Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium
| Autor: | Robin R. Ali, James W B Bainbridge, Henry Klassen, John G. Flannery, Eric A. Pierce, Elise Heon, Robert S. Molday, Deniz Dalkara, David G. Birch, Thomas A. Reh, Bart P. Leroy, Paul A. Sieving, Alessandro Iannaccone, Vadim Y. Arshavsky, S.P. Daiger, John R. Heckenlively, K. Thiran Jayasundera, Rajesh C. Rao, Kari Branham, José Sahel, Isabelle Audo, Artur V. Cideciyan, Paul Yang, Simon M. Petersen-Jones, Cagri G. Besirli, Abigail T. Fahim, Jacque L. Duncan, Debra A. Thompson, Mark E. Pennesi, David N. Zacks, Roberto Gattegna, Naheed W. Khan, Dror Sharon, David C. Musch, Enrica Strettoi |
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| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
medicine.medical_specialty
clinical trials Blinding Surrogate endpoint business.industry inherited retinal diseases Biomedical Engineering Outcome measures Review counseling patients Analysis of clinical trials Precision medicine Retina Clinical trial Ophthalmology Patient safety outcome measures Retinal Diseases medicine Position paper Humans natural history studies Precision Medicine Intensive care medicine business |
| Zdroj: | Translational Vision Science & Technology |
| ISSN: | 2164-2591 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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