Thompson, DA;
Iannaccone, A;
Ali, RR;
Arshavsky, VY;
Audo, I;
Bainbridge, JWB;
Besirli, CG;
... Zacks, DN; + view all
(2020)
Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium.
Translational Vision Science and Technology
, 9
(7)
, Article 2. 10.1167/tvst.9.7.2.
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Abstract
Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.
| Type: | Article |
|---|---|
| Title: | Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1167/tvst.9.7.2 |
| Publisher version: | https://doi.org/10.1167/tvst.9.7.2 |
| Language: | English |
| Additional information: | This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License. http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| Keywords: | inherited retinal diseases; clinical trials; natural history studies; outcome measures; counseling patients |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10101671 |
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