About Us

Hepatitis C virus (HCV) infection has been the subject of a great deal of conversation in healthcare circles and the media this year. A chronic disease, HCV infection can damage the liver, is potentially deadly, and affects more than 3 million Americans. It poses a significant burden for patients, their families, and our healthcare system.

Groundbreaking new therapies have been approved recently, but questions remain about how best to screen, diagnose, and treat HCV infection. The questions focus on disease symptoms, drug side effects, treatment adherence, quality of life, population differences, and other issues that affect patient preferences and outcomes.

With this in mind, it’s no surprise that many healthcare stakeholders—patients, clinicians, researchers, purchasers and payers, and industry—have asked PCORI to consider funding studies that investigate clinical care for HCV infection.

Stakeholders at the Table

Amid a range of opinions on how to position and use these new, interferon-free, oral therapies for HCV infection, PCORI is well positioned—as an independent, nonprofit research funder—to convene a broad range of stakeholders to identify some of the questions that matter most to patients and their families.

On October 17, we hosted such a stakeholder workshop, asking whether patient-centered comparative effectiveness research (CER) might help answer critical questions about diagnosis and treatment of hepatitis C. Workshops like these are part of our established process for identifying high-priority, targeted topics for research funding opportunities.

Although we know there are questions about the cost of the new therapies, that wasn’t our focus. In fact, we don’t study cost effectiveness. Instead, we look at comparative clinical questions about what works and what doesn’t work in the prevention, diagnosis, and treatment of disease. We consider a broader range of outcomes, including longer-term outcomes, than typically required for approval by the Food and Drug Administration. Our premise is that more, reliable information is always better than less. Replacing assumptions with objective evidence enhances discussions about all treatment-related issues.

The day-long workshop brought together about 40 representatives of major stakeholder audiences to refine the topic and identify relevant research questions for potential funding. The conversations were open to the public via webinar and teleconference; if you listened in, you know that the discussions were spirited, with a wide variety of perspectives voiced.

In advance of the workshop, invitees submitted dozens of comparative clinical questions for consideration, strongly indicating that they thought there were important topics PCORI could help to study. These questions fell into four broad areas—Care Delivery, Head-to-Head Trials, Patient Populations and Timing of Treatment, and Screening and Diagnostic Tests. In the course of the day, workshop participants prioritized these questions. Examples of the higher-rated questions, by area, were:

  • How can delivery systems work to support medication adherence during treatment and achieve maximal sustained virologic responses (SVRs) in various vulnerable and at-risk populations? (Care Delivery)
  • Which of the new options for treating chronic genotype 1 hepatitis C infection are best at maximizing SVR and minimizing adverse effects and harm in various patient subpopulations? (Head-to-Head Trials)
  • What impact does the timing of treatment have on HCV patients with minimal or mild liver disease? (Patient Populations and Timing of Treatment)
  • Which evaluation methods are best for staging patients with HCV infection? (Screening and Diagnostic Tests)

We’ll be releasing a complete summary of the deliberations and prioritizations of this workshop within the next several weeks, including how prioritizations varied by stakeholder group.

In making their assessments, workshop participants considered the same criteria we apply in prioritizing any research questions we’re considering as the subject for potential funding:

  • Patient-centeredness: Is the proposed comparison relevant to patients, caregivers, clinicians, or other key stakeholders, and are the outcomes relevant to patients?
  • Impact of the condition on the health of individuals and populations: Is the condition associated with a significant burden in the US population, in terms of prevalence, mortality, morbidity, individual suffering, or loss of productivity?
  • Assessment of current options: Does the comparison reflect an important evidence gap related to current options that is not being addressed by ongoing research?
  • Likelihood of implementation in practice: Would new information generated by research likely have an impact in practice?
  • Durability of information: Would new information on this topic remain current for several years, or would it be rendered obsolete quickly by new technologies or subsequent studies?

Our Prioritization Process

We took the first step in determining whether there is a role for us in evaluating HCV treatment when our multi-stakeholder Advisory Panel on Assessment of Prevention, Diagnosis, and Treatment Options assigned the topic a high priority at its September 12 meeting. The panel is one of several that provide us with advice on potential topics for research funding.

The workshop was the next step. The input we gathered there will be folded into our overall process of deciding which research questions to address. Ultimately, our Board of Governors decides on the topics for our funding announcements and, once applications are submitted and assessed by our multi-stakeholder review panels, the Board approves those to be funded.

This process will take a bit more time, but it’s the right approach when funding large studies. It’s essential that we ask the right questions and that the studies we fund are structured to produce the most relevant, useful findings, so they will be taken up in practice. Making good choices is especially important in areas where so many stakeholders have a keen interest.

We’ll be reporting back to you within the next few months on our Board’s decision about whether to target funding to research on HPC infection treatment. I invite you to stay tuned.

Selby is PCORI’s Executive Director

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