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