CPHI Podcast Series: Rare Disease Day – Pharma fulfilling an unmet need

In this podcast Digital Editor Lucy Chard is joined by Rachel Smith of Parexel to discuss the challenges surrounding the development of medicines for rare disease patients. 

Rare diseases affect around 300 million people globally. By their nature, rare diseases are incredibly difficult to research and develop medicines for, but this just makes it all the more urgent to fulfil this unmet need for rare disease patients. 

In this podcast I am joined by Rachel Smith; Rachel is the Executive Director, Global Head of Rare Diseases at Parexel (MA, USA). Rachel brings more than a decade of experience in every development phase of rare disease and cell and gene therapy clinical trials to her work with Parexel’s rare disease clients. Previously, she served as Vice President of Clinical Operations, Portfolio Director of Rare Disease, and Global Head of the Cell & Gene Center of Excellence at Veristat LLC (MA, USA). She led programmes in ADA-SCID, Canavan disease, congenital adrenal hyperplasia, Fabry disease, Gaucher disease, metachromatic leukodystrophy, and Wiskott-Aldrich Syndrome. Rachel has expertise in clinical development strategy, novel and adaptive trial design, decentralised trials, and other creative solutions for rare disease products with a non-classical route to market.

We discuss the disparities in healthcare across the world and the impact this has on making treatment for rare diseases accessible; the true meaning of patient advocacy; and the stories of individuals that demonstrate how important work into understanding rare diseases really is.