PCORI Response
General Comments
Comparative effectiveness research (CER) is crucial to providing critical information health care
consumers, providers and payers need to make informed choices. While PCORI is statutorily
forbidden from assessing cost, CER should provide the information necessary to allow patients
and purchasers to evaluate the cost-effectiveness of treatment options. Without this
component of transparency, preferences and decision making may be incomplete.
CER should consider a range of comparisons to maximize transparency and inform a potentially
wide range of patient preferences. First, CER should not be limited to the comparison of two or
more treatments. It should also assess the option of "doing nothing.” Second, CER should
compare all treatment modalities in a head-to-head comparison. Research should not be
limited to medical-to-medical or surgical-to-surgical outcomes. Third, CER should compare
complications with the expected benefits. For example, a treatment that results in a successful
outcome also may be associated with the risk of serious side effects. Consequently, patients
may choose another, albeit less effective, treatment, to reduce their risk of serious side effects.
Public health is an essential component to improving individual outcomes and population
health status. CER should focus on public health impacts of individual decisions related to
health outcomes. For example, educating patients about how their individual decision to
forego immunization may impact public health, or creating informed decision-making
documents to address such outcomes.
Research Priorities
1. Assessment of Prevention, Diagnosis, and Treatment Options.

CER should study how treatment variation impacts clinical outcomes. Specifically,
treatment protocols for cancer care differ significantly. This type of CER could
enhance understanding of optimal care and identify treatments that improve quality
of life and increase survival among cancer patients.

This research priority is highly dependent upon Priority #5 (Accelerating PatientCentered and Methodological Research). While useful in evaluating prevention,
diagnosis and treatment protocols, clinical information is difficult to obtain for some
who pay for health care and sometimes is unreliable. Further, techniques for
appropriately collecting and disseminating information (e.g., registries) would result
in greater objectivity and defensibility.
2. Improving Healthcare Systems.

The impact of non-physician clinicians on patient outcomes should be assessed.
Particularly for primary care because of the national shortage of primary care staff
and the potential for greater coordination. As the most easily accessible health care
professional, pharmacists play key roles in delivering regular care to individuals,
especially to those with chronic conditions. Research should also encompass the role
of pharmacists and their impact on patient outcomes.

CER should focus on alternative “models” of care delivery and the effectiveness of
those models. Examples include telehealth to improve patient outcomes when
integrated provider networks share in financial risk, based on quality and
performance metrics.

CER should study the impact of reducing hospital re-admissions and emergency
room visits on outcomes.

CER should study the impact of practitioners sharing medical records for their
common patients, including the impact of electronic health record systems, on
outcomes.

CER should study care settings and locations of service with the goal of determining
if there is any variation in outcomes/quality.

CER should assess the impact of greater care coordination, patient education, as well
as, monitoring and evaluation by primary care physicians on health outcomes.

There should be assessments of the impact of defensive medicine on health
outcomes. Although much is discussed regarding the misuse and underuse of
medicine, overuse can also lead to adverse outcomes.
3. Communication and Dissemination.

Patient and patient advocate empowerment should be assessed as it relates to
patient outcomes. This should include concepts regarding cultural competence.
There needs to be comparative information on what type of communication and
education techniques result in improved outcomes and empower patients, and the
best way to teach those techniques to all providers.

CER should study ways to provide patients with greater transparency and decisionmaking support regarding lifestyle choices that can lead to improved health and
outcomes.

CER should study pathway compliance for Oncology and Cardiology protocols,
especially regarding reimbursement-based decisions versus clinical data-based
decisions related to specific oncology care and cardiac procedures such as Stents.
4. Addressing Disparities.

CER should compare various efforts to address social, environmental, as well as
clinical barriers to health and quality health care. There should be an assessment of
which efforts would have the largest impact on a marginal basis. These efforts
cannot focus on incidence or prevalence alone.

CER should focus on healthcare specific to populations and diseases, especially when
treatment options and approaches differ among certain groups and regions.

CER should research the consequences of reducing disparities for selected
populations and implications for the entire population, especially the potential to
“free up” resources and access needed to improve patient outcomes for all
populations.

There are various techniques regarding cultural competence that have shown
promise of impacting health care disparities, but there is a reluctance to invest
heavily in the concept, particularly in the private sector. There needs to be rigorous
scientific study of the most promising concepts. And there needs to be assessment
of the best way to teach those concepts to the health care workforces in a way that
translates into improved outcomes.
5. Accelerating Patient-Centered and Methodological Research.

CER should consider the relationship between improved outcomes, patient privacy
and individual rights and responsibilities.

CER should focus on defining specific data elements to improve outcomes, not just
process or administrative activities. For example, CER should identify discrete
actions patients and practitioners can take that improve outcomes.
Additional Areas for Comment:
1. Specificity of Research Agenda.
 PCORI should declare whether its research agenda is limited to address only the
delivery of health care in the United States. For example, PCORI should indicate
whether its scope of research is limited to medications approved by the Food
and Drug Administration. PCORI should advise whether its research will include
foreign providers or alternative therapies and services available only in foreign
countries. Because of need, developing countries have found innovative ways to
deliver care (See McKinsey Quarterly, The Emerging Market in Health Care
Innovation, May 2010). These alternatives should be considered.

PCORI priorities should consider where the greatest impact can be made in the
shortest time span. Although not intending to exclude any diseases or
conditions, PCORI should identify areas of greatest concern and specific actions
to be taken.

CER should be specific in order to draw reasonable conclusions. PCORIsponsored research specifically should fund studies examining childhood obesity,
cancer trends and treatments, and the bacterial and viral resistance of infectious
disease.
2. Allocation of Funding.
 PCORI should employ guiding principles to determine allocation of funding.
Principles include, but are not limited to, the uniqueness of the research
question, public health impact posed by the research question and overall
quality of the research question.

PCORI should place great weight on improving healthcare systems and
communication and dissemination. Practitioners have and maintain a depth and
breadth of knowledge, but the need is greatest in efforts to coordinate,
integrate, and communicate this knowledge to patients, payers, and other
healthcare stakeholders.
3. Avoiding Redundancy of Efforts.
 PCORI should work with and leverage the work of more well-established patientcentered medical home organizations and disease registries.

PCORI should also work with foundations so that both sectors can leverage each
other’s efforts. This could be efficiently done through interaction with
philanthropic networks at the state and federal level. While large, national
philanthropic organizations have fantastic initiatives, there is a rich source of
information and experimentation at the state and local levels that are funded by
more geographically focused foundations.
4. Criteria for Funding Decisions.
 PCORI should support multiple studies on the same or similar topic while
minimizing research question redundancies. Research methods vary and
different approaches to a research question could enhance our overall
understanding of the issue and benefit overall public health.

PCORI’s research should be more short-term focused, because of the rapidly
changing capability of medical technology and advances in care delivery
methods.