According to the US Food and Drug Administration, Clinical Outcome Assessments (shortened to “COAs”) are defined as standardized instruments (such as questionnaires, assessments, and diaries) that measure, describe, or reflect how a patient feels, functions, or survives*. COAs provide insights that go beyond traditional clinical measures to support the development of new patient-centered medications and treatments. COAs are instrumental in evaluating the effectiveness and safety of new treatments, as well as assessing clinical practice. They can be crucial for gaining regulatory approval and informing health technology assessments.
This blog post is intended for anyone new to the use of COAs within their clinical research -- understanding these tools can enhance your study's impact, improve patient outcomes and increase the chances that important treatments can come to market.
Different types of Clinical Outcome Assessments
The four primary types of COAs are: Patient-Reported Outcomes, Clinician-Reported Outcomes, Observer-Reported Outcomes and Performance Outcomes. Each type offers a unique perspective of patient health based on who provides the assessment data and how it is collected.
Patient-Reported Outcomes (PROs)
PROs are assessments completed directly by the patients, reflecting their perception of their health, quality of life, and the impact of disease and treatment. PROs are subjective and particularly valuable for understanding the patient’s perspective on symptoms, side effects, and overall well-being while on treatment. PRO responses are without amendment or interpretation of the patient’s response by a clinician or any third party.
PROs are often used in chronic disease management, oncology, and mental health studies. For instance, in cancer trials, PROs can capture patient experiences with pain, fatigue, and emotional distress, providing a view of the treatment's impact on quality of life.
Clinician-Reported Outcomes (ClinROs)
ClinROs are assessments completed by healthcare professionals based on their clinical judgment and observation of a patient’s condition, providing essential insights for evaluating clinical signs, disease severity, and treatment response from a medical perspective.
ClinROs are often used in clinical trials for conditions where professional assessment is critical, such as in neurodegenerative diseases like Parkinson's disease or Alzheimer’s disease, where clinical expertise is necessary to evaluate symptoms like motor function or cognitive decline.
Observer-Reported Outcomes (ObsROs)
ObsROs are assessments provided by an “observer,” or someone other than the patient or clinician, for example a caregiver or family member. These assessments are especially useful when patients are unable to self-report, such as in pediatric studies, or for patients with cognitive impairments.
ObsROs can capture information about daily activities, behavior changes, and functional status from those who are closely involved in the patient's care. In pediatric asthma studies, for example, parents can report on their perceptions of their child's breathing difficulties and response to medication and treatment.
Performance Outcomes (PerfOs)
PerfOs are objective measures of a patient's performance on specific tasks, often conducted in a controlled setting. These outcomes can provide quantifiable data on functional capabilities, such as walking speed, grip strength, or cognitive tests. Data for PerfOs can also be captured through the use of Digital Health Technologies, also known as DHTs, like wearable sensors or pedometers.
PerfOs are particularly useful in clinical trials for musculoskeletal disorders, where physical performance measures can demonstrate treatment efficacy. For example, in multiple sclerosis research, PerfOs like the timed 25-foot walk test can objectively assess mobility and physical function.
Selecting the Appropriate COA
Choosing the right type of COA depends on the specific goals of the study, the disease or condition being studied, and the patient population. Often, using a combination of COAs will be the best way to capture a holistic view of treatment effects. For example, a study on rheumatoid arthritis might use PROs to gather patient feedback on pain and fatigue, ClinROs to assess joint swelling and tenderness, and PerfOs to measure physical function through a grip strength test.
The choice and implementation of COAs can impact regulatory submissions, as regulatory bodies like the FDA and EMA now increasingly emphasize the importance of patient-centered data in their decision-making processes. Including well-validated COAs in clinical trials can support claims about treatment benefits and enhance the likelihood of regulatory approval.
COA Challenges and Best Practices
Implementing COAs in clinical research comes with challenges, such as ensuring the reliability and validity of the instruments, identifying the COA copyright owner and conditions of use, addressing linguistic and cultural differences in multinational trials, and selecting the appropriate set of COAs to limit the burden on patients and investigators.
Organizations like Mapi Research Trust and ICON’s Language services team are skilled in helping COA users navigate and overcome these challenges by selecting validated instruments, providing education and training, and migrating COAs to electronic data capture methods to streamline processes.
Embarking on a new journey of incorporating COAs into clinical research may seem daunting, but the insights gained from these assessments will significantly enhance the value and relevance of your clinical studies. And with seasoned partners like Mapi Research Trust, which brings more than 3 decades of experience in the COA space and exclusive access to more than 800+ COAs, you can launch your journey with confidence, generate robust patient-centered data for regulatory submissions, and ultimately improving patient outcomes.
If we can support your research or education within the COA space, please contact us.