About the Accelerated Process to Targeted PFAs

In December 2012, PCORI’s Board of Governors approved the development of three targeted topics for research funding to jump-start our long-term topic generation and research prioritization strategy. These topics were selected through a rigorous process that complements our long-term strategy.

Step 1: Initial Review of 488 Topics from Five Sources

Step 1: Initial Review of 488 Topics from Five Sources
To initiate our accelerated effort to develop targeted funding announcements, we reviewed previous efforts to identify gaps in comparative effectiveness research (CER), obtaining topics and questions from five organizations:  the Institute of Medicine (IOM), Agency for Healthcare Research and Quality (AHRQ), National Institutes of Health (NIH), American Nurses Association, and Friends of Cancer Research.  We also reached out formally to other professional organizations through our engagement work, although we did not uncover additional lists of vetted CER topics or questions to consider.  Based on this information, we identified 488 high-priority topics and questions. Most of these were previously prioritized without respect to condition or population, but some were generated based on their relevance to a particular population or interest area (eg, those related to cancer). See more about this step in the process here.

Step 2: Identification of Topics from Two or More Sources

Step 2: Identification of Topics From Two or More Sources
This list was examined to identify “overlapping” topics and questions—those raised by two or more organizations—using a broad definition of “overlapping” that included a shared topical area, specific comparators, and thematic domains. For example, topics or questions that contained the phrase “cardiac arrhythmia,” “supraventricular tachycardia,” “atrial fibrillation,” “thrombosis in atrial fibrillation,” or “side effects of drugs” to treat any of these conditions were treated as overlapping. If a topic or question was previously prioritized primarily because it had not been well-studied in a particular population (eg, African-Americans), it was broadened to include all other relevant populations (eg, Latinos). If a topic or question was prioritized twice (eg, once in the elderly and once for women only), it was broadened to include both the elderly and women. PCORI staff used decision-analysis software to rank these questions. This effort resulted in 40 CER topics. PCORI staff developed abstracts for each topic. These included brief descriptions of each topic, the impact of the condition, potential research questions for consideration, and answered the following questions:  What are the important potential research questions in this area that could lead to better information and answers for patients and other stakeholders? Are there specific opportunities for PCORI in this area, such as the ability to strengthen an important study, or speed delivery of results by cofunding with another agency, or by adding to previously funded projects?  See more about this step of the process here.

Step Three: Balance and ReviewStep 3: Balance and Review
We presented a ranked list of topics to the Program Development Committee (PDC) of PCORI’s Board of Governors for consideration. To further narrow the list, we worked with the PDC and applied several “balancing” criteria, including populations affected and potential for meaningful improvement in patient health, well-being, or quality of care. These criteria were designed to ensure that the final list of topics was diverse in terms of the patient populations and diseases/conditions studied. This process yielded a short list of 11 topics.

From this list, we again reviewed the portfolio balance. We also utilized targeted outreach with clinical and research experts in each field, as well as continued discussion with the PDC and Board to evaluate the level of enthusiasm for a potential set of topics for our first targeted PFAs.

Step 4: Five Topics Identified and Reviewed

Step 4: Five Topics Identified and Reviewed
Based on these reviews and information-gathering processes, PCORI staff recommended three topics to the PDC for consideration as accelerated targeted PFAs:  Treatment Options for Uterine Fibroids; Treatment Options for Severe Asthma in African-Americans and Hispanics/Latinos; and Preventing Injuries From Falls in the Elderly. Particular emphasis was placed on opportunities to fund patient-centered studies that would otherwise not find adequate support. The Board approved these three topics and asked that two additional topics be identified. To identify the two additional topics, we again used the decision-analysis software. Incorporating some lessons learned from the first prioritization (see Step 2), we used a scale of one to five instead of one to nine and a narrower list of criteria that was recommended by pilot participants. Following this process, the Board selected two additional topics, Treatment Options for Back Pain and Obesity Treatment Options in Diverse Populations.

Step 5: Ad Hoc Workgroups and PFA Development ProcessStep 5: Ad Hoc Workgroups and PFA Development Process
To provide input to our staff and Board on the critical near-term research questions that should be answered in each topic, we will convene ad hoc workgroups to collect information from researchers, patients and other stakeholders. These workgroups are being established solely to assist in assessing the research topics selected for accelerated development of targeted PFAs with a goal that each workgroup will include between four to seven patients and four to seven other stakeholders. Potential workgroup members have been invited to participate and their names will be posted when the process is finalized. They are not the same as PCORI Advisory Panels, which will help with our longer-term process to establish targeted PFAs and for which we are now seeking applicants.

Workgroup meetings will be accessible through audio-conference, webcast, or other forms of communication. We will solicit additional public comments, suggestions and general input through our website for up to two weeks before, during, and for two weeks after each workgroup meeting. Records of the workgroups will be made public in accordance with PCORI’s policies and procedures. Read about the methodology for selection ad hoc workgroups and the accelerated PFA development process.

PCORI will synthesize input from the workgroup discussions and from broader healthcare community input to develop specific CER questions in each topic area. This information will be presented to our Board of Governors for consideration for PFA development. The Board will have final approval of each resulting PFA and determine the allocation of funding for each funding announcement. Read about the methodology for selection ad hoc workgroups and the accelerated PFA development process.


Posted February 8, 2013; Updated December 17, 2013