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Commentary
Patient-reported outcome measures should not be the primary outcome in glaucoma clinical trials of disease modification
  1. Alessandro Rabiolo1,
  2. Keith Barton1,
  3. Andrew Ian McNaught2
  1. 1 Moorfields Eye Hospital NHS Foundation Trust, London, UK
  2. 2 Gloucestershire Hospitals NHS Foundation Trust, Cheltenham, Gloucestershire, UK
  1. Correspondence to Professor Andrew Ian McNaught, Gloucestershire Hospitals NHS Foundation Trust, Cheltenham GL53 7AN, Gloucestershire, UK; andrew.mcnaught{at}nhs.net

https://doi.org/10.1136/bjo-2021-320722

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    In the 19th century, Sir William Osler, one of the fathers of modern medicine, stated that ‘The good physician treats the disease; the great physician treats the patient who has the disease’, indicating that, as clinicians, we should pursue a patient-centred, rather than disease-centred approach. In the attempt to provide more patient-centred holistic care, there has been increasing interest in patient-reported outcomes (PROs) and patient-reported outcomes measures (PROMs), which are any self-reported health status directly reported by patients and the tools (usually questionnaires) used to measure them, respectively. A variety of validated PROMs, either with generic or disease-specific constructs, are available in ophthalmology to measure the effect of a given eye condition on the overall health function and disease-specific features (eg, symptoms, treatment side effects, coping), respectively. The widespread use of PROMs across all fields of medicine has caused a paradigm shift in clinical research, with patients’ perception becoming a major determinant of therapeutic interventions’ outcomes. As a result, PROMs are increasingly used as endpoints in clinical trials across all medical fields, including, recently, glaucoma.

    The choice of primary outcome is critical in randomised controlled trials (RCTs), and determines the sample size. A poorly chosen primary outcome may invalidate the results of otherwise well-designed and rigorously conducted trials, producing unreliable results and wasting resources.1

    The primary outcome is the outcome measure that has the greatest importance according to the various stakeholders (investigators, patients, policymakers, funding bodies, pharmaceutical companies).1 Preventing visual disability from glaucoma and preserving the visual-related quality of life (VR QoL) are arguably the ultimate therapeutic goals in glaucoma management. Glaucoma is an initially asymptomatic and usually slowly progressing disease, so attaining such a hard endpoint as symptomatic visual disability is (fortunately) infrequent, and besides, nearly always requires a long period of time, perhaps measured in …

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    Footnotes

    • Contributors AR: Conception and design, manuscript drafting. KB: Conception and design, manuscript review. AIM: Conception and design, manuscript review. All the authors have approved the final version of the manuscript and are accountable for all aspects of the work.

    • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

    • Competing interests None declared.

    • Provenance and peer review Commissioned; externally peer reviewed.