June 2022
Volume 63, Issue 7
Open Access
ARVO Annual Meeting Abstract  |   June 2022
Generation of qualitative evidence to support the content validity of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis
Author Affiliations & Notes
  • Christine Nichols Kay
    University of Florida, Gainesville, Florida, United States
  • Isabelle S Audo
    Sorbonne Université, INSERM, CNRS, Institut de la Vision, CHNO des Quinze-Vingts, REFERET national rare disease center, INSERM-DGOS CIC1423, Paris, France
  • Helena Bradley
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Melissa Barclay
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Joel Sims
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Kieran Boparai
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Francesco Patalano
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Christel Naujoks
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Claudio Spera
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Daniel Viriato
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • M Dominik Fischer
    Centre for Ophthalmology, University of Tübingen, Tübingen, Germany
    Oxford Eye Hospital, Oxford University NHS Foundation Trust, Oxford, United Kingdom
  • Jane Green
    Discipline of Genetics, Faculty of Medicine, Memorial University of Newfoundland, St. John's, Newfoundland, Canada
  • Todd Durham
    Foundation Fighting Blindness Inc, Columbia, Maryland, United States
  • Nicola Williamson
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Judit Banhazi
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Footnotes
    Commercial Relationships   Christine Kay AGTC, Foundation Fighting Blindness, Alkeus, Gyroscope, REGENXBIO, Nightstar Therapeutics/Biogen, Spark therapeutics, Novartis, Iveric Bio, ProQR Therapeutics, MeiraGTx, Janssen, Atsena Therapeutics, 4D Molecular Therapeutics, and Kodiak, Code C (Consultant/Contractor); Isabelle Audo Adelphi Values, Novartis, Sparing Vision, Roche and Biogen, Code C (Consultant/Contractor); Helena Bradley Adelphi Values, Code E (Employment); Melissa Barclay Adelphi Values, Code E (Employment); Joel Sims Adelphi Values, Code E (Employment); Kieran Boparai Adelphi Values, Code E (Employment); Francesco Patalano Novartis Pharma AG, Code E (Employment); Christel Naujoks Novartis Pharma AG, Code E (Employment); Claudio Spera Novartis Pharma AG, Code E (Employment); Daniel Viriato Novartis Pharma AG, Code E (Employment); M Dominik Fischer Adelphi Values, Advent France Biotechnology, Alphasights, Atheneum, Axiom Healthcare Strategies, Biogen, Decision Resources, Dialectica, Frontera Therapeutics, Janssen Research & Development, Navigant, Novartis, Roche, Sirion and STZ eyetrial, Code C (Consultant/Contractor); Jane Green Adelphi Values, Code C (Consultant/Contractor); Todd Durham Novartis, Code C (Consultant/Contractor); Nicola Williamson Adelphi Values, Code E (Employment); Judit Banhazi Novartis Pharma AG, Code E (Employment)
  • Footnotes
    Support  Novartis Pharma AG
Investigative Ophthalmology & Visual Science June 2022, Vol.63, 1711 – F0029. doi:
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      Christine Nichols Kay, Isabelle S Audo, Helena Bradley, Melissa Barclay, Joel Sims, Kieran Boparai, Francesco Patalano, Christel Naujoks, Claudio Spera, Daniel Viriato, M Dominik Fischer, Jane Green, Todd Durham, Nicola Williamson, Judit Banhazi; Generation of qualitative evidence to support the content validity of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis. Invest. Ophthalmol. Vis. Sci. 2022;63(7):1711 – F0029.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Visual impairments associated with Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) have significant impacts on patients’ vision-dependent activities of daily living (ADL) and broader health-related quality of life (HRQoL). Patient and observer-reported outcome (PRO/ObsRO) instruments can capture the patient or caregiver perspective in clinical trials and provide insights beyond clinical biomarkers. However, it is critical that such instruments have sufficient evidence of content validity (are appropriate for the target population). This study aimed to assess the content validity of the Visual Symptom and Impact Outcomes PRO (ViSIO-PRO) and ObsRO (ViSIO-ObsRO) instruments in RP/LCA.

Methods : Qualitative, semi-structured concept elicitation and cognitive debriefing interviews were conducted with 66 participants (33 adults, 10 adolescents, 8 children and 15 caregivers of children) in the US, France, Germany, and Canada. Patients had a clinical and genetic diagnosis of RP/LCA. Participants completed the ViSIO-PRO or -ObsRO instruments using a ‘think aloud’ technique to assess understanding, comprehension, and relevance of the items, instructions, response scales and recall period. Interviews were conducted in two rounds, allowing for modifications and subsequent retesting. Framework analysis of interview transcripts was performed.

Results : Participants demonstrated good understanding of the ViSIO-PRO and -ObsRO items (questions) and instructions across both rounds. Concepts assessed in the ViSIO-PRO and -ObsRO items were considered relevant to the majority of participants. The 7-day recall period and response scales were well understood and appropriately endorsed. Participant and expert clinician feedback supported the following modifications between rounds: 5 items were added to the instruments to assess additional concepts and 1 item was removed from the ViSIO-PRO due to lack of relevance.

Conclusions : Findings support content validity of the ViSIO-PRO and ViSIO-ObsRO instruments as outcome assessments for use across RP/LCA genotypes. Further research to evaluate the psychometric validity of the instruments is underway and will support future use of the instruments as efficacy endpoints in clinical trials to support treatment benefit and in clinical practice to track disease severity.

This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.

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