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Outcomes Research

Table of Contents

What is outcomes research?

Understanding health outcomes research and its significance is no longer confined to scrutinizing clinical trial results.  Understanding health outcomes has become an ongoing and evolving process, and in today’s world, data is extracted from all aspects of the lives of patients. Within this context, outcomes research has the potential for profound depth of knowledge, complexity, and wisdom that has only begun to be extracted over the past decades.  The field of outcomes research seeks to use observational and clinical health data and optimize how health outcomes can be understood and utilized to benefit the patient and society at large. 

The field of outcomes research illustrates how interventions work in a real-world setting. In contrast to clinical trials that evaluate treatment efficacy through randomization, outcomes research presents a broader focus – to provide insights on the causes that contribute to illness. Outcomes research places a greater value on the patient experience and on what can be observed during treatment. This type of research has a broader scope and focuses more on the value of an intervention, including the benefits, risks, costs, and patient experiences that result from treatment. 

Outcomes research also highlights the patient perspective. Patients also have a significant stake in outcomes research because it facilitates their decision-making, both in deciding what intervention is best for them given their circumstances and as members of the public who ultimately have to pay for medical services.

What is the purpose of outcomes research?

The intent of outcomes research is to identify shortfalls in health care practice and treatment and to develop strategies to improve care. 

Outcomes research studies are playing an increasingly significant role in healthcare decision-making and are central to healthcare discussions. The purpose of outcomes research involves:

  • Setting costs for medical interventions;
  • Setting public healthcare policy;
  • Deciding how to optimize resources to support patient health; and
  • Helping patients make informed choices about their health.

Outcomes research is sometimes coupled with health economics principles under the discipline of health economics and outcomes research (HEOR).. The field of HEOR combines principles of economics with the most reliable real-world evidence from healthcare consumers to help bring to light the differentiation of and value differences between medical and pharmacological treatment options. 

Research questions in outcomes research

In outcomes research, the scientist examines a research question to investigate how an intervention is working in a real-world setting. Study outcomes should be formulated before the scientist analyzes results to ensure objectivity in the study. 

A sample question would be: What are the comparative benefits and risks of nursing home, assisted living, and home-based care for older adults with dementia? 

Outcomes research studies compare how each variable or intervention (nursing home, assisted living, home-based care) contributes to patient health. The “intervention” being evaluated is not limited to medications or new clinical procedures, but may also include the provision of particular services or resources, or even the enforcing of specific policies and regulations by legislative/financial bodies.

What are the benefits of outcomes research?

By linking the care people get to the outcomes they experience, outcomes research has become the key to developing better ways to monitor and improve the quality of care for healthcare consumers. The benefits of outcomes research include: 

  • Improve quality of life for patients
  • Patients can make more informed decisions about their health
  • Can help healthcare decision-makers choose the most effective interventions
  • Can bring together a variety of stakeholders into the public health discussion (patients, clinicians, purchasers, researchers, academia/universities, and policymakers).

What are examples of health outcomes?

Outcomes, sometimes called endpoints or end results, are variables that are used in a study to “document the impact that a given intervention or exposure has on the health of a given population.”  

Examples of health outcomes include, but are not limited to:

  • Cure rates
  • Activities of daily living
  • Mortality 
  • Hospital admissions
  • Clinical worsening
  • Patient satisfaction

Outcomes, or “endpoints,” can also be broken down between “hard” endpoints, such as mortality and hospital admissions, and “soft” endpoints, such as quality of life, drug-related problems, and patient satisfaction.  Outcomes can be either patient-centered or systematic, focusing on the benefit to the larger population or community.

Origins of outcomes research

With origins pointing back over the last two centuries, outcomes research strategies have burgeoned over the last sixty years, proliferating  reliable tools for outcomes assessment.  Florence Nightingale is deemed as an original initiator of outcomes research, constantly looking at medical statistics to reveal the nature of infections in hospitals and on the battlefield during the 1850s.  Avedis Donabedian, John Wenneberg, and Paul Ellwood’s work have also been touted as imperative for outcomes research over the last sixty years.

How does Outcomes Research support patient-centered care?

Outcomes research has contributed to an evolution in healthcare and clinical research – where observation of progress and patient experience drive the discussion and decision making.  With outcomes research, the rules have changed, and patient-centered care is not simply an option, but a necessity and provides the data to drive the most relevant and essential research. Outcomes research serves to promote the value of the patient outcomes and experiences in driving healthcare decision-making, the cost of interventions, and the choices made available to patients.  

Patient Registries

One significant trend in outcomes research is the use of patient registries to provide data that is catered towards a specific disease state or research topic. According to the Patient-Centered Outcomes Research Institute, a patient registry is a system that uses observational study methods to collect data and evaluate outcomes for a specific population cohort (defined by a disease, condition, etc.) and that serves a predetermined scientific, clinical, or policy purpose. 

Who is using Health Outcomes Research?

Many stakeholders rely on outcomes research including patients, physicians, purchasers, pharmaceutical companies, research centers, regulators (FDA, EMA, etc.) and policymakers.

Challenges in outcomes research

Challenges within outcomes research include:

  • How to synthesize an exorbitant amount of available health data 
  • How to design studies that effectively analyze complex interventions 
  • How to include appropriate “hard” or “soft” outcome measures

As the field of health outcomes research has expanded its reach well beyond the endpoints of clinical trials, challenges and opportunities lie in its ability to extract insights from the vast data, resources, patient perspectives and observations, and stakeholder input.