NPC Public Comment on the Patient-Centered Outcomes Research Institute National Priorities and Research Agenda

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The National Pharmaceutical Council (NPC) welcomes the opportunity to comment on the Patient-Centered Outcomes Research Institute Draft National Priorities for Research and the Research Agenda. NPC is a policy research organization dedicated to the advancement of good evidence and science, and to fostering an environment in the United States that supports medical innovation. Founded in 1953 and supported by the nation’s major research-based pharmaceutical companies, NPC focuses on research development, information dissemination, education, and promotion of the critical issues of evidence, innovation and the value of medicines for patients.

We recognize the importance of identifying the National Priorities and the Research Agenda, and the likely debate among patients, clinicians, purchasers and policymakers. We believe there are opportunities to incorporate both a broad, open solicitation of ideas with a focused research agenda composed of specific clinical questions. We look forward to future discussions and offer the following comments:

1) Please rate how well the five draft Priorities cover the comparative effectiveness research PCORI should do? (Very Well, Well, Fairly well, Not well, Not well at all)

  • Please rate how well “Assessment of Options for Prevention, Diagnosis, and Treatment” covers the comparative effectiveness research PCORI proposes to do. Blank
  • Please rate how well “Improving Healthcare Systems” covers the comparative effectiveness research PCORI proposes to do. Blank
  • Please rate how well “Communications and Dissemination Research” covers the comparative effectiveness research PCORI proposes to do. Blank
  • Please rate how well “Addressing Disparities” covers the comparative effectiveness research PCORI proposes to do. Blank
  • Please rate how well “Accelerating Patient-Centered Outcomes Research and Methodological Research” covers the comparative effectiveness research PCORI proposes to do. Blank

Other Comments:

Be specific based upon input received from stakeholders in a public process.

  • To enhance the likelihood that the most critical questions are pursued while ensuring an adequate portfolio of projects, we suggest being specific in setting research priorities and defining the research questions that will be funded. As outlined in Health Affairs (December 2011), different organizations take different approaches to specificity.i Some organizations such as the Centers for Medicare and Medicaid Services and the BlueCross BlueShield Association Technology Evaluation Center provide detail in their research priorities by delineating the condition or problem; specifying a patient population or end users; and specifying outcomes. By contrast, the World Health Organization and National Institutes of Health simply identify important areas and allow researchers to propose specific research projects. By defining a specific set of research priorities, the research will more closely align with the needs of specific stakeholders.
  • Further, not all outcomes of interest to patients may be answered in a single study or be sufficiently striking on its own to change practice. This creates the need for a thoughtful and well-orchestrated portfolio of priorities that can only be created through specificity and coordination.
  • Specificity also allows for greater collaboration with other research entities. NPC is pleased to see that PCORI will work “diligently to avoid redundancy and coordinate with other research entities” including public entities, private foundations, and the pharmaceutical, life sciences and health care industries. Without greater specificity, it will be difficult to determine not only where redundancy is occurring but also more importantly where synergies may exist.

2) How well would you say you understand of each of the five Priorities? (Very Well, Well, Fairly Well, Not well, Not well at all)

  • How well do you understand “Assessment of Options for Prevention, Diagnosis, and Treatment”? Blank
  • How well do you understand “Improving Healthcare Systems?”Blank
  • How well do you understand “Communications and Dissemination Research?”Blank
  • How well do you understand “Addressing Disparities?”Blank
  • How well do you understand “Accelerating Patient-Centered Outcomes Research and Methodological Research?” Blank

Please provide suggestions or questions that would help clarify the Priorities:

No comments

3) Please indicate what percentage of the PCORI comparative effectiveness research budget you think should be invested in each of the five Priorities and why?

  • Assessment of Options for Prevention, Diagnosis, and Treatment Blank
  • Improving Healthcare Systems Blank
  • Communications and Dissemination Research Blank
  • Addressing Disparities Blank
  • Accelerating Patient-Centered Outcomes Research and Methodological Research Blank

Please indicate the reason for your suggested budget:

In our review of existing CER entities, few organizations explicitly considered the health care delivery system. As PCORI seeks to contribute in a unique manner, we suggest increased funding towards improving health care systems. Funding research in health care systems would also enable cross-cutting research across the spectrum of health (e.g., prevention, diagnosis, and treatment) or across multiple conditions.

4) Please provide any other questions or comments on the National Priorities for Research.
Clearly delineate the prioritization criteria and characteristics to balance the final selection of projects in an open, transparent, and consensus manner.

How and when PCORI seeks to balance the portfolio of research based upon “characteristics of study populations such as age, gender, race/ethnicity, socio-economic status, and disease or conditions” will be important. These characteristics may also be weighed against the allocation of funds for rare and orphan diseases vs. more common illnesses, or across various types of research methodologies (e.g., systematic reviews or analyses of existing data sources vs. longer and higher cost studies such as randomized controlled trials). We propose a pre-specified quantitative approach to balance and blend research projects across the various criteria. Without an a priori and transparent process to provide balance, the research portfolio and the objectivity of the process may be questioned.

5) The National Priorities do not currently focus on diseases. Rather, they focus on types of research issues that address major challenges in health care delivery. In the future, portions of the Research Agenda may focus on specific conditions. If future versions of the Research Agenda focus on specific conditions and diseases, how should PCORI choose which ones to target? (check all that apply)

  • XX According to needs determined by stakeholders (e.g., patient groups, physician groups, etc.)
  • XX According to needs determined by previously funded research

Other comments:

As PCORI considers addressing specific conditions, it will be important to ensure that all stakeholders are engaged in all aspects of the prioritization process, including identifying and prioritizing among the research topics. PCORI has stated its desire for an active, two-way dialogue with the public, meaning public participation should extend beyond the topic submission into the prioritization of the research agenda. Most organizations reviewed in the Health Affairs analysis limited public input to topic identification.i To truly engage the public and decision-makers, we support the use of ad hoc panels to prioritize and identify specific research areas which have sufficient priority, are likely to influence care and have research success.

