Up to 4 million people nationwide have hepatitis C, according to the Centers for Disease Control and Prevention, and that number is increasing. Although the virus often remains undetected for many years and may not cause problems, hepatitis C can lead to cirrhosis and liver cancer if untreated. New once-daily pills are much more convenient and effective than previous treatments and have fewer side effects. Of the two most widely used drugs, Harvoni reached the market only in 2014, and Zepatier came to the market in 2016.
The new drugs’ efficacy comes with a high price tag. A 12-week course of Harvoni and Zepatier costs $94,500 and $54,600, respectively. In 2015, the Medicaid program spent more than $2 billion on Harvoni alone, making it one of the program’s top five drug expenditures.
Researchers at the University of Florida are embarking on a PCORI-funded study to compare the effectiveness of those two medications. They made sure that the study participants include people from groups that have been underrepresented in research on the infection, such as those who have substance use disorders, multiple medical conditions, minorities, and mental illness. In this study, researchers are tracking severity of liver disease and changes in the quality of patients’ daily lives during treatment and for the following three years.
In observance of Hepatitis Awareness Month, we spoke with study leader David Nelson, MD, a specialist in liver diseases at the University of Florida, and Summer Wadsworth, a middle and high school theater teacher who has had hepatitis C and is a patient partner in the study.
Patients with hepatitis C sometimes have little information on treatment options other than what’s popular and advertised on television most frequently.
Ms. Wadsworth, what was your experience as a patient?
Summer Wadsworth: As I discovered firsthand, you can have hepatitis C and not have any symptoms. I learned of my diagnosis in the mid-1980s. The treatment options included 9 to 12 months of injections with fairly brutal side effects, including extreme flulike symptoms and anemia. Since I was young, in good health, and had no evidence of liver damage according to a biopsy, doctors thought the risks of treatment outweighed the benefits for me, and that waiting for better solutions would be best.
I was fortunate. Over decades of living with hepatitis C, having multiple liver biopsies and liver enzyme tests several times a year, my liver showed little damage. However, I did suffer from a liver complication that causes severe itching during pregnancy and was a threat to the baby’s life. I had to have labor induced three weeks early.
In 2013, in my fifties, I finally decided to undergo treatment. I was treated for three months with a combination of drugs and in the end, I was 100 percent cured.
Now, there are drugs available that are easier to take, over shorter periods, with fewer side effects. It is far different from when patients had to take hepatitis C treatments for longer times, as injections instead of pills, and with lower success rates.
PCORI has funded six research projects, totaling $42.8 million, related to hepatitis. These include a project comparing the effectiveness of two approaches to care for patients with hepatitis C virus infection and who use illegal drugs, and another testing a mobile application to increase hepatitis B and C screening in Asian Americans. (See related story, Using Mobile Technology to Increase Screening for Hepatitis B and C among Asian Americans.)
See the full list of the six PCORI-funded research projects related to hepatitis.
Dr. Nelson, you’re undertaking a pragmatic study. What does that mean?
David Nelson: The study should look like what happens in real life. We’re expecting to involve 2,650 patients at 35 centers, who will be randomly assigned to get Harvoni or Zepatier. These drugs are really expensive, but they’re our best bets against the virus. Each center will use its own process for caring for patients, which should help our results have real-world applicability.
We’re making extra efforts to recruit participants from groups that have been underrepresented in previous research. Patients with multiple conditions are typically not included because of the concern they may not be able to tolerate treatments. Individuals who have an active substance use disorder may be denied entry into studies and therapy by their insurance companies, out of concern that they will not take the medications or may reinfect themselves when using injectable drugs. Before 2015, patients with psychiatric disorders were warned away from some then-staple hepatitis treatments, such as interferon, because it can increase neuropsychiatric symptoms.
Randomized controlled trial results are the only thing that will change available medication options and influence policy. By comparing the new hepatitis treatments and making this information available to the public, we can give patients and providers power when it comes to choosing treatments.
Why is it important for this study to focus on patients’ perspectives?
Summer Wadsworth: Patients with hepatitis C sometimes have little information on treatment options other than what’s popular and advertised on television most frequently. For these new medications, how do we know what patients experience?
Instead of just looking at how effective a medicine is, the study is also looking at patients’ quality of life and the long-term effects. These are practical things that patients like myself really want to know.
David Nelson: Patients have made a real impact on the development of our study. Our patient group helped us develop our study endpoints including defining what would be considered meaningful differences in both efficacy and safety of these regimens. Patients also helped develop recruitment materials to place in clinic waiting rooms.
Summer Wadsworth: The researchers included me at every step, from every conference call to every in-person meeting. I helped choose which patient-recorded outcomes questionnaire to use, did a voice recording for the study website, and along with other patient partners, put together a flier about our experiences and why we thought the study would be important. I’ve been made to feel like an equal voice.
By comparing the new hepatitis treatments and making this information available to the public, we can give patients and providers power when it comes to choosing treatments.
What would you like to accomplish with this study?
Summer Wadsworth: I hope to see the effects of treatment several years out. Current treatments have a very high success rate, but they are still new. I would like to know how patients feel after treatment and whether there are long-term effects from the medicines or from the previous infection.
David Nelson: Comparing the two drugs, on how effective and safe they are will help us find the right drug for the right person. In addition, the biggest impact would be if we could increase recognition of the value of comparative clinical effectiveness research. Without it, there will not be appropriate data to best help patients. I agree with PCORI that you get a better end product of research when the right stakeholders are at the table.
The views expressed here are those of the authors and not necessarily those of PCORI.