Clinical endpoints special session, ISCEV 2025, Utrecht

Documenta Ophthalmologica, vol. 153, pp. 3–11

Abstract

This report summarises the Clinical Endpoints Special Session held during the 62nd annual symposium of the International Society for Clinical Electrophysiology of Vision (ISCEV), convened at Tivoli Vredenburg, Utrecht, The Netherlands. The session brought together clinicians, regulators, industry representatives, and a patient voice to consider the state of clinical endpoints in trials for inherited retinal disorders (IRDs). Discussions covered the adequacy of current endpoints, challenges of disease heterogeneity, regulatory expectations, operational feasibility, and the perspectives of patients. The session highlighted the need to anchor emerging endpoints to clinically meaningful outcomes and to minimise assessment burdens.

Authors 10

1. Ruth Hamilton

   Royal Hospital for Children · University of Glasgow

   Affiliation as printed

   College of Medicine, Veterinary & Life Sciences, University of Glasgow, Glasgow, UK

   Department of Clinical Physics & Bioengineering, Royal Hospital for Children, Glasgow, UK

2. David G. Birch

   Retina Foundation of the Southwest

   Affiliation as printed

   Retina Foundation of the Southwest, Dallas, USA

3. Katarína Štingl corresponding

   University of Tübingen

   Affiliation as printed

   University Eye Hospital, Center for Ophthalmology, University of Tübingen, Elfriede-Aulhorn-Straße 7, 72076, Tübingen, Germany. Katarina.Stingl@med.uni-tuebingen.de

4. K. Fujinami

   Keio University · Tokyo Medical Center · National Hospital Organization · Moorfields Eye Hospital · University College London · The University of Tokyo

   Affiliation as printed

   Department of Biological Sciences, Graduate School of Science, The University of Tokyo, Tokyo, Japan

   Department of Ophthalmology, Keio University School of Medicine, Tokyo, Japan

   Laboratory of Visual Physiology, Division of Vision Research, National Institute of Sensory Organs, National Hospital Organization Tokyo Medical Center, Tokyo, Japan

   Moorfields Eye Hospital, London, UK

   University College London Institute of Ophthalmology, London, UK

5. K. Holopigian
   Affiliation as printed

   Novartis Pharmaceuticals Corporation, East Hanover, USA

6. Camiel J. F. Boon Aachen

   Leiden University Medical Center · Amsterdam University Medical Centers · University of Amsterdam

   Affiliation as printed

   Amsterdam University Medical Center, Amsterdam, The Netherlands

   Leiden University Medical Center, Leiden, The Netherlands

7. Jane Moseley

   European Medicines Agency

   Affiliation as printed

   European Medicines Agency, Amsterdam, The Netherlands

8. J. Jason McAnany

   University of Illinois Chicago

   Affiliation as printed

   Department of Biomedical Engineering, University of Illinois Chicago, Chicago, IL, USA

   Department of Ophthalmology and Visual Sciences, University of Illinois Chicago, Chicago, USA

9. Artur V. Cideciyan

   University of Pennsylvania

   Affiliation as printed

   Center for Hereditary Retinal Degenerations, Scheie Eye Institute, University of Pennsylvania, Philadelphia, USA

10. Maria M. van Genderen

    University Medical Center Utrecht · Bartiméus

    Affiliation as printed

    Bartiméus Diagnostic Center for Complex Visual Disorders, Zeist, The Netherlands

    University Medical Center Utrecht, Utrecht, The Netherlands