On Target: Jump-Starting Our Push for Specific Patient-Centered Research Projects

We here at PCORI get a steady stream of useful feedback on our research efforts from all of our many stakeholder communities. One of the clearest of those pieces of input has been about the critical need for us to identify specific, high-priority topic areas for study.

In response, our Board of Governors has approved the development of three targeted funding announcements that will seek research proposals on specific topics that are especially meaningful to patients and those who care for them.  These announcements will complement the broad funding announcement process we launched in the spring by, in effect, jump-starting our patient- and stakeholder-initiated approach to topic-specific research funding, one of two complementary paths we're following as guided by our National Priorities for Research.

The initial topics selected for our targeted announcements are 1) treatment options for uterine fibroids; 2) the safety and benefits of treatment options for severe asthma; and 3) fall prevention in the elderly. Each addresses a medical concern that affects millions of people and raises important unanswered questions about the most effective treatment or prevention approaches, depending on patients’ circumstances and preferences. Our research will focus on providing these patients and those who care for them with trusted information they can use when choosing which prevention, diagnostic or treatment option is most appropriate for them.

The three topics were chosen through a multi-step process that built upon previous stakeholder-driven efforts, from across the healthcare community, to prioritize comparative effectiveness research needs. Sources included the Institute of Medicine, Agency for Healthcare Research and Quality, National Institutes of Health, and several other organizations. We identified nearly 500 questions that these earlier efforts considered worth further study. We sorted this list to identify overlapping questions, ie, questions suggested by at least two organizations.

This resulted in 40 topics. Six PCORI staff then ranked this list using decision-analysis software, using two sets of criteria. The first looked at:

• Salience: Of obvious and recognizable importance, meaning most people would know this question is important to patients.
• Short-term feasibility: The study results could be available within a two-to-three-year period.
• Unmet research need: The research would be unlikely to be funded without PCORI support.
• Reasonable resource needs: Moderate investments in research could suffice or could leverage existing co-funding opportunities.

These criteria were intentionally designed to identify research topics that had the potential to yield results that could substantially improve patient outcomes over a relatively short period of time. Our desire to identify these topics reflects our understanding of the need that patients, caregivers and clinicians have for high-quality information that can significantly improve their ability to make critical health and healthcare decisions.

Along with these criteria, we reviewed the topics based on PCORI’s merit criteria, which let us focus on questions most important to patients and caregivers.  These criteria, which we apply to all of funding proposals, are:  (1) patient-centeredness; (2) prevalence and impact of the condition; (3) potential to improve the condition through research; (4) impact on healthcare performance; and (5) inclusiveness of different populations.

These filtering processes allowed us to rank-order the 40 topics according to both sets of criteria. We presented the top 25 of these to the Program Development Committee (PDC) of our Board of Governors. The PDC then assessed the potential impact of each on various populations and specific diseases or conditions and narrowed the number to 11.

Through targeted outreach to experts in the field, we then came up with three topics that we believed represented high-priority areas for research that covered a variety of study populations and conditions. The Board advised PCORI staff to develop funding announcements for those three and instructed us to two more topics to consider for possible announcements later on.

In preparing the funding announcements, PCORI will convene experts and stakeholders to refine research questions in each area. We plan to conduct review in early 2013 with panels of multiple stakeholders, as well as seek additional public input, before finalizing and releasing the targeted funding announcements in the first half of 2013. We’ll share details of the funding announcement development process through our website as soon as they are available. These announcements, our first such targeted calls for research proposals, represent another landmark achievement in PCORI’s development as a successful national research organization.

This short-term process for selected targeted funding announcements jumpstarts our long-term topic generation and research prioritization effort, which will create a sustainable, transparent and patient-centered approach to research prioritization and selection. We are proud of the progress we’re making in developing a strong research portfolio, and we look forward to continuing our work together towards better outcomes for patients.