The Pearls and Perils of Investigating Rare Neuro-Ophthalmic Diseases of Childhood by Dr. Robert Avery
Robert Avery, DO, MSCE, Associate Professor of Ophthalmology and Neurology Perelman School of Medicine University of Pennsylvania and Division of Ophthalmology Children's Hospital of Philadelphia My Bibliography - NCBI (nih.gov)
Description
"The Pearls and Perils of Investigating Rare Neuro-Ophthalmic Diseases of Childhood, bby Dr. Robert Avery"
Natural history studies and registries are an efficient way to improve the management of relatively rare conditions. Two recent prospective longitudinal studies highlight the challenges and successes of executing multi-center registries of rare pediatric neuro-ophthalmologic diseases. We will discuss how factors including sponsor support, sub-contracts, IRB approval, enrollment criteria, data complexity, site engagement, subject confidentiality, and inter-department collaboration all impact study outcomes. Specific examples of downstream benefits from registries and natural history studies (i.e., improved clinical care; historical controls for future trials) will be provided. Lessons learned and recommendations for optimizing study success will be discussed.
Natural history studies and registries are an efficient way to improve the management of relatively rare conditions. Two recent prospective longitudinal studies highlight the challenges and successes of executing multi-center registries of rare pediatric neuro-ophthalmologic diseases. We will discuss how factors including sponsor support, sub-contracts, IRB approval, enrollment criteria, data complexity, site engagement, subject confidentiality, and inter-department collaboration all impact study outcomes. Specific examples of downstream benefits from registries and natural history studies (i.e., improved clinical care; historical controls for future trials) will be provided. Lessons learned and recommendations for optimizing study success will be discussed.
