Every day, patients, caregivers and clinicians must make any number of complex health care decisions. They might have to choose between several options for preventing, diagnosing or treating a disease or condition. They might need to decide between doing something specific and not doing anything at all. And often, they make these decisions without the evidence or information that answers the questions that matter most to patients.
The Patient-Centered Outcomes Research Institute (PCORI) was established to address this problem by funding comparative clinical effectiveness research to help patients make better-informed health and health care decisions, taking full and meaningful account of the concerns they have about the issues they face. Based on these principles, we developed our National Priorities for Research and Research Agenda, which guides our decisions about the kinds of research to fund.
To do this most effectively, we have implemented two complementary but equally critical paths: a broad, “investigator-initiated” funding process and a more directed “patient- and stakeholder-initiated” path. Together, they will help us build a robust portfolio of patient-centered outcomes research that will address the unmet needs of patients, caregivers, clinicians and other health care stakeholders.
PCORI’s Investigator-Initiated Path
This process began in May when we issued the first PCORI Funding Announcements (PFAs). These broad announcements call on the research community to submit proposals in response to our first four national priorities. Announcements related to our fifth priority will be issued in the coming months.
This approach (Figure 1, click to enlarge) leverages the broad research community’s wisdom, but brings patients and stakeholders into the process by requiring that they be included as a meaningful part of research teams and including them in the application review process. Indeed, all of our funding proposals are reviewed by multi-stakeholder teams that evaluate the plans on PCORI’s patient-centered criteria, which are outlined in each funding announcement.
PCORI’s Patient- And Stakeholder-Initiated Path
We recognize that our investigator-initiated process, even with the collaboration of stakeholders, could miss important questions that matter to patients and must be implemented judiciously. So we initiated a second path (Figure 2, click to enlarge) that directly involves patients and other stakeholders in generating questions that address specific problems identified as having a significant impact on them and the health care system as a whole.
This approach has the advantage of allowing us to focus dedicated or additional resources on areas identified as high priority. It is a more time-consuming approach because it involves reaching out to the community to solicit questions, vetting questions to ensure that they constitute true research gaps, and constituting and preparing the multi-stakeholder Advisory Panels that will help us in prioritization. But this effort is necessary if we wish to avoid funding research that doesn’t find its way into practice. It is essential to have involvement by and consensus among key stakeholders from the outset in identifying the questions and agreeing they are important if we want our research to have impact. Patients and other stakeholders need to feel a sense of ownership of the research process, and embracing research is easier when its origins are transparent, traceable and reflective of your needs.
PCORI is moving aggressively on both of these fronts, but on the second, we’re taking what we think is an especially interesting, robust, systematic, and multi-stakeholder approach to identifying and prioritizing research questions for targeted funding. We expect to have a fully developed process in place early in 2013. Our Board of Governors will make the final decision as to which specific topics might be included in the funding announcements that would follow.
Our patient- and stakeholder-initiated pathway has a series of steps:
Identifying topics. We will identify research topics from many sources, including patients and other stakeholders, previous research prioritization processes, and tapping into the knowledge of other research funders. We received many suggestions for specific research topics during the public comment period for our National Priorities and Research Agenda. Those suggestions will be considered along with input from other PCORI forums and feedback processes. To make it easier for patients and stakeholders to submit suggestions, we’ve launched a web survey form, where anyone can provide a question and explain why we should study it. We encourage everyone to “Suggest a Question.”
PCORI is just one of many research institutions working to address, through comparative studies, questions that can improve patient outcomes. To avoid duplication and to build on previous efforts, we will consider topics identified by a variety of public and private organizations, including the National Institutes of Health (NIH), the Agency for Healthcare Research and Quality (AHRQ), the National Quality Forum, the Institute of Medicine, and research and patient advocacy groups.
Ensuring that topics identified address a research gap. We’ll also systematically evaluate current research and evidence to ensure that questions identified have not been previously answered and are not being studied by others. A team of researchers will undertake this check on each topic received. The AHRQ assessment program will help determine if others already are looking at a question we’ve identified. Additional reviews of research inventories and our growing relationships with stakeholders will be vital in directing our research funding to areas where significant knowledge gaps exist. Research that addresses a recognized gap may be more likely to be noticed and, if appropriate, more quickly put into practice.
Prioritizing topics based on PCORI criteria. Any specific research questions we might pursue also must meet the criteria unique to our patient-centered mission. These PCORI criteria will be applied by multi-stakeholder advisory panels and include:
- Is the proposed research patient-centered and focused on questions and outcomes of specific interest to patients, their caregivers and clinicians?
- Is the condition or disease associated with a significant burden in the U.S. population, in terms of prevalence, mortality, morbidity, individual suffering, or loss of productivity? Or, alternatively, is it a rare disease?
- Are there indications of possible important differences in benefits and harms between the interventions under consideration sufficient to warrant conducting a research study?
- Will the study reduce the uncertainty around the treatment effects?
- How likely is it that the findings will be implemented in practice?
- How long will the information from the research be valid?
- Does the research promise potential improvements in convenience or elimination of wasted resources, while maintaining or improving patient outcomes?
- Does the research topic have the potential to include a diverse population with respect to age, gender, race, ethnicity, geography, or clinical status, or alternatively to include an understudied population?
These criteria are the same as those outlined in our PFA Application Guidelines and emphasize that we are interested in funding topics that are both patient-centered and most likely to change practice by producing information truly useful to patients, caregivers, and clinicians in the decision-making process.
We are currently developing details of the process that will be used by our advisory panels to prioritize research topics. A working group made up of PCORI Board and Methodology Committee members and PCORI staff is developing this process, with input from patient and stakeholder representatives. Patients and stakeholders will pilot this process this month and next; it will be vetted further in our December 5 public workshop, “PCORI Methodology Workshop for Prioritizing Specific Research Topics.” After the process is implemented, our Board will review prioritized lists of potential topics and make final selections.
Creating funding announcements for selected topics. In this final step, we will create targeted funding announcements based on the specific topics identified and vetted by patients and other stakeholders and approved by our Board. The result will be high-priority patient- and stakeholder-generated research projects that become a major part of PCORI’s broader portfolio.
We take very seriously our mandate to support rigorous comparative effectiveness research and patient-centered outcomes research that will build on the important work of others but bring the voice of patients, caregivers, and other stakeholders to the process in new and critically important ways. We’re confident the result will be a rich body of research that will make a difference in patients’ lives.