scholarly journals Selection of Endpoints for Clinical Studies of Ophthalmic Drugs

2021 ◽  
Vol 11 (3) ◽  
pp. 167-173
Author(s):  
M. O. Komarova
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Studies ◽  
Age Related Macular Degeneration ◽  
Surrogate Endpoints ◽  
Social Significance ◽  
Ophthalmic Drugs ◽  
Clinical Endpoints ◽  
Age Related ◽  
Selection Of

Until now, there have been no effective treatments for some ophthalmic diseases that have high social significance. Development of therapeutic approaches to such diseases may be complicated due to challenges in diagnosis and selection of clinical trial endpoints. The aim of the study was to analyse current approaches to selection of endpoints in clinical trials of ophthalmic drugs. Clinical efficacy studies of new medicinal products use surrogate endpoints in addition to clinical endpoints. However, currently used surrogate endpoints are not always relevant and do not fully reflect changes in the status of patients with chronic or progressive diseases. The study analysed published approaches to the selection of endpoints in clinical studies of ophthalmic drugs intended for the treatment of glaucoma, uveitis, dry eye syndrome, and age-related macular degeneration. It was demonstrated that the choice of surrogate endpoints in a clinical trial should take into account specific aspects of a particular disease. The assessment of dynamic patterns of changes in visual functions generally requires a complex approach for a comprehensive characterisation of the eye condition in a particular disease. The paper analyses the possibility of using potential surrogate endpoints in studies of the most common eye diseases, and highlights that none of them has been recommended for use in clinical trials or routine clinical practice.

scholarly journals Use of composite endpoints in early and intermediate age-related macular degeneration clinical trials – state-of-the-art and future directions

2020 ◽  
Author(s):  
Jan Henrik Terheyden ◽  
Steffen Schmitz-Valckenberg ◽  
David P. Crabb ◽  
Hannah Dunbar ◽  
Ulrich F. O. Luhmann ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Clinical Studies ◽  
Composite Endpoint ◽  
Age Related Macular Degeneration ◽  
Sensitivity Analyses ◽  
Surrogate Endpoints ◽  
Qualitative Synthesis ◽  
Age Related ◽  
Composite Endpoints

The slow progression of early AMD stages to advanced AMD requires the use of surrogate endpoints in clinical trials. The use of combined endpoints may allow for shorter and smaller trials due to increased precision. We performed a literature search for the use of composite endpoints as primary outcome measures in clinical studies of early AMD stages. PubMed was searched for composite endpoints used in early/intermediate AMD studies published during the last 10 years. A total of 673 articles of interest were identified. After reviewing abstracts and applicable full-text articles, 33 articles were eligible and thus included in the qualitative synthesis. The main composite endpoint categories were: Combined structural and functional endpoints, combined structural endpoints, combined functional endpoints and combined multi-categorical endpoints. The majority of the studies included binary composite endpoints. There was a lack of sensitivity analyses of different endpoints against accepted outcomes (i.e. progression) in the literature. Various composite outcome measures have been used but there is a lack of standardization. To date no agreement on the optimal approach to implement combined endpoints in clinical studies of early stages of AMD exists and no surrogate endpoints have been accepted for AMD progression.

scholarly journals BIOMARKER STRATEGIES FOR GEROSCIENCE-GUIDED CLINICAL TRIALS

2019 ◽  
Vol 3 (Supplement_1) ◽  
pp. S745-S746
Author(s):  
Jamie N Justice ◽  
George A Kuchel ◽  
Nir Barzilai ◽  
Stephen Kritchevsky
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Biological Aging ◽  
Expert Committee ◽  
Biomarkers Of Aging ◽  
Age Related ◽  
Trial Outcomes ◽  
Biology Of Aging ◽  
Potential Use ◽  
Selection Of

Abstract Significant progress in the biology of aging and animal models supports the geroscience hypothesis: by targeting biological aging the onset of age-related diseases can be delayed. Geroscience investigators will test this hypothesis in a multicenter clinical trial, to determine if interventions on biological aging processes can prevent accumulation of multiple age-related diseases and aging phenotypes in older adults. Prodigious activity is underway to develop markers of biological aging, but currently there is no aging biomarker consensus to support geroscience-guided clinical trial outcomes. We convened an expert committee to establish a framework for selection of blood-based biomarkers, emphasizing: feasibility/reliability; aging relevance; ability to predict clinical trial outcomes; and responsiveness to intervention. We applied this framework and identified a short-list of blood-based biomarkers with potential use in multicenter trials on aging. We review progress on efforts to test these candidate biomarkers of aging and development of biomarkers strategy for geroscience-guided clinical trials.

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