DIA 2016 Annual Meeting

Component Type: Symposium
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-602-L01-P; CME 1.25; IACET 1.25; RN 1.25

This symposium provides a unique opportunity to hear three interconnected but distinct perspectives on rare disease clinical trials. We will have an overview of the rare disease clinical environment in terms of therapy focus, geography and volume along with a compare and contrast of operational metrics with respect to start up and recruitment to set the stage. Next, the challenges related to ensuring robust safety and efficacy outcomes from a statistical perspective in an environment where limited patient sample sizes is a reality will be explored along with creative methods for overcoming these challenges. Finally, a rare disease in-depth case study involving Latin America and pediatric patients will showcase best practices on how to harness the unique cultural and environmental aspects of this region to meet enrollment targets.

Learning Objectives

Recall the most prolific areas in rare disease work and industry trends; Use metrics to validate start-up and enrollment challenges; Identify the operational challenges of rare disease trials in terms of recruitment as well as in conventional statistical analysis approaches; Discuss the unique cultural and environmental aspects of enrolling pediatric patients in a rare disease pediatric trial in Latin America; Share best practices on how enrollment can be met.

Chair

Maureen Smith

Speaker

Rare Disease: Understanding Operational Challenges
Linda Martin, MBA

Orphan Indications and Clinical Trials: Coping with Unique Challenges and Why Rare Diseases Warrant Special Treatment
Stephan de la Motte, DrMed, MD

Solving Enrollment Challenges for Rare Disease Global Clinical Trials in Latin America
Sara G Tylosky, MBA