Webinar - Clinical Research Design for Rare Disease: Strategy and Pitfalls Developing Experimental Therapeutics for Vascular Anomalies

On-Demand Webinar:

To be a pioneer in organizing and designing a clinical research program for a rare disease can be a rewarding, career making opportunity. Pediatric hematologists and oncologists have optimized models of clinical trial design for our most prevalent life-threatening hematopoietic disorders, paving a path and a model to apply these towards rare diseases under our care. The subspecialty of pediatric vascular anomalies has made recent strides adapting these models by 1st) building a community of motivated providers, engaging patient advocacy communities, and developing a standardized clinical nomenclature, 2nd) creating disease specific databases to conduct retrospective natural history studies to define disease outcome, surveying current clinical practice and developing consensus driven guidelines to help standardize disease diagnosis and management and 3rd) partnered with industry and basic science community to develop prospective observational and experimental therapeutic trials to produce evidence-based data to guide diagnostic and treatment decisions. The goal of this webinar is to review the rewards, strategy, and pitfalls of adapting clinical trial design to the study of rare diseases, exemplified by the study of rare pediatric vascular anomalies.

Learning Objectives:

• Outline steps taken to develop a rare disease community, consortium, and the approach to standardize of disease nomenclature in pediatric vascular anomalies.
• Review the strategy and pitfalls of retrospective review, surveys, and consensus derived study design when applied to rare vascular anomalies.         
• Review strategies and pitfalls when collaborating with industry and developing a rare disease experimental therapeutics for vascular anomalies.

Speakers:

• Taizo Nakano, MD (moderator)
• Sally Cohen-Cutler, MD
• Bryan Sisk, MD
• Alexandra Borst, MD