As a pediatrician, a research methodologist involved in patient centered and comparative
effectiveness research, and as Principal Investigator of one of seven AHRQ-CMS CHIPRA
Centers of Excellence, I read the PCORI Draft Priorities with great interest.
I endorse the need for developing an infrastructure to support patient centered research, both
outcomes and process research as outlined by PCORI and its statement of priorities. I share
concerns, as expressed by Academy Health, that the semantics and language in the Draft
Priorities fail to acknowledge adequately the role of parents and other caregivers. It is critical
that this agenda meet the needs of adults, children, and families.
I suggest modification to two other specific formulations: “1) ways to improve access to care,
receipt of care, coordination of care, self-care, and decision-making, 2) use of non-physician
healthcare providers, such as nurses and physician assistants, and the impact on patient
outcomes”
Quality of care has a variety of components ranging from access and availability, to technical
and interpersonal quality, to appropriateness of the medical decision making, to safety. I read
statement 1 to address some of these components and not others. My perspective considers
patient-centered care to be of high quality at its core and hence I urge a broadening of the
stated perspective to include the broader domains of quality and patient safety. I also believe
that patient centered outcomes research will often be grounded in studies of the structures and
processes of care.
I am puzzled by statement 2. We have witnessed a shift in care in favor of efficiency and cost
without close measurement of quality changes that have resulted. The shifting of RNs to
nurse’s aides may be as important to consider as shifts from physicians to physician assistants,
for example. My experience as the son of a 94 year old mother with significant chronic illnesses
suggests that quality and patient experience has suffered as work load has shifted from
registered nurses to aides and other less clinically trained personnel. Certainly she has
witnessed and complained of a degradation in the quality of patient centered care concomitant
with these changes. I would urge the consideration of an agenda that examines more generally
the impacts of various approaches to staffing and distribution of work on quality, patient
experience. I think these outcomes should be studied with an awareness of the potential that
there are false efficiencies to be achieved and that capacity may have intrinsic value to patients
and to the health care system. We should be open to whatever findings result.
I have several concerns that the methodological and communications agendas do not include
the full range of issues critical to patient centered outcomes research. For example
heterogeneity of effect is common and a source of the notion of personalized care. The funding
of research to develop and enhance methodologies to account for heterogeneity of effects is
critical. Such heterogeneity may represent issues of family or social context, of biological
heterogeneity, or caused by the presence of comorbidities, among other reasons. Meaningful
and useful patient centered outcomes research will require development of better methods to
analyze and communicate findings in the context of heterogeneity. The need to compare
treatments in real world contexts, including the presence of comorbidities, will define the patient
centered research agenda in the decade to come.
Comparative studies of outcomes often involve the indirect comparisons of effect size. That is,
treatment A is compared to a baseline treatment and Treatment B is compared to the same or
another baseline treatment in a different study and Treatments A and B are then compared.
Our group has presented both at Academy Health and at the Federal Committee on Statistical
Methodology to demonstrate that the treatment that appears to be preferable may depend upon
whether it is reported in terms of an odds ratio or a risk ratio. This is not well recognized and
the implications of this for interpreting and communicating findings in patient centered outcomes
research is critical. The methodological underpinnings of our PCORI research enterprise need
to be examined and enhanced in order to promote clear and accurate understanding of
research findings.
Our work in the AHRQ/CMS CHIPRA Center of Excellence has demonstrated to us the critical
importance of recognizing medicine as a clinical practice that is based upon an ever advancing
science. We believe that the implications of this include the need to specifically model
uncertainties so that the extent to which physician or patient preferences are consistent with
science can be explicitly understood and that deference to preference can be honored in those
areas, and that boundaries at which practice moves to underuse and/or overuse may be explicit
and understood. Attempts to use evidence to inform practice will always be limited by the
nature of the evidence. The intentional modeling of evidence, the limitations of evidence, and
the nature of clinical practice can support rational and practical approaches to improving
practice.
I appreciate the opportunity to comment on these draft priorities. I look forward to the
leadership of PCORI both to stimulate a national conversation on these issues and to move the
final agenda forward.
I strongly support the considerations of health equity and health disparities referred to in the
Draft Priorities.
Sincerely,
Larry Kleinman
Lawrence C. Kleinman, MD, MPH, FAAP
Vice Chair, Department of Health Evidence & Policy, Mount Sinai School of Medicine
Principal Investigator, Mount Sinai Collaboration for Advancing Pediatric Quality Measures
Adjunct Associate Professor, Department of Society, Health, and Human Development, Harvard
School of Public Health