6) Please rate the degree to which you agree or disagree with the following statement: PCORI has an important role to play in new public health issues. (Strongly Agree, Agree, Neutral, Disagree, Strongly Disagree)

Neutral

7) How might PCORI engage in emerging public health issues?

We agree that there are many areas of interest in public health where additional evidence is needed; however, in many instances, existing public health entities provide this information. For example, PCORI could play a role in research to inform the optimal management of cardiovascular health, to increase physical activity, or to improve smoking cessation rates. Other public health issues such as new infectious diseases or ongoing cancer surveillance may best be addressed by the Centers for Disease Control or state or local health departments. Understanding how PCORI may partner with these organizations and optimize such research through data networks will become more apparent as research funding and plans are specified.

PCORI investments in research to optimally communicate and disseminate evidence will pay dividends for the public health to better inform both patients and clinicians. Research that encourages patient participation, awareness, or tools such as health coaching to promote adherence and appropriate self-care may be beneficial not only for PCORI, but for the nation’s health.

8) In the Affordable Care Act, PCORI is tasked with addressing rare and understudied diseases. Please suggest ways PCORI could address conditions that are rare and currently understudied? (check all that apply)

  • XX Provide additional research funding to organizations and researchers working on those conditions
  • XX Target a specific set of conditions (please tell PCORI what criteria you think should be used to select specific conditions in the comment box below)
  • XX Create research partnerships with organizations currently working on these conditions
  • XX Support electronic data sources where patients can securely volunteer their health data and the appropriate researchers can use it to answer PCORI research questions on rare conditions

Other comments:

Many have suggested a blended approach that considers not only obvious high-burden conditions but also under-addressed topics, using either weighting or a pre-specified percentage of allocated funds.iii, iv

9) The five priorities are: Assessment of Options for Prevention, Diagnosis, and Treatment; Improving Health Care Systems; Communication and Dissemination Research; Addressing Disparities; and Accelerating Patient-Centered Outcomes Research and Methodological Research. What do you suggest might be important Research Agenda topics that should be included in any of the five priorities?

No comment

10) Please provide any other questions or comments on the Research Agenda.

  • Comparative Assessment for Options for Prevention, Diagnosis, and Treatment. PCORI plans to understand "all outcomes that may be important to patients." Since there will likely be many outcomes of interest and a single study is unlikely to be able to address all of them, multiple studies on a topic will be needed. Without greater specificity in PCORI’s agenda, multiple studies on a particular topic may not be funded, leading to important and unaddressed patient outcomes. Alternatively, specification of desired research questions with funding of multiple studies would more likely gain the complete set of information needed by patients.

    We suggest expanding patient characteristics considered when evaluating heterogeneity to include genetic, cultural, or co-morbid conditions. Researchers and other stakeholders are more likely to consider, pre-specify, and analyze patient characteristics if they are outlined in the actual research agenda.
  • Improving Healthcare Systems. We suggest that further clarification is needed on supporting and improving patient access to care. Does this include access to providers and health settings, or broader reimbursement and payment programs?
  • Communication and Dissemination Research. In addition to research that aims to improve shared decision-making and patient awareness, we recommend that research include areas such as health coaching and health literacy to improve patient actualization and receptivity.
  • PCORI Research Criteria. We recommend clarifying the intent of “will more information in this priority area help improve health care treatment or get better health outcomes for the money invested.” While we agree understanding utilization (days of hospitalization, visits, services and treatments) is important, this could be misinterpreted by others to include cost-effectiveness analysis.
  • Addressing Disparities. We recommend expanding the factors associated with disparities in health status and health care to include cultural differences. Without such awareness and recognition of cultural differences, additional funds may not overcome ingrained cultural barriers.
  • Appendix.It is unclear how the "Features of PCORI Research Agenda" will be used or emphasized in the selection of research. If this is part of the selection criteria for research, this should be specified and communicated in terms so that the public and researchers will understand how this will be assessed.

We offer these suggestions as a way to enhance the process to develop national priorities and the research agenda. In particular, signaling PCORI’s specificity will benefit PCORI, other organizations seeking collaboration, and the public. We propose engaging stakeholders and ad hoc panels in the specification of research projects, and outlining the process to “balance” the research portfolio in an a priori and transparent manner. The National Pharmaceutical Council looks forward to further dialogue and engagement with the PCORI Board, Methodology Committee and staff.

Thank you.

i  Dubois RW, Graff JS. Setting Priorities for Comparative Effectiveness Research:  From Assessing Public Health Benefits to Being Open With the Public. Health Aff (Millwood). 2011;30(12):2235-2242.
ii Appendix. Summary Chart of CER Prioritization Strategies. Dubois RW, Graff JS. Health Aff (Millwood).  2011;30(12).  Online at [http://content.healthaffairs.org/content/suppl/2011/12/05/30.12.2235.DC1/2011-0136_Dubois_Appendix.pdf]   Accessed March 9, 2012.
iii Donaldson MS, Sox HC, editors. Setting priorities for health technology assessment: a model process. Washington (DC): National Academies Press; 1992.
iv Mitton C, Donaldson C. Health care priority setting: principles, practice, and challenges. Cost Eff Resour Alloc. 2004;2(1):3.