Episode 009: Rare Diseases

While regulatory changes have made the path to approval easier for rare disease therapies, it is by no means easy - and Judith Ng-Cashin, MD, Chief Scientific Officer of Syneos Health, warns developers getting into the market to do so with eyes wide open. Host Jeff Stewart caught up with Dr. Ng-Cashin at a recent conference to discuss her perspective on the opportunities and challenges inherent in bringing a rare disease therapy to market, how the definition of value varies across stakeholders and why true patient engagement is critical to success. Be sure to check out the pieces below for more on rare disease and implications for drug development: Know Your Rare Disease Market explores the unique aspects of the rare disease market and how these will drive the commercialization process. How payers and manufacturers can find common ground in rare diseases highlights the key findings from recent research with Payer organizations regarding factors driving coverage decisions in the rare disease market, including their view of the patient voice. The information, data, and other content contained in this podcast and any associated articles, sponsorships, advertisements, announcements or other communications are provided for informational purposes only and should not be construed as professional advice of any kind, on any subject matter. The content of the podcast contains general information and may not reflect current legal developments, verdicts or settlements. Moreover, the content is not guaranteed to be complete, correct, timely, current or otherwise up-to-date. Syneos Health reserves the right to make alterations or deletions to the content at any time without notice to you. Syneos Health and its subsidiaries expressly disclaim all liability in respect to actions taken or not taken based on any or all of the podcast content. The information, data and other content contained in this podcast is not a reflection of, endorsed by or otherwise affiliated with, nor should it be attributed to, any Syneos Health clients, customers or other contacts. The views expressed in this podcast belong solely to the speakers and do not represent those of their organization.  If you want access to more future-focused, actionable insights to help biopharmaceutical companies better execute and succeed in a constantly evolving environment, visit the Syneos Health Insights Hub. The perspectives you’ll find there are driven by dynamic research and crafted by subject matter experts focused on real answers to help guide decision-making and investment. You can find it all at https://www.syneoshealth.com/insights-hub. Like what you’re hearing? Be sure to rate and review us!  We want to hear from you! If there’s a topic you’d like us to cover on a future episode, contact us at [email protected].