The Patient-Centered Outcomes Research Institute (PCORI) was established with the passing of the Affordable Care Act in 2010 to oversee and set guidelines for comparative effectiveness research (CER). PCORI was also designed with a sustainable funding model, having received $210 million from 2010 to 2012 and expected appropriations of $150 million annually via fees imposed on Medicare and private health insurance companies. The international comparative effectiveness research community has recognized PCORI as the most funded CER initiative in existence, far surpassing the annual budgets of comparable international programs such as U.K.’s National Institute for Health and Clinical Excellence (NICE).
Despite the considerable resources designated to PCORI, its future remains tenuous. For one, the ACA legislation also created a “sunset date” of September 2019, giving PCORI seven years to convince Congress to reauthorize funding for the institute. Further, legislative efforts have already been made to repeal PCORI; H.R. 3827 was introduced by Rep. Brett Guthrie (R-KY) in January 2012, asking for immediate elimination of PCORI.
While PCORI is the first national comparativeness effectiveness research institute to be established in the U.S., it fortunately can draw several lessons from its international counterparts which have been operating for two decades. The U.K.’s NICE, considered by most to be the most comprehensive and effective CER program, will be the main comparator for this analysis.
PCORI must address two themes that are at tension with one another. On one hand, PCORI needs to foster stakeholder involvement in order to establish itself as a legitimate body of CER knowledge that is relevant and accepted in clinical practice. However, it also must avoid the pitfalls of having a democratic, iterative process that moves too slowly to issue findings and recommendations in a timely manner.
In a recent Health Affairs Blog post, Joe Selby, the executive director of PCORI, outlines the various measures PCORI is taking to further its mission. In particular, he details the meaningful steps already made to promote engagement among patients and physicians such as the development of patient-centered research methods. However, there are additional strategies described below that the research institute can implement to maximize stakeholder involvement. Further, PCORI needs to ensure it can expeditiously demonstrate its usefulness. This second point is particularly crucial given the need to convince its relevance to Congress by the 2019 sunset date.
Fostering Stakeholder Involvement
Stakeholder buy-in and involvement is an integral component for ensuring the relevance, acceptance, transparency, and legitimacy of comparativeness effectiveness research. PCORI was designed to be independent from government, though measures to uphold transparency and engage all stakeholders do not end there. Prior to selecting national research priorities and disbursing major research funding, PCORI has public comment periods for stakeholders to identify what they consider to be the relevant research areas worth addressing. Further, a public comment period will also occur after PCORI releases any findings or issues any guidelines.
Designing an Independent Research Entity that Involves Providers
Many of PCORI’s strategies to encourage wide stakeholder buy-in and to secure its own legitimacy parallel the steps NICE took during its own inception. For one, NICE also acts as an independent body of the National Health Service (NHS), with advisory bodies comprised of clinicians, professional groups, academics, and patient and public representatives, all of which are independent from NICE.
NICE’s independent arm’s-length-from-government design appears to be a positive predictor for a national CER program’s success. Specifically, the in-house research capabilities of NICE are very limited, requiring outsourcing of health technology assessments to academic institutions and professional groups. By relying on the existing academic research infrastructure to complete assessments and make guidelines, provider involvement is built into the CER process and buy-in from providers and patients becomes more manageable.
Germany’s Institute for Quality and Efficiency in Health Care (IQWiG) provides an illustrative contrast. IQWiG has a very large in-house research capacity and, in turn, performs most of its own CER with limited dissemination of research to academic institutions. This design may explain, in part, limited provider buy-in of IQWiG and clinical guidelines. Only a minority of German physicians report using clinical guidelines.
Given PCORI has funded 84 academic investigators in 23 states in its most recent funding cycle, the institute appears to be incorporating provider involvement into the research process. Like NICE, this design will encourage wider stakeholder involvement and help in securing the legitimacy of PCORI’s future findings and guidelines.
Providing Channels to Involve Industry
NICE has channels that provide a comprehensive system of addressing stakeholder concerns and demands. Through the Partners’ Council and a clearly delineated appeals process, NICE is able to increase transparency and acceptance as well as leverage the benefits of wider stakeholder involvement.
The Partners’ Council is operated by NICE as a channel to engage with industry, NHS managers, and clinicians to discuss the strategic challenges of the institute as well as to inform them of current and future comparative effectiveness research priorities. A particular strength of the Partners’ Council is that it has led NICE to work increasingly with industry to outline evidence requirements and scope assessments. For example, if a particular health technology is found to have inconclusive or insufficient evidence from a comparative effectiveness analysis, NICE will often make the recommendation that the examined therapy be used only in research.
This recommendation often incentivizes pharmaceutical and device manufacturers to partake in new clinical trials and observational studies in order to accumulate enough evidence to change NICE’s recommendation. Incentivizing industry to contribute to comparative effectiveness research has the benefits of involving industry directly in the comparative effectiveness research process as well as adding to the body of evidence.
As it is currently designed, PCORI seems ill-prepared to handle the input it will receive from industry, particularly when PCORI will issue comparative effectiveness analyses that shed an unfavorable light on specific technologies. Industry players may attempt to undermine the fledgling PCORI’s legitimacy if the institute issues findings that go against their particular technologies. By having an established appeals process and issuing recommendations that call for more evidence on unproven therapies, PCORI could mitigate industry opposition to their findings and incentivize larger industry involvement in the CER process.
In addition to NICE, PCORI has a domestic example it can draw upon to establish constructive industry-oriented engagement. CMS has an analogous approach in which it grants Medicare coverage with an evidence requirement, meaning a therapy will be funded on the condition that additional studies of its effectiveness are conducted.
Making PCORI’s Findings Accessible to all Stakeholders
One of PCORI’s main goals is to be a patient-focused institute that presents findings and guidelines to be used by providers and patients in personal decision making about medical care. In order to achieve these ends, PCORI must ensure that, when disseminating its findings, its context and language are accessible to all stakeholders.
NICE determined early that one report of findings will not meet the needs of all stakeholders and now routinely generates three communications: a technical report, a summary report, and information for patients. Further, NICE established the Patient and Public Information Programme, which sets editorial guidelines for the outside academic researchers performing the research, ensuring the institute’s decisions are communicated to patients in plain language. PCORI should take similar steps, establishing an entity like NICE’s to set editorial guidelines and ensure the information it generates is relevant and transparent to a wide set of stakeholders.
In recent years, NICE has also begun communicating in its reports the generalizability of its findings in different settings. This came along due to increased stakeholder criticism that NICE’s conventional research methods only reported findings for the “population average” and did not factor for individual patients. Hence, NICE began reporting generalizability of results as well as tailoring some recommendations based on population subgroups (e.g. age, gender, race, severity of disease). This is a stepping away from conventional CER methods and a move towards patient-centered outcomes research. PCORI would be wise to take a similar approach, with two key goals in mind.
The first goal is to more clearly delineate recommendations that should be applied for specific patients, making the interpretability and clinical application of findings more discernible for providers and patients. The second is to mitigate a common criticism brought up against other national CER programs and by the current opponents of PCORI; namely, that CER is not relevant in clinical management because it does not factor in the particular aspects of individual patients. As some CER experts have noted, a “one-size-fits-all” approach is particularly not preferable in the U.S., given its diverse population and practice settings.
Timeliness: Side-Stepping the Pitfalls of Wide Stakeholder Involvement
Many experts believe the strategies PCORI has implemented to foster wide stakeholder involvement will be effective in the long-term. However, there is growing concern that these strategies may be slow in showing meaningful results and are not appropriately responsive to the urgent circumstances imposed by the 2019 funding sunset date. Therefore, complementary strategies should be employed to make sure that stakeholders’ participation at each point in the CER pipeline does not bog down the process. In order for PCORI to quickly demonstrate its importance, high-impact problems must be studied and solved in a timely manner.
Collaborating with Accountable Care Organizations
An innovative approach to producing timely, high-impact evidence would be to engage payers and providers participating in accountable care organizations (ACOs) to implement PCORI’s findings into their clinical management decisions. By collaborating with ACOs, PCORI can ensure that a subset of clinical decision makers will translate their recommendations to clinical practice. PCORI can then follow this subset of recruited ACOs, track the clinical outcomes before and after they begin following their recommendations, and compare outcomes with non-participating ACOs and providers.
Under this scenario, PCORI need only issue a few key recommendations before the sunset date. PCORI would track the outcomes related to its issued recommendations for the recruited ACOs, compare them to the outcomes of non-participating providers, and then directly measure the clinical impact of the evidence generated. A few key successes would provide strong evidence for the institute’s relevance when it is assessed for reauthorization by Congress in 2019.
To be sure, if PCORI were to enlist ACOs to implement its recommendations, it would have to be careful to not overstep the limitations laid out by the ACA legislation. In particular, the ACA specifically prohibits using PCORI’s findings to dictate coverage, reimbursement rates, or clinical guidelines for federal health programs. The legislation also specifically prohibits cost-effectiveness studies and use of specific cost-effectiveness instruments like quality-adjusted life-years (QALYs).
Clearly, PCORI cannot simply mandate that providers or payers translate their recommendations into clinical practice. But with a tactful approach, it can work collaboratively with payers, ACOs, or both without being perceived as a governmental body dictating coverage and clinical decisions.
For example, in launching the Pioneer ACO Model initiative, CMS identified providers deemed to be already successful at coordinating care for patients and enrolled them to experiment with the Medicare Shared Savings Program. Most of these providers perceived joining the Pioneer ACO Model as a mark of distinction and collaborated with CMS’ initiative. PCORI could identify a similar group of providers that has already shown success in using evidence-based medicine and clinical practice guidelines and enroll them in a program to implement PCORI recommendations.
The recommendations offered above are pragmatic steps the institute can take to ensure timely results and necessary stakeholder involvement. Nonetheless, PCORI has the considerable challenge of establishing itself as a legitimate national comparative effectiveness research program in six years before it comes to Congressional reauthorization in 2019. The upcoming years will be a watershed period for the future of comparative effectiveness research in the U.S. Hopefully, PCORI can harness this opportunity to become a national clearinghouse of CER used by patients and providers alike and a vehicle to improve quality of care in the U.S.