Comparative effectiveness and patient-centered outcomes research: enhancing uptake and use by patients, clinicians and payers

There is a growing consensus among stakeholders in the USA that we must shift the emphasis of the healthcare system from one based on volume to one based on value. To guide value-based decision making, patients, caregivers, clinicians and policy makers need access to, and the ability to use evidence that compares the relative merits of available treatment options, and be adequate for their decision-making needs.

Comparative effectiveness research (CER), which has gained attention over the past decade, offers part of the solution. The purpose of CER is to facilitate decision-making and improve health outcomes by developing and disseminating evidence to patients, clinicians and other decision makers, about which interventions are most effective for a given application. CER emphasizes understanding the comparative benefits and harms of treatments as used in actual clinical practice. This evidence is increasingly seen as a building block for shared decision-making that takes into account the trade-offs between the benefits and the potential harms of treatment options as viewed by the patient and their families, as well as healthcare professionals.

Early efforts to support CER focused primarily on the development of research methods and on the training of researchers. For example, as early as 2006, the Agency for Healthcare Research and Quality (AHRQ)  held conferences about methods in CER and published related resources [1–3]. With the advent of ‘patient-centered outcomes research’ (PCOR) and the Patient-Centered Outcomes Research Institute (PCORI) in 2010, such efforts expanded [4]. Importantly, funding to train CER researchers was made available by AHRQ, PCORI, the NIH, the Pharmaceutical Research and Manufacturers Association (PhRMA) Foundation, and other organizations in the form of individual and institutional training awards, conference grants, center grants and contracts. The momentum behind PCOR was a logical consequence of CER, introducing another facet to understanding value that recognized studies about group-based (average) treatment benefits are inadequate to guide clinical decision making at the individual level. Not only do we seek to understand clinical heterogeneity so that treatment effectiveness can be better tailored to individual characteristics, but the patient's perspective – their preferences for different treatments – are a crucial part of the value proposition in healthcare.

Today, we have significant resources and expertise in the conduct of CER and PCOR. As a result, CER/PCOR evidence is rapidly accumulating in the literature. However, there remains a need to better integrate CER/PCOR evidence into decision making [5–9]. A major limitation may be that potential users of this evidence need to be educated on the strengths and weaknesses of CER and its place within the hierarchy of evidence levels. A recent survey identified user application of CER and its role in decision-making among the top educational needs [5]. Other surveys reported that the majority of healthcare professionals were not adequately prepared to use CER/PCOR [7]. Others have noted that insufficient attention is paid to communication about CER/PCOR evidence to end users [6,8]. Together, studies point to the need for better education of users of CER/PCOR, understanding of the barriers for using CER/PCOR evidence, design of effective strategies and tools to ensure uptake and use of CER/PCOR by clinicians, patients, payers/policy makers. Last, there is a need to teach patients, payers and policy makers how to use CER results to achieve value when selecting among healthcare products and services.

To begin to address these issues, the PhRMA Foundation, together with the Academy of Managed Care Pharmacy, hosted an invitational conference in Washington, DC, on 26–27 January 2017. Attended by 70 experts and opinion leaders representing clinicians, patients, funding agencies and payers – the conference was titled “Comparative Effectiveness and Patient-Centered Outcomes Research: Enhancing Uptake and Use by Patients, Clinicians and Payers”. The 70 invited participants represented key ‘users’ of CER/PCOR evidence: academic organizations, professional associations, healthcare provider groups, insurance companies and other payer organizations, patient advocacy groups, government agencies, research groups, pharmaceutical and biotech manufacturers and others in the CER/PCOR field. Potential attendees were nominated by the organizing committee, or identified by web searches, or recommended by officers of foundations, professional societies/organizations and patient advocacy groups, based on interest in the aims of the conference and ability to represent one of stated stakeholder viewpoints. The overarching goal of the conference was to advance the use of CER/PCOR by patients, clinicians and other decision-makers by identifying the needs of users and promoting best practices for the education of users of CER and PCOR. This issue of CER includes several articles that stem from that conference [10,11]. A full description of the conference presentations and participants is available at the PhRMA Foundation website [12].