Authors: Sharma G, Chiva-Razavi S, Viriato D, Naujoks C, Patalano F, Bentley S, Findley A, Johnson C, Arbuckle R, Wolffsohn J.
Geographical coverage: Not reported
Equity focus: None
Study population: Review considers outcome measures for individuals with presbyopia, rather than the individuals themselves.
Review type: Other review
Quantitative synthesis method: Narrative review
Qualitative synthesis method: Not applicable
Background: Presbyopia is the age-related loss of near distance focusing ability. A patient-reported outcome (PRO) is a health outcome reported directly by the patient about the status of the patient’s health without amendment or interpretation of the patient’s response by a clinician or anyone else. In 2009, the US Food and Drug Administration (FDA) released their influential ‘Guidance for Industry on Patient-Reported Outcome Measures’, which describes the review and evaluation criteria for PROs used to support claims in approved medical product labelling. In order for PROM data to support an FDA product label claim, the PROM must assess relevant and important concept(s) of interest and has been developed and validated to the standards specified in regulator guidelines (including evidence of both content validity and psychometric validity in the population of interest) and included in a well-designed and adequately controlled clinical trial.
Objectives: To identify the most cited PROMs designed for use in presbyopia or similar conditions and critically evaluate the evidence of content validity and psychometric properties, and as such their adequacy for use to support endpoints in a Novartis’s pivotal trial testing a new pharmacological therapy for phakic individuals with presbyopia.
In total, 13 PROMs were identified; a further 23 publications pertaining to the development and validation of these measures were retrieved. Of the 13 measures, only one was presbyopia-specific; 11 were generic eye disease measures used across a range of patient groups, including those with refractive correction (refractive surgery, spectacles and contact lenses) and cataracts; and one was a numeric rating scale (NRS) assessing overall vision satisfaction and ocular comfort.
Most PROMs were developed prior to release of the Food and Drug Administration (FDA) 2009 patient-reported outcome guidance and did not satisfy regulatory standards. The Near Activity Visual Questionnaire (NAVQ) was identified as the most appropriate for assessing near-vision functioning in presbyopia. While the NAVQ was developed in line with the FDA guidance, the items do not reflect changes in technology that have occurred since the questionnaire was developed in 2008 (for example, the increase in smartphone use), and the measure was not validated in a purely phakic presbyopia sample. Further research is ongoing to refine the NAVQ to support trial endpoints related to changes in near-vision functioning associated with phakic presbyopia.
The review included studies using Patient-Reported Outcome Measures (PROMs) in presbyopic individuals. Exclusions were made for non-English articles, non-human studies, conference abstracts, case studies, and reports. Studies not exclusively involving presbyopic individuals, those without a PROM, or those using non-standardized measures for treatment satisfaction were also excluded. Studies focusing solely on dry eye symptoms due to contact lens use in presbyopic individuals were not considered.
Literature searches were performed in Medline, EMBASE and Evidence-Based Medicine Reviews (Cochrane Database of Systematic Reviews and Database of Abstracts of Reviews of Effects) via Ovid SP up until October 2017 (when the search was conducted) using specific search strings. The first-level screening was performed based on the title and abstract of the citations, and full-text copies of the studies with individuals with presbyopia were obtained for the next round of review, which involved critical full-text appraisal based on the aforementioned criteria.
To evaluate the identified PROMs, additional resources were accessed for information on their development and psychometric validation. This included a PROM-specific search using Ovid, bibliographic searches, and information from the PROM developers. Only PROMs with accessible full-text publications on their development and validation history were reviewed. The PROMs were assessed against FDA guidance for clinical endpoint submission for drug approval. Modern psychometric techniques like Rasch analysis were also reviewed. Rasch models convert ordinal scores into linear data for easier interpretation. PROMs meeting most psychometric assessment criteria underwent further face validity evaluation, including item wording and missing concepts examination.
Applicability/external validity: Authors do not raise any concerns in relation to applicability/validity, although they did indicate that the inclusion of non-English literature may have led to the outcome measures being evaluated differently.
Geographic focus: Authors do not consider geographic relevance of outcome measures evaluated.
Summary of quality assessment:
There were several limitations in the approaches used to identify, include and critically appraise studies. The search was restricted to material published in English only and there is no evidence of reference lists being consulted or unpublished material considered for inclusion. Studies were only considered for inclusion by one author. While the concept of data analysis is less relevant to this article, compared with a standard systematic review, it is clear that only one author extracted data from the included studies. For these reasons, we have low confidence in the findings of this review.
Sharma G, Chiva-Razavi S, Viriato D, Naujoks C, Patalano F, Bentley S, Findley A, Johnson C, Arbuckle R, Wolffsohn J. Patient-reported outcome measures in presbyopia: a literature review, BMJ Open Ophthalmol. 2020; 5(1): e000453. Published online 2020 Jul 12.