We support research that will provide reliable, useful information to help people make informed healthcare decisions and improve healthcare delivery and outcomes. The research we fund is guided by patients, caregivers, clinicians, and other healthcare stakeholders. This research, which can be described as Patient-Centered Outcomes Research, or PCOR, is a type of comparative clinical effectiveness research, or CER. It includes some large pragmatic studies and large-scale observational studies. We also fund research to address gaps in methodology relevant to PCOR.
Patient-Centered Outcomes Research
PCOR is a relatively new research field that considers patients’ needs and preferences and focuses on outcomes most important to them. PCOR findings can help patients and other healthcare stakeholders, such as caregivers, clinicians, insurers, policymakers and others, make better-informed decisions about their health and healthcare options. This research answers patient-centered questions, such as:
- “Given my personal characteristics, conditions, and preferences, what should I expect will happen to me?”
- “What are my options, and what are the potential benefits and harms of those options?”
- “What can I do to improve the outcomes that are most important to me?”
- “How can clinicians and the care delivery systems they work in help me make the best decisions about my health and health care?”
To answer these questions, PCOR:
- Assesses the benefits and harms of preventive, diagnostic, therapeutic, palliative, or health delivery system interventions to inform decision making, highlighting comparisons and outcomes that matter to people
- Is inclusive of an individual's preferences, autonomy, and needs, focusing on outcomes that people notice and care about such as survival, function, symptoms, and health-related quality of life
- Incorporates a wide variety of settings and diversity of participants to address individual differences and barriers to implementation and dissemination
- Investigates (or may investigate) optimizing outcomes while addressing burden to individuals, availability of services, technology, and personnel, and other stakeholder perspectives
By including patient and other stakeholder perspectives throughout the research process, PCOR is more likely to address potential barriers to implementation and lead to healthcare strategies that will achieve the desired health outcomes. (Learn more about how we established this definition of PCOR.)
Comparative Clinical Effectiveness Research
CER is a field of research designed to compare the effectiveness of two or more interventions or approaches to health care, examining their risks and benefits. CER findings assist clinicians, patients and other stakeholders in making informed decisions that improve health care for both individuals and populations. The direct comparison of two or more interventions distinguishes CER from studies explore outcomes related to one intervention alone. CER can not only validate a particular intervention but also identify which of available treatments best meet the needs of a given population.
At PCORI, we are looking for research focused on approaches known to be efficacious but not adequately compared in previous studies. We particularly encourage randomized controlled trials (RCTs) that are conducted in typical clinical populations and address outcomes important to patients.
We are interested in CER that examines:
- Strategies for prevention, screening, diagnosis, treatment, or management of clinical conditions
- Methods to improve delivery of care
- Features of healthcare systems
- Interventions to reduce or eliminate disparities in health and health care
- Health communication techniques
Types of PCOR/CER
Randomized Controlled Trials
In performing experiments to compare interventions, it can be difficult to separate the effects of the interventions from other factors that might vary between patients. The randomized controlled trial, or RCT, which is often considered the most powerful clinical research tool, provides a means to deal with this challenge.
In RCTs, researchers randomly assign participants to an intervention and then measure particular outcomes. Because of this assignment process, the distribution of factors that might affect the outcomes is likely to be similar across the groups. So, differences in outcome can be attributed to the intervention, even if individual participants differ in other factors. However, RCTs may be impractical for assessing rare outcomes or effects that take a long time to develop. They can also be hindered by participants’ low compliance with an intervention or high drop-out rates.
Pragmatic Clinical Studies
Pragmatic Clinical Studies are intended to provide information that can be directly adopted by healthcare providers. They tend to be conducted in routine clinical settings. They are often large, in part because expected differences in effectiveness may be small, yet important, or effectiveness may differ in patient subgroups. Compared with typical clinical trials, protocols for these studies are often less complex and less intrusive to routine clinical practice, so they’re sometimes called “large simple trials.”
We seek to fund large pragmatic studies that compare two or more alternatives for addressing prevention, diagnosis, treatment, or management of a disease or symptom; improving healthcare system–level approaches to managing care; or for eliminating health or healthcare disparities.
Proposed studies must address critical clinical choices faced by patients, their caregivers, clinicians, and/or delivery systems. These studies must involve broadly representative patient populations and be large enough to provide precise estimates of hypothesized differences in effectiveness and support evaluation of potential differences in patient subgroups.
Observational studies can also complement RCTs in answering questions important to patients and other stakeholders. In observational studies, interventions are decided not by random assignment but as part of normal clinical care. Such studies may be useful when researchers can’t assign participants to treatment or control groups for practical or ethical reasons or when an effect is rare or slow to appear. Electronic health databases make it possible to conduct observational studies in large populations treated in standard clinical settings. Some observational studies use information from previously collected data, such as a registry, so avoid the time required for data collection.
The challenge of observational studies is that the variability and complexity of patients and their circumstances, as well as the care they receive, often make it difficult to conclude whether a specific intervention is responsible for the observed outcomes. However, statistical techniques are strengthening the results of observational studies. Several analytic tools aim to mathematically control the effects of confounding variables, thereby producing a valid estimate of an intervention’s effect even in complex situations.
In addition to the clinical research described here, PCORI also supports studies designed to advance the field of PCOR and CER through research on the way such studies are conducted and the infrastructure used to undertake the research.
Improving existing PCOR and CER methods will benefits patients, caregivers, and clinicians in making healthcare decisions, as well as researchers planning investigations and policymakers weighing the value of different approaches to care. Understanding how a study was designed and conducted is critically important to determining the usefulness of the findings that result. To be trustworthy, findings must be based on valid, rigorous, patient-centered methods. The PCORI Methodology Report and Methodology Standards highlights gaps in the evidence that our program of methodological research should address.
Topics that we are interested in funding include:
- Methods for patient and other stakeholder engagement in research
- General and design-specific analytic methods
- Issues related to human subjects protections
- Strategies for increasing recruitment and retention of study participants
To improve the nation’s capacity to conduct PCOR and CER efficiently, we are supporting the development of a large, representative, national clinical data network. This electronic-data infrastructure—known as PCORnet, the National Patient-Centered Clinical Research Network—will foster a wide range of experimental and observational patient-centered studies. Patients and other stakeholders are participating in the development and implementation of network activities. When fully realized, PCORnet will improve the pace, quality, and patient-centered nature of clinical research.
Research on Rare Diseases
We fund patient-centered comparative effectiveness research (CER) that addresses outstanding questions about the prevention, diagnosis, and treatment of rare diseases. PCORI’s authorizing law calls for a focus on these conditions, defined as affecting fewer than 200,000 people in the United States. We have developed a formal list of guidelines to advise the research community.
Posted: September 10, 2014; Updated: October 20, 2015