Patient-centered outcomes research
If we ask ourselves the question, “Why are we doing research and what are we hoping to accomplish?”, the answer will most likely relate to our desire to have an impact. One of the ways to have an impact is by using the lens of our patients and other stakeholders and focusing on the outcomes that they perceive to be most relevant. This principle is central to doing research that is patient centered.11 Patient-centered outcomes research focuses the attention on the patient’s beliefs, preferences, and needs, in contrast to physician-centered care. Thus, active participation of the patient as a stakeholder is an essential element of patient-centered outcomes research (PCOR).12 Additionally, PCOR engages stakeholders and focusing on outcomes and processes that are prioritized by individuals that are impacted the most which allows for upfront focus on effective implementation and dissemination.
When it comes to PCOR, the Patient-Centered Outcomes Research Institute (PCORI) dominates. In 2010, Congress authorized the establishment of PCORI. It is a publicly supported, independent, non-profit research institute which is devoted to funding comparative clinical effectiveness research (CER) that addresses questions and concerns important to patients. PCORI funds projects that generate and disseminate evidence that is ‘relevant, trustworthy, and useful’ to patients and others its serves. By 2021, PCORI had invested >US$3.4 billion to fund patient-centered, stakeholder-engaged CER studies and other initiatives. Despite concerns that PCORI may not be reauthorized, in 2019, Congress did reauthorize PCORI funding for another 10 years. PCORI’s strategic goals13 are to:
increase quantity, quality, and timeliness of useful, trustworthy research information available to support health decisions;
speed the implementation and use of patient-centered outcomes research evidence;
influence research funded by others to be more patient centered.
Through a series of ‘National Priorities for Health’ that ‘anchor the work’, the PCORI Research Agenda serves as a guide to the development of the research portfolio of PCORI-funded CER projects. PCORI is not the only funding agency interested in PCOR. For example, the Agency for Healthcare Research and Quality partners with PCORI and The Department of Health and Human Services through the Patient-Centered Outcomes Research Trust Fund to disseminate evidence about PCOR and CER.14
Patient and stakeholder engagement is key to the development of successful PCOR projects. Given the central role of the patient perspective in PCOR, methods to incorporate patient-reported outcomes (PROs) are critical. PROs are defined by the Food and Drug Administration and National Quality Form (NQF) as ‘… a report that comes directly from the patient … about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else’.15 16 PROs are distinct from other patient outcomes such as physiological measures, process measures, clinician-reported measures, and caregiver-reported measures. A good PRO should include the following attributes: non-intrusive, realistic, relevant, reached by consensus, and easy to understand.17 Other methodology to incorporate patient-focused metrics and outcomes include strategies such as use of a discrete choice experiments (DCE) to help determine the best primary and secondary aims of a study.18 DCE are a quantitative technique used to measure individual preferences by administering surveys that then ask individuals to choose options between two or more hypothetical scenarios.19 This allows for patient preference to be directly linked to an outcome measure of interest.
In 2021, the Coalition for National Trauma Research received a PCORI Engagement Award to create The Community of Trauma Care—Partnering with Patients and Caregivers to Improve Injury Outcomes. The objectives of this partnership are to:
establish stakeholder Injury Research Engagement Panel (I-REP) to partner in PCOR/CER from conception to dissemination;
develop emergency-setting informed consent strategies and methods for increasing follow-up engagement;
conduct reciprocal education between patients and researchers.
The project seeks to create a sustainable I-REP to engage stakeholders in trauma research, ensuring appropriate research methods and questions, to develop patient/family-centered communications on participating in trauma PCOR/CER, and to disseminate findings to stakeholders to PCOR/CER to improve engagement strategies and outcomes.