Patient-reported outcome measures: the fundamentals
What are patient-reported outcomes and patient-reported outcome measures? How may they improve patients’ care? In this article, we explore these questions and more!
In clinical trials of novel or experimental therapeutics, investigators will define endpoints that must be met for the therapeutic agent to be considered effective for its target indication. For example, in a clinical trial of a new migraine treatment, the primary endpoint may be defined as a reduction in the number of severe migraine days experienced by a patient per month.
However, in recent years, there has been increasing recognition by stakeholders across the healthcare research and decision-making enterprise that patients are experts in their own conditions, needs and what health outcomes are most important to them.
Many have recognized the value and importance of obtaining self-reported measures of patients’ quality of life changes.
By definition, patient-reported outcomes – often abbreviated to PROs – are information provided directly from patients about changes to any aspect of their health, without any interpretation by a clinician or other stakeholder. By contrast, traditionally measured clinician-reported outcomes are measurable changes to a patients’ health that a physician observes.
Measuring patient outcomes in a more direct way may help improve patient care and patient centricity by supporting prioritization and goal setting, as discussed later.
Patient-reported outcome measures: generic versus condition specific
Patient-reported outcomes are constructs normally measured or collected using patient-reported outcome measures – or ‘PROMs’. These questionnaires or instruments represent standardized, validated methods of assessing physical, mental and social health parameters, such as patients’ quality of life [1].
Patients’ responses – such as their rating of pain intensity due to a specific cause or the difficulty they experience in performing a routine, daily activity – are converted into scales and scores that can be used to help determine whether an intervention improves a patient’s health or care. Ideally, patient-reported outcomes are collected before and after an intervention has been proposed, to better determine its impact.
Patient-reported outcome measures can be general, pertaining to a person’s overall health status and quality-of-life change, or condition specific, relating to a particular disease.
Generic patient-reported outcome measures have the advantage of allowing comparisons to be made across disease and treatment types, regardless of factors such as the patients’ health condition or demographics such as gender.
By contrast, condition-specific – or disease-specific – patient-reported outcome measures are used to evaluate the effectiveness of an intervention for a single indication only. These allow providers to determine the improvement on a patients’ quality of life resulting from a given treatment regimen in a particular population [2].
However, critics of generic patient-reported outcome measures argue that, in reality, their usefulness may be limited due to impacts of factors such as patients’ ages.
Examples of traditional patient-reported outcome measures include the Sickness Impact Profile and the Instrumental Activities of Daily Living. Today, commonly employed generic patient-reported outcome measures include the EuroQOL EQ-5D and the Short Form SF-36, which are frequently employed in cancer trials [2–3].
Patient-reported outcome measures in clinical practice
Consideration and use of patient-reported outcomes in clinical research has been fairly well established. There have also been examples of using patient-reported outcomes in large population surveys, though less commonly.
However, increasing calls for the adoption and incorporation of patient-reported outcomes in clinical decision-making have been made. Cited potential benefits of using patient-reported outcomes in clinical practice include supporting diagnostic decision-making, informing risk stratification, supporting prioritization and goal setting, and facilitating communication. All of these could help advance the delivery of patient-centered healthcare.
#ProjectPatientVoice
On 23 June 2020, the US FDA announced its pilot launch of Project Patient Voice – an online platform from which the agency will publicly share patient-reported outcome and symptom data from cancer clinical trials of marketed therapeutics [3].
The novel platform will, for the first time, allow US patients and their healthcare providers to access these patient-reported data, which have historically been considered by the agency during drug approval processes but rarely included on products’ labels.
“The Project Patient Voice pilot is a significant step in advancing a patient-centered approach to oncology drug development,” explained Amy Abernethy, Principal Deputy Commissioner at the FDA.
References:
[1] Porter I, Gonçalves-Bradley D, Ricci-Cabello I et al. Framework and guidance for implementing patient-reported outcomes in clinical practice: evidence, challenges and opportunities. J. Comp. Eff. Res. 5(5): 507–519 (2016)
[2] Besson A, Deftereos I, Chan S, Faragher IG, Kinsella R, Yeung JMC. Understanding patient-reported outcome measures in colorectal cancer. Future Oncol. 15(10): 1135–1146 (2019)