Symposium on Best Practices in Clinical Study Design for Rare Diseases to Take Place in Washington DC this April

March 27, 2013

Children’s National Medical Center

Submitted by: Abby Bronson

Currently, there are fewer than 400 approved treatments for 7000+ rare diseases, affecting more than 30 million Americans and more than double that worldwide.

The challenges developers of therapies for rare diseases face are many and diverse, ranging from difficulty in obtaining investment to pursue development in rare diseases to understanding the natural disease progression in order to design effective clinical studies. The Clinical and Translational Science Institute Grant (NIH UL1TR000075), the Specialized Center in Research in Pediatric Developmental Pharmacology Grant (NIH 1U54HD071601-01) at Children’s National Medical Center, and The George Washington University are organizing a symposium to discuss some of the unique challenges and solutions rare disease product development faces.

The format of the Symposium will be roundtable style, focusing on: Statistical and Study Design Challenges, Drug Development Tools such as the use of biomarkers, Considerations for Best Practices in Rare Disease Product Development, the Sustainability of Rare Disease Product Development and Infrastructure for Rare Disease Product Development. Please join us for this important meeting!

Symposium on Best Practices in Clinical Study Design for Rare Diseases will take place at the Jack Morton Auditorium in Washington DC on April 29th and 30th, 2013.

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