaling $50 million for clinical effectiveness research on treatments for chronic hepatitis C infections. (PCORI does not fund cost-effectiveness research.)
Two new treatment regimens for patients with hepatitis C were approved this year by the Food and Drug Administration. But both drugs are extremely expensive, which has raised questions about which of the millions of potential patients should undergo a course of treatment with them. About 3 million people in the U.S. have hepatitis C, which can lead to cirrhosis or liver cancer.
Gilead Sciences, the Foster City, Calif.-based drug manufacturer, is the leader in marketing the new treatments. Sovaldi, which the FDA approved a year ago, generated $8.5 billion in revenue in the first nine months of 2014. A typical course of treatment costs $84,000. Another Gilead drug, Harvoni, received approval in October. Its price tag? About $100,000 for a course of treatment.
Sovaldi has a cure rate of 95%, which is higher than the 40% cure rate associated with previous treatments. The 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.
AbbVie, the Chicago-based spinoff of Abbott Laboratories, is next in line to get a new hepatitis C treatment approved. The drug is expected to receive regulatory approval this month.
The drug regimens that existed before Sovaldi and Harvoni are considered more complex to administer, have more toxic side effects and have much lower cure rates. None of the new treatments have undergone comparative effectiveness research, 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 funding awards are expected to be released in February. Areas of focus will likely include comparing patients who start treatment right after a diagnosis to those who undergo active surveillance until disease begins to manifest, whether there are more side effects or adverse events than what was reported in the clinical trials, and how best to treat patients who are high risk of transmitting the disease, such as men who have sex with men and active injection-drug users.
The broader questions relate to the overall effectiveness of the new treatments. Are the new “oral agents as sure of a sign of long-term cure as ... the older longer treatment involving interferon?” Selby asks.
Along with the FDA approvals of new treatments, another factor at play is better understanding among clinicians for how disease progresses in patients with hepatitis C.
“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, a staff clinician in the National Institutes of Health's liver diseases branch, wrote in an editorial in JAMA this week.
In an interview, Ghany said the new treatments are considered a major improvement and have revolutionized the field. Still, physicians and researchers want to understand the long-term safety issues with the new therapies.
“We need to show that achieving a cure is doable and maintained long term,” Ghany said.
Follow Jaimy Lee on Twitter: @MHjlee
