PCORI to give $138M to disease researchers

The Patient-Centered Outcomes Research Institute (PCORI) has issued 6 new funding announcements offering up to $138 million in support for studies comparing how well different approaches to care work for patients given their particular circumstances and concerns.

One of the new PCORI Funding Announcements (PFAs) offers up to $50 million for up to 4 comparative clinical effectiveness studies on the best ways to diagnose and treat hepatitis C virus (HCV) infection. The other 5 announcements issued under PCORI’s National Priorities for Research provide up to $88 million for a broad range of research projects, with a pool of $12 million of that total set aside for studies that focus on rare disease.

Dr SelbyMany healthcare stakeholders have asked PCORI to consider funding comparative clinical effectiveness (CER) studies that would investigate various aspects of clinical care for hepatitis C infection, said PCORI Executive Director Joe Selby, MD, MPH.

Hepatitis C is a major chronic condition that affects more than 3 million people in the United States, the majority of whom are undiagnosed. About one-third of these individuals will develop chronic liver disease-including liver cancer-if left untreated.

Today there is a gap of evidence as to how best to detect hepatitis C for different populations, what organizational approaches are most effective for different populations and what are any health consequences of treating patients with these new agents in real world conditions and over the long run.

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“This is a particularly topical issue given the recent approval of transformational oral medications to treat HCV,” Dr Selby said. “These appear to be highly effective, but limited post-marketing data are available to date. Thus, a substantial number of CER questions have emerged about drug side effects, treatment adherence, quality of life, population differences, and other issues focused on patient preferences and outcomes.”

A PCORI landscape assessment found no other major funders supporting CER in this area, according to Dr Selby.

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“Given this and the range of opinions about how to best use the new HCV drugs, PCORI was 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,” he said.

Additionally, PCORI is especially interested in the investigation of strategies that address care for patients with life-threatening or chronically debilitating rare diseases.

“We recognize the special challenges and efforts needed to study rare diseases effectively,” Dr Selby said. “These illnesses are of such low prevalence that special efforts, such as combining data across large populations, may be needed to address them. 

underscores the importance of research on rare disease through its requirement that we establish an Advisory Panel on Rare Disease to advise on such studies.”

Health professionals and health systems often have little experience with each of the nearly 7,000 recognized rare diseases, according to Dr Selby.

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“And while individual rare diseases affect only a small number of people, there are collectively more than 25 million people overall affected by such conditions,” he said.

Information and key dates related to all of PCORI’s funding opportunities can be found in the Funding Opportunities page on PCORI’s website.