Expert consensus on the efficacy outcome measurements in clinical trials for inherited retinal diseases in China

Title: Expert consensus on the efficacy outcome measurements in clinical trials for inherited retinal diseases in China
Edition: Original
Classification: Experts consensus
Field: Others
Countries and regions: China
Guidelines users:
Evidence classification method: Not applicable
Development unit: 中华医学会眼科学分会眼底病学组, 中国医师协会眼科医师分会眼底病专业委员会
Registration time: 2022-06-18
Registration number: IPGRP-2022CN351
Purpose of the guideline: Rapid development of recently emerging precision medicine techniques represented by gene therapy has brought hope for the treatment of rare blinding eye diseases such as inherited retinal diseases (IRDs) for which there was no effective treatment previously. Although the globally growth of clinical trials for IRDs has increased rapidly over the past decade, due to the high genetic and clinical phenotypic heterogeneity of IRDs, as well as limited data on epidemiology and natural history of the disease, such studies lack standard outcome measurements and endpoints, posing multiple challenges in terms of study design and clinical practice application. At present, there is no systematic and standardized guidance on clinical outcome measurements and endpoints for clinical trial design in IRDs. Therefore, in order to standardize the evaluation of IRD clinical research outcomes and end-point indicators, the Fundus Disease Group of Ophthalmology Society of Chinese Medical Association and Chinese Medical Doctor Association organized domestic experts to put forward standardized opinions on outcome measurements and endpoints for clinical study design in IRDs, aiming to advance the study design of IRDs natural history research and clinical trials and to effectively evaluate disease progression and intervention efficacy.