Tag: Duke University School of Medicine

Rare disease drug development: Time for a new approach?

Written by
A rare disease is one that affects a small percentage of the population.

A limited patient population is a key challenge in rare disease drug development. Professor Shein-Chung Chow of Duke University School of Medicine, USA and his research partners explore innovative approaches to overcome this challenge. These include using external control, selecting appropriate study endpoints, and justifying sample size based on probability statements. They propose the use of a complex innovative two-stage […]

Read More… from Rare disease drug development: Time for a new approach?