RI Perspective: The pressing need for PROMs

The time is now – The pressing need for PROMs development for retinal degenerative conditions

20th May, 2021 – International Clinical Trials Day

Fiona Waters – Community Engagement & Outreach Officer, Retina International

Today is International Clinical Trials Day, and Retina International is celebrating the numerous ongoing and innovative clinical trials taking place the retina space, which bring us closer to our vision; preserving and restoring vision through innovation in retinal research. On this day, RI seek to shine a light on the pressing need for valid and reliable PROMs for retinal degenerative conditions, that will maximise the impact of these trials for all – and most importantly, for the patient community we serve.

Clinical Trials Day


When conducting a clinical trial, there are many things an investigator is eager to measure. For clinical trials on conditions of the retina, some things an investigator may measure are: eye pressure, changes in blood supply, images of the retina, or changes in visual acuity. However, these medical outcomes tell us little about the patient experience, or whether the intervention has had any impact on their day-to-day lives. This is, after all, the goal of healthcare – to improve patient outcomes, quality of life, and overall wellbeing.

At times, there may be differences in priorities and expectations between a patient taking part in a clinical trial, and the investigator who is conducting it. This can result in the efforts of those developing therapies not actually meeting the needs of the patients who need them.

Patient Reported Outcomes Measures (PROMs) help investigators to understand the patient perspective and measure a person’s quality of life, the burden of their symptoms, and delivery of their care. They can tell the researcher a fuller story on the impact of the potential treatment. More and more, PROMs are being viewed as crucial to the planning of clinical trials, as the demand for shifting clinical practice towards patient-centred care increases.

PROMs for retinal degenerative conditions are still a work in progress, and require the entire community of stakeholders to work together to put the perspective of the patient at the heart of clinical trials. Listening to, and prioritising the patient need has the power to drive research and innovation, and lead to the improvement in wellbeing of those living with these conditions.

There has been significant PROMs development for common ophthalmological conditions such as cataract, glaucoma, uveitis, and low vision. However, there are few PROMs for less common conditions, particularly Inherited Retinal Degenerations (IRDs), which are considered rare diseases. For those living with acquired retinal degenerative conditions (e.g. Age-related Macular Degeneration, Diabetic Retinopathy) and IRDs, there are a wide range of ways that quality of life can be impacted. Many of these issues are completely unique to each disease group, meaning that each condition would need its own set of PROMs to accurately describe its impact on patients’ lives.

Nearly 300 IRD-causing genes have now been described, and this number is growing all the time. Particularly now, with greater understanding of the need for genetic testing, and the emergence of gene therapies onto the market – with many more in the clinical trial pipeline –  PROMS will improve our understanding of the efficacy of new gene therapies, and maximise their value to patients.

The time is now for the whole community to come together and develop valid, reliable PROMs that truly represent the patients’ voice and values, and place them at the centre of medicines research & development.

Read more:

Patient-reported outcome measures in inherited retinal degeneration gene therapy trials

Exploring the quality of life issues in people with retinal diseases: a qualitative study

“The patient is speaking”: discovering the patient voice in ophthalmology

Types of Patient-Reported Outcomes

The importance of patient-reported outcomes in clinical trials and strategies for future optimization

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