The assessment of the level of benefit through the measurement of treatment outcomes is the cornerstone of clinical trials. Traditionally, these measurements were physical recordings taken by a medical professional or clinical trial staff.
However, over the last 10-20 years, patient-reported outcomes (PROs) have become commonplace. With the acceleration of digitisation introduced by the COVID pandemic, digital PROs are now increasingly utilised within trials.
This educational blog will explore what PROs are, explains why they of growing importance in the digital age and how they are perceived by health technology assessment (HTA) agencies.
What are PRO’s?
The Food and Drug Administration (FDA) define a PRO as “any report of the status of a patient’s health condition that comes directly from the patient without interpretation of the patient’s response by a clinician or anyone else”. Given that the emphasis is on the patient themselves, it is typical that a PRO will be associated with capturing the impact of a treatment on their own health, quality of life or functional status. Therefore, the use of PROs allow trials to capture a wider, more holistic range of patient benefits, beyond that of disease morbidity and mortality.
As Weldring and Smith (2013) explain succinctly: For example, a new drug may demonstrate good clinical outcomes in terms of improving the length of survival for a particular patient group, while PROs may identify that patients are non- compliant with the drug regime due to reported adverse or side effects, complexity of the drug regime, and or a poor quality of life1
Therefore, PROs allow us to understand not only the length of life new treatments offer but also the quality of life provided to those patients.
As well as clinical trial use, PROs can be utilised by healthcare institutions. For example, the National Health Service (NHS) have been collecting PROs as indication of quality of care and treatment outcome in patients undergoing hip and knee replacements since 2009, and publish an annual report.
How are PRO’s captured?
The tools/instruments used to collect PROs are patient-reported outcome measures (PROMs). Typically a self-reporting questionnaire, the PROM has to be suitably easy for the patient to understand and complete the information. There are two types of PROMs: generic, and disease-specific.
Generic PROMS are usually sets of standardised questions that can be used across multiple disease areas. Perhaps the most famous example is the EQ-5D Index, a non-disease-specific PROM that aggregates responses provided by patients across five general areas (anxiety/depression, pain/discomfort, self-care, mobility and usual activities) into one value.
Alternatively, there can be PROMs that are specific to the disease. These are often used alongside generic PROMs to provide a more nuanced assessment on the impact of a particular disease on the patient’s perception of their health (e.g. the Asthma Quality of Life Questionnaire (AQLQ)).
Acceptability by HTA agencies
The key aspect to the acceptability of PROMs by HTA agencies, both non-specific and disease-specific, is whether they have been validated (i.e. there is common consensus that the instrument is measuring what it is intended to measure). If a unvalidated PROM is used within a trial, this can generates large uncertainty for the HTA agency in assessing the clinical benefit of the product.
The acceptability of PROs/PROMs is now widespread, given the focus on patient quality of life within HTA submissions, with some HTA agencies (e.g. NICE) requiring PROMs to be used for the calculations of QALYs.
What next for PROs/PROMS?
Digitalisation is affecting all areas of the healthcare and pharmaceutical industries, and the collection of PROs is no different. While the use of ePROMs has been increasing anyway, it is likely that COVID will accelerate this trend.
The advantage to collecting PROs electronically is obvious, in that what was previously entered written down on paper and processed manually (e.g. scanning) can now be entered onto a phone, tablet or computer and processed instantly. Evidence suggests that ePROMs are preferred by patients, and that the method of collection imparts no bias on the outcome2. However, there are technical costs that must be considered, and the consider technical fluency of the patients must be considered
With the expected explosion of digital health over the next decade, it is likely that ePROMs will form a major part of measuring their patient benefit. Being aware of the fundamentals of ePROMs, and how they are perceived by HTA agencies, will be an important aspect of market access over the next decade.
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