Visualising treatment effects in low-vision settings: proven and potential endpoints for clinical trials of inherited retinal disease therapies
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Published on: February 11, 2014
Arun J Thirunavukarasu1,2, Shabnam Raji3,4, Jasmina Cehajic Kapetanovic3,4
1Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK. enquiries@ndcn.ox.ac.uk.
Abstract:
Inherited retinal diseases are a devasting and incurable cause of blindness which frequently affect patients at a young age, and developing effective treatments has been an important research priority in recent decades. Treatments must be validated in randomised-control trials, which involve measuring benefit according to prospectively defined endpoints. A wide variety of conventional clinical endpoints and emerging anatomical, physiological, and functional biomarkers may be selected. Different options may be better or worse at capturing clinically significant differences and identifying real differences between experimental groups. This review provides an overview of some proven and potential endpoints for randomised-control trials involving inherited retinal disease patients. Clinical endpoints and biomarkers are discussed, and the work required to validate biomarkers for use in trials is outlined. Unlike in general medicine, ophthalmological clinical endpoints may all be conceptualised as surrogates for maintained vision. Selecting optimal endpoints is essential to ensure that treatments are assessed fairly, such that resources are directed towards interventions that stand to truly benefit patients with inherited retinal diseases.
Insights
Choosing the right clinical endpoints and biomarkers is crucial for validating treatments for inherited retinal diseases. This ensures that new therapies genuinely benefit patients by accurately measuring vision maintenance.
Area of Science:
- Ophthalmology
- Genetics
- Clinical Trials
Background:
- Inherited retinal diseases (IRDs) are a leading cause of incurable blindness, often affecting young individuals.
- Developing effective treatments for IRDs is a significant research challenge.
- Randomized controlled trials (RCTs) are essential for validating new IRD therapies.
Purpose of the Study:
- To review established and potential endpoints for IRD clinical trials.
- To discuss the validation process for biomarkers in IRD research.
- To emphasize the importance of selecting appropriate endpoints for fair treatment assessment.
Main Methods:
- Literature review of clinical endpoints and biomarkers for IRD RCTs.
- Discussion of the characteristics and validation requirements of various endpoints.
- Conceptual analysis of ophthalmological endpoints as surrogates for vision.
Main Results:
- A range of clinical endpoints and biomarkers exist for IRD trials, each with varying strengths.
- Biomarker validation is a critical but often complex step.
- Ophthalmological endpoints can be viewed as proxies for preserved visual function.
Conclusions:
- Optimal endpoint selection is vital for accurately assessing treatment efficacy in IRDs.
- Appropriate endpoints ensure that resources are allocated to interventions with genuine patient benefit.
- Further work is needed to validate emerging biomarkers for use in clinical trials.
