Patient Centered Outcomes Research Institutes National Priorities for Research and Research Agenda – Laying a Foundation for Comparative Effectiveness Research
One of the bigger pieces of the Patient Protection and Affordable Care Act (PPACA) was the idea to lower healthcare costs through comparative effectiveness research (CER). To achieve this goal, PPACA (Section 6301 and Section 10602, Public Law 111-148), created the Patient-Centered Outcomes Research Institute (PCORI), an independent, non-profit health research organization. PCORI will have an estimated $3 billion over the next decade to fund CER.
PCORI was created to conduct research to provide information about the best available evidence to help patients and their health care providers make more informed decisions. PCORI’s research is intended to give patients a better understanding of the prevention, treatment and care options available, and the science that supports those options. Its mission is to fund research that offers patients and caregivers the information they need to make important healthcare decisions. PCORI focuses on CER that compares options for preventing disease and providing treatment and care by:
- Identifying national priorities for research.
- Creating a research agenda based on identified priorities.
- Funding research consistent with these priorities and agenda.
- Providing patients and their caregivers with useful research information.
Consequently, PCORI recently issued its draft list of National Priorities for Research and Research Agenda. PCORI will provide a 53-day public comment period on its first National Priorities for Research and Research Agenda. The comment period begins on Monday, January 23, 2012 and concludes at 11:59 p.m. ET on Thursday, March 15, 2012.
Responses received through www.pcori.org will be displayed for public view on the website. Click here to fill out the online form to provide comments on the draft priorities and research agenda. Public comments can also be mailed to:
Patient-Centered Outcomes Research Institute
1701 Pennsylvania Ave. NW,
Washington, DC 20006
PCORI will also hold a National Patient and Stakeholder Dialogue on Monday, February 27, 2012, from 10:00 a.m.-5:00 p.m. EST, at the National Press Club in Washington, D.C. The event will dedicate three and a half hours to receiving public comment, and include presentations by PCORI and a roundtable discussion involving patient advocates, clinicians and others from the health care community. Individuals may attend in person or remotely via webcast and teleconference.
PCORI will conduct additional forums in cities across the United States, which will include focus groups, involving patients, caregivers and clinicians, to obtain and incorporate more public input and feedback on the draft national priorities for research and research agenda.
After incorporating public input, the first National Priorities for Research and Research Agenda will be adopted by the PCORI Board of Governors before primary research funding announcements are issued. A report will be published on www.pcori.org that summarizes the input received with an explanation of how the input led to any changes in the draft priorities and agenda. The revised National Priorities for Research and Research Agenda will be adopted by PCORI’s Board of Governors during a special public meeting in April.
Subsequent priorities and agendas will be achieved through a “due diligence” process that includes dialogue with a broad range of stakeholders, input through a formal public comment process and additional forums, including focus groups, PCORI presentations to various audiences, outreach through PCORI’s website and other vehicles, advisory panels and stakeholder meetings.
Nine different Priority Setting Organizations contributed to the formulation of PCORI’s draft research priorities/agenda, including AHRQ, IOM, National Quality Forum; and HHS; many of which received significant public input. The priorities and agenda will inform the development of PCORI’s first primary research funding announcements, which will be made in May. "These priorities and agenda give a framework to and identify the broad questions that must be addressed so that patients can make better and more personalized decisions in partnership with their clinicians across all areas of health," said PCORI Executive Director Joe Selby, MD, MPH.
The institute plans to announce an initial round of about 40 grants, drawn from 856 applications, in May. A second round of grants, for projects linked to the research agenda, will be issued in December, he said. PCORI is a learning organization. Once the initial priorities and agenda are adopted, PCORI will work closely and continuously with all stakeholders to refine the agenda and to identify specific areas and questions where PCORI’s research can have the greatest impact.
This process will include analysis of PCORI’s Pilot Projects Grants Program and the applications in response to PCORI’s first primary research funding announcements. Any future revisions to the priorities and research agenda will include a formal public comment process and additional forums.
However, Robert Dubois, chief science officer for the National Pharmaceutical Council noted that, if PCORI gets “1,000 grants submitted on 400 different topics, who is going to make the decision on, say, whether they should fund a project on low back pain, but not one on multiple sclerosis?” Dubois says the thematic approach taken by PCORI will lend itself to one of the institutes’ goals – creating a self-sustaining and robust research environment. But he says a portion of the funding should be directed “toward actual, unanswered clinical questions.”
Additional challenges facing such research also include political controversy and the difficulty of translating research findings into changes in medical practice. Republican opponents of the law say the institute will lead to government-directed rationing as it judges treatments. Representative Thaddeus McCotter, a Michigan Republican, has introduced legislation to eliminate the agency.
Over time, more money should be shifted to comparing treatments, said U.S. Representative Kurt Schrader, an Oregon Democrat who wrote legislation to create a comparative effectiveness research agency in 2009. The provision of the health law that created Selby’s institute echoes Schrader’s bill, which had some Republican support at the time. The research plan is “a little broader in scope” than he envisioned when he wrote his bill, Schrader said in a telephone interview. “The worry I would have is they are not going to get down to specific projects.”
Ten CER priorities areas from existing literature were identified. The first five priority areas (prevention, acute care, care coordination, chronic disease care, and palliative care) encompass the complete health cycle from staying healthy to treating conditions to reducing pain and suffering. The second five (patient engagement, safety, overuse, information technology (IT) infrastructure, and the impact of new technology) include issues that are systemic in nature to healthcare. All but one of the 10 priorities (impact of new technology) appear in at least three of the seven processes. Prevention appears in all seven and patient engagement in six.
Research List and Agenda
PCORI prioritized five research areas for patient-centered comparative clinical effectiveness research:
Comparative Assessments of Prevention, Diagnosis, and Treatment Options. The research goal is to determine which option(s) work best for distinct populations with specific health problems. Comparing the effectiveness and safety of alternative prevention, diagnosis, and treatment options to see which ones work best for different people with a particular health problem. 40% of funding.
Improving Healthcare Systems. Focuses on ways to improve healthcare services, such as the coordination of care for patients with multiple chronic conditions. Comparing health system-level approaches to improving access, supporting patient self-care, innovative use of health information technology, coordinating care for complex conditions, and deploying workforce effectively. 20% of funding.
Communication and Dissemination. Looks at ways to provide information to patients so that they, in turn, can make informed healthcare decisions with clinicians. Comparing approaches to providing comparative effectiveness research information and supporting shared decision-making between patients and their providers. 10% of funding.
Addressing Disparities. Assures that research addresses the healthcare needs of all patient populations. This is needed as treatments may not work equally well for everyone. Identifying potential differences in prevention, diagnosis or treatment effectiveness, or preferred clinical outcomes across patient populations and the healthcare required to achieve best outcomes in each population. 10% of funding.
Accelerating Patient-Centered and Methodological Research. Includes patients and caregivers in the design of research that is quick, safe, and efficient. Improving the nation’s capacity to conduct patient-centered outcomes research, by building data infrastructure, improving analytic methods, and training researchers, patients and other stakeholders to participate in this research. 20% of funding.
PCORI’s Proposed Research Agenda
Comparisons of Prevention, Diagnosis, and Treatment Options. Research should focus on 1) clinical options with emphasis on patient preferences and decision-making, 2) biological, clinical, social, economic, and geographic factors that may affect patient outcomes.
Improving Healthcare Systems. Research should focus on 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, 3) system-level changes affecting all populations, diseases, and health conditions.
Communication and Dissemination. Research should focus on 1) strategies to improve patient and clinician knowledge about prevention, diagnosis and treatment options, 2) methods to increase patient participation in care and decision-making and the impact on health outcomes, 3) communication tools that enhance decision-making and achieve desired outcomes, 4) ways to use electronic data (“e-health records”) to support decision-making, 5) best practices for sharing research results.
Addressing Disparities. Research should focus on 1) ways to reduce disparities in health outcomes, 2) benefits and risks of healthcare options across populations, 3) strategies to address healthcare barriers that can affect patient preferences and outcomes.
Accelerating Patient-Centered and Methodological Research. Research should focus on 1) ways to improve the quality and usefulness of clinical data in follow-up studies, 2) methods to combine and analyze clinical data that follow patients over time, 3) use of registries and clinical data networks to support research about patient-centered outcomes, including rare diseases, 4) strategies to train researchers and enable patients and caregivers to participate in patient-centered outcomes research.
Establishing the Scope of the Research Agenda
By design, the agenda did not cover all the activities that PCORI sees as part of its mission. For instance, it does not describe how PCORI intends to invest in efforts to maintain active engagement with patients and all other stakeholder groups over time. It does not describe how PCORI, in partnership with the Agency for Healthcare Research and Quality (AHRQ) and the U.S. Department of Health and Human Services (HHS), will invest in efforts to disseminate research findings or build capacity for patient-centered outcomes research through training programs or infrastructure development.
This document is the foundation upon which the first set of PCORI funding announcements will be developed. Taken together, the series of funding announcements that PCORI will produce, beginning in mid-2012, will create a portfolio of research that builds from this agenda and is consistent with Congress’ intended purpose.
The Research Agenda by design did not specify a restricted set of conditions or treatments that PCORI will study. Although some previous prioritization efforts have focused on specific high-prevalence or high-cost conditions, PCORI’s priorities and agenda do not place such limits on the scope of research that will be supported.
Rather than focusing on specific conditions or treatments, PCORI’s initial approach specifies a set of questions and topics PCORI believes are most in need of attention – across a range of conditions and treatments. Within any topical area, studies may focus on specific diseases, conditions and interventions, or they may be cross-cutting, including broader study populations or examining interventions or other questions that apply across multiple diseases and conditions.
This approach recognizes the likelihood that as PCORI begins its funding program, researchers partnered with stakeholders are well-positioned to present a range of compelling questions. Ultimately, decisions about funding will depend on the quality of applications – with special attention to the likelihood that the research may lead to improvement in patient outcomes, as determined by alignment with PCORI criteria.
Targeted opportunities may focus on specific conditions or diseases, treatment modalities, outcomes or on themes that are cross-cutting. PCORI will work diligently to avoid redundancy, coordinate with other research entities, and will seek opportunities for collaboration with these entities.
The portfolio of research supported by PCORI will be balanced based upon the characteristics of study populations such as age, gender, race/ethnicity, socio-economic status, and disease or conditions. PCORI has a commitment to include studies of patients with rare conditions as well as those with more common illnesses. Especially needed are studies to improve care and outcomes for patients faced with multiple conditions. All funded studies will have a strong orientation to the patient perspective and all will have patients involved in the development of the research, its governance and oversight, and its dissemination strategy.
The Research Agenda does not specify preferred study designs or analytic approaches. Instead, it recognizes that various PCOR questions might be investigated by a variety of scientifically-valid methods and approaches. In accordance with Act (Section “(C) FUNCTIONS”), research focused on developing new methods and/or improving the science and methods of patient-centered outcomes research (PCOR) is specifically included in the Agenda. PCORI’s Methodology Committee is currently developing standards for the design and conduct of different types of PCOR. These standards will be disseminated and used in the evaluation of PCORI applications.
Overall the selection of topics by PCORI shows that they understand that in order to successfully transform the health care system, one has to start at the foundation and ask the big questions. Much of the focus of PCORI support and criticisms has been focused on PCORI studying treatments, but if the system is inefficient, then studying treatments may not necessarily be the best use of public funds.
If one really gives thought to the problem subjects such as compliance, socio economic access to therapies and social customs such as men delaying seeing a physician play a much larger role in the outcomes of patients than the treatments themselves.