PCORI's Next Steps on Hepatitis C Research Opportunities
PCORI recently convened a large stakeholder workshop to discuss whether new comparative clinical effectiveness research (CER) studies we might fund could answer key questions about how to best diagnose and treat hepatitis C (HCV) infection. It was a particularly timely event given recent regulatory approvals of groundbreaking new treatments for this serious condition. I summarized the workshop event in an earlier blog post and now want to update you on our next steps.
Based on the discussions at the workshop, our Scientific Oversight Committee will recommend to the PCORI Board of Governors at its December 8 meeting that the Board approve development of a targeted PCORI Funding Announcement (PFA) addressing key questions in detection and treatment of HCV infection. If approved, the PFA would commit up to $50 million in research funding and seek proposals to address CER questions in each of four specific areas identified by workshop attendees:
- Best approaches to screening and diagnostic testing in various patient populations
- Care-delivery interventions to support treatment effectiveness
- Head-to-head clinical trials comparing the new oral therapies for a range of patient-relevant outcomes over time
- Comparisons of immediate treatment versus monitoring for clinical progression in patients at low risk for developing serious liver disease
We believe this substantial investment of research funds will create a cluster of critical studies that will help patients, clinicians, healthcare systems, and payers develop a sound approach to identification and optimal management of this challenging chronic condition.
HCV infection provides a good example of the role PCORI aims to play in helping patients and those who care for them make better-informed decisions. PCORI supports CER that addresses issues important to patients and other healthcare stakeholders. In our work, we have the opportunity to evaluate treatment effects over longer periods and in real-world settings to learn which available treatment strategies work best for patients, given their specific conditions, concerns, and preferences. We look at a broad range of outcomes, including longer-term benefits and risks. This information is critically important to patients considering treatment, the clinicians who care for them, and many other healthcare decision makers.
After HCV was nominated as a topic by key stakeholder groups, our Advisory Panel on Assessment of Prevention, Diagnosis, and Treatment Options in September 2014 identified it as a very high priority. A month later, we brought together representatives of the entire community of stakeholders for a healthy conversation on how CER could help patients understand their options and identify preferred approaches in HCV management.
The workshop discussions were robust and passionate, and they overwhelmingly focused on issues related to care, not to the costs of the new treatments. An interesting disagreement was noted near the end of the discussion regarding the fourth key area above.
Most stakeholders endorsed the concept of a randomized controlled trial (RCT) comparing two approaches that could be implemented after diagnosis of HCV infection in US patients at low risk of developing serious liver disease—immediate therapy versus close monitoring followed by treatment as soon as there is evidence of clinical progression. Those stakeholders, including numerous representatives of pharma, payers, physicians, and researchers, felt that an RCT would be the quickest and most convincing way to settle the question of whether immediate treatment has benefits or, put another way, whether delayed treatment brings harms. But patient representatives attending the workshop felt strongly that such a trial would be unethical. In their view, the question was already settled; the smaller pre-approval trials provided enough evidence that the treatments were both highly effective and relatively safe, so immediate treatment is warranted.
In the end, we decided not to recommend pursuing trials that include randomized monitoring, but to consider proposals that compare the two approaches by studying actual outcomes due to variation in practice or coverage in the real world.
Our Board will review the Scientific Oversight Committee’s recommendations on Monday and decide what role we might best play in this important research area. I invite you to attend this meeting in person or join us by teleconference/webinar. Find out more on our website.
Selby is PCORI’s Executive Director