Introducing a New PCORI Research Funding Initiative–Large Pragmatic Clinical Trials

December 18, 2013 by Joe Selby, MD, MPH

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PCORI has a challenging mandate. We are required to fund primary patient-centered comparative effectiveness research (CER) and evidence synthesis, to disseminate the results of such research, and to do so in ways that provide patients, clinicians, and other healthcare decision-makers with information they can use to make better-informed decisions.

We follow a two-path approach, guided by our national research priorities, to select the research topics we study in pursuing our mandate. On one path, we use our broad funding announcements under each priority area to ask the research community for its best ideas. On the other path, we look to patient and other stakeholder communities to help us initiate and refine the process of identifying high-impact questions of greatest importance, which we then develop into targeted funding calls.

We’ve built a substantial portfolio of patient-centered outcomes research using this two-pronged approach. So far, our awards and funding commitments total $514.4 million, which includes studies of high-burden conditions, such as cancer, mental illness, and cardiovascular disease, as well as of specific topics, such as asthma in African Americans in Hispanics/Latinos, injuries from falls in the elderly, and uterine fibroids. 

More Support for Bigger, Longer Real-World Trials

As we move into 2014, and enter a three-year period of major research allocations averaging approximately $500 million annually, we’re embarking on a third path to research funding, a new initiative that combines aspects of both the investigator-initiated and the targeted approaches.  Through this effort, we’ll seek the research community’s best ideas for studies that address a more limited list of high-impact topics identified by patients, clinicians, or other stakeholders.

Applications for this initiative must propose studies that will directly compare outcomes between two or more approaches to addressing an important clinical challenge.  We refer to these as “pragmatic clinical trials” or “large simple trials.” Funding announcements for this initiative will appear in February 2014. We have outlined our general approach in a pre-announcement  posted on December 18, 2013. Thereafter, the announcements are expected to appear semi-annually.

We believe this approach is both timely and necessary.  With it, PCORI hopes to launch important studies addressing critical CER questions relatively quickly. These pragmatic studies will typically require larger and sometimes longer funding commitments than the three-year awards, with a maximum of $1.5 million in direct costs, featured in our current broad funding announcements.   We will therefore make funding available for these trials in the range of $5 million to $15 million in total costs, with terms of up to five years.

To be successful, applicants must partner with relevant patient, clinician, and other stakeholder organizations. These organizations must strongly endorse the proposed study and plan to be involved with research teams throughout the conduct of the study, so that research questions and outcomes are more relevant and findings more likely to be disseminated and implemented.  Applicants should propose studies that are large enough and long enough to capture the relevant outcomes and allow examination of possible differences in effectiveness in key patient subgroups.

Answering Questions that Matter to Patients and Other Healthcare Stakeholders

In funding pragmatic clinical trials, we’re looking exclusively for research that compares the benefits and harms of at least two approaches known to be effective for a particular clinical condition. These can include drugs, devices, and procedures, as well as other alternatives, such as medical and assistive devices and technologies, behavioral modifications, complementary and alternative medicine, and delivery-system interventions.

The proposed studies should focus on outcomes that are meaningful to patients, such as morbidity, mortality, symptoms, functional status, quality of life, and absenteeism from work or school.  We are particularly interested in trials that cut across clinical conditions and focus on patient-reported outcomes not previously studied, including pain, depression, or functional status.

PCORI and the healthcare community have already made significant progress in identifying questions to study. A number of key stakeholder organizations, along with the Institute of Medicine and Agency for Healthcare Research and Quality, through its Future Research Needs projects, have raised numerous high-priority topics that will be carefully considered as the potential subjects of funding announcements. Additional priority questions may be identified by applicant researchers and stakeholder groups, including those representing patients, clinicians, insurers, employers, and policymakers.

PCORI’s Advisory Panels continue to review these questions as well.  In the pre-announcement, we include a list of questions prioritized by these panels. We expect this list to be significantly larger by the time the announcement is released in February.

We think this new focus on funding large pragmatic trials will be an important addition to our portfolio, providing the scientific community, in partnership with patients and other healthcare stakeholders, a robust way to participate in identifying—and answering—critical research questions.

Most of all, these awards will boost our ability to achieve our goal of providing patients and those who care for them with the useful, authoritative evidence they need to make the better-informed decisions that will lead to better outcomes.

We’re interested in what you think about this new funding initiative—and all our work. Please send your comments and suggestions to info@pcori.org.

Selby is PCORI’s Executive Director

 

Related Links:

Pre-Announcement: Large Pragmatic Trials to Evaluate Comparative Clinical Effectiveness



3 Responses to“Introducing a New PCORI Research Funding Initiative–Large Pragmatic Clinical Trials”

  1. Jerry Krishnan

    Dr. Selby,
    This is welcome news! Provides the funds and duration needed to address additional, high priority topics. CONCERT intends to participate in this effort. Jerry Krishnan

    Reply
  2. Colby H. Grossman MD

    . I appreciate the opportunity to send you this comment. I practice internal medicine in a rural area. I have done clinical research for over 20 years. I submitted a very interesting proposal early on in the PCORI beginning. It looks at how aggressive education on prediabetic from a dietary education standpoint and exercise. The cardiac project analyzes prediabetic patients with respect to obesity, diabetes, blood pressure and lipid profile. This seems to fit your latest round of grants beginning in February. .. Would you suggest resubmission

    Reply
    • PCORI

      Thank you for your note. We encourage applicants to refer to the reviewer critiques to determine the level of enthusiasm for the project and opportunities to strengthen the proposal. If you would like to discuss your project with a Program Officer, you can request a meeting by submit an inquiry here: http://www.pcori.org/funding-opportunities/programmatic-inquiry or by calling 202-627-1884.

      Reply

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