Mazur and colleagues1 point out nicely that the Patient-Centered Outcomes Research Institute’s (PCORI’s) authorizing legislation defines a purpose and mission for PCORI that are distinct from those of other large funders of health research, including the National Institutes of Health (NIH). As they explain, PCORI’s unique role is to address research questions raised by patients, caregivers, clinicians, and other stakeholders in the health care arena, whereas a primary focus of the NIH is “to seek fundamental knowledge about the nature and behavior of living systems.”2 We agree with the authors that PCORI was intended and should be expected to differ from NIH in terms of the types of studies it funds and the researcher pool it engages.
To our knowledge, Mazur and colleagues are the first to publish a scientific paper analyzing data taken directly from PCORI’s Web site, pcori.org. We appreciate the authors’ interest in PCORI’s current research portfolio and their use of the Web site in conducting their research. The Web site is intended to provide a transparent and useful record of PCORI’s research and stakeholder engagement activities. We invite others to use this Web site to evaluate PCORI’s portfolio and performance. The usefulness of the Web site can only improve as a result of iterative use, feedback, and modification.
The authors’ findings suggest that PCORI has funded a broader, more diverse group of researchers, including more physicians, fewer PhD researchers, and more researchers who are neither MDs nor PhDs but hold other degrees including professional degrees. For PCORI awardees, the authors used Internet search engines to identify the highest degree of each principal investigator (PI). We looked into additional self-reported data provided to PCORI by applicants and found some differences from the distribution of degrees reported in the paper. For the same time frame (September 2011 through July 2014), 53% of all PCORI PIs reported holding an MD degree, with or without a PhD, compared with 61% reported by the authors; and another 43% held a PhD degree, compared with 38% found by the authors.1 Three percent held other doctoral degrees (in nursing, pharmacy, osteopathy, and other professional categories), and 1% (four awardees) had no doctoral-level degree. This additional information is on PCORI’s Web site, but not yet in an easily retrievable format. That can be fixed. Although the authors’ data were incomplete, their conclusion was correct: PCORI awardees are much more likely to hold an MD degree than are NIH PIs (53% vs. 33%) and are less likely to have a PhD only (43% vs. 67%). This reflects PCORI’s focus on questions representing clinical choices faced by patients and clinicians rather than on basic science questions, which are predominantly the domain of PhD scientists.
The broader, more diverse distribution of PCORI’s portfolio across the top 40 biomedical departments again reflects a greater diversity in PIs, and a greater focus at PCORI on clinical questions, on multidisciplinary research, and on questions that do not align with single body systems or conditions.
Among the 40 institutions most highly funded by NIH, PCORI’s funding distribution was almost identical to that seen for NIH’s R01 portfolio. As the authors suggest, this is not necessarily surprising. Highly successful, established research institutions possess infrastructure, mentors, and collaborators to support applicants that are not as available in newer or less experienced institutions. Nevertheless, nearly 50% of all PCORI funding went to institutions not in the top 40 recipients of NIH funding, and funding success rates at PCORI for these institutions are only moderately lower than for institutions in the top 40: 9% versus 14%. PCORI invests, through engagement awards and other mechanisms, to stimulate more and better applications from a broad range of institutions, within the constraints of rigorous merit review.
A central premise of Mazur and colleagues’ article seems to be that, because patients seek and receive more than 50% of care in primary care settings, PCORI funding should be proportionately directed to primary care departments and based in primary care settings. We have concerns with this assertion, with the authors’ definition of primary care patients, and possibly with their conception of primary care research.
Primary care patients are not only those patients drawn directly from primary care practices. Mazur and colleagues asked PCORI-funded PIs where they recruited participants for their studies. The responses they gathered, shown in Figure 4 of the article, include several other recruitment sites rich in primary care patients, including the community, online sources, electronic medical records and secondary data sources, and federally qualified health centers. Further examination of the studies with participants from these sites—even though they are not primary care practices—could well lead to the alternate conclusion that the large majority of PCORI-funded research focuses on primary care patients and the questions that matter to them and their primary care clinicians.
More generally, a patient who requires the attention of a specialist does not in our view cease being a primary care patient or cease to require the clinical prevention, care coordination, and advice that primary care provides. Further, we do not believe that primary care researchers should be excluded from conducting research on the clinical care of complex patients or that primary care research should be restricted to questions of a non-disease-specific nature.
To the extent that these views differ from those of Mazur and colleagues, the differences provide an opportunity to raise more fundamental questions about who and what we should expect to find in PCORI’s research portfolio and in the portfolios of primary care researchers. PCORI was not established primarily to study care delivery or primarily to fund a specific department’s research. Rather, as the authors note, it was created to address the questions of patients and other health care system stakeholders. Thus, the portfolio should reflect the highest-priority questions that patients encounter. Many of these questions, and particularly those that are comparative in nature, occur in patients with more complex illnesses, those who require specialty care, and those who face decisions about complex diagnostic and treatment procedures, novel or costly medications, or other complex therapies.
In our view, there is no reason why researchers based in primary care academic departments should constrain the scope of their research interests or why they should not lead creative applications on questions related to screening, diagnosis, and treatment of specific conditions such as coronary artery disease, diabetes, epilepsy, breast or prostate cancer, and hundreds of others. From PCORI’s perspective, leadership by researchers with a primary care perspective could help ensure that the research asks and answers practical questions that are relevant to broad patient populations and to the primary care clinicians who serve them. The findings of such research may also be more likely to be widely disseminated and implemented in primary care settings.
We applaud Mazur and colleagues for raising these questions about PCORI’s research portfolio and look forward to further conversations on how we can best meet our mission to fund comparative clinical effectiveness research of the highest priority to patients, caregivers, and clinicians. Our experience so far leads us to anticipate that diverse research teams, including teams led by primary care researchers and specialists, will continue to flourish at PCORI and that their work will benefit primary care, patients, and the research enterprise.
Acknowledgments: The authors thank Laura Forsythe, PhD, and Lori Frank for provision of data on Patient-Centered Outcomes Research Institute (PCORI) awards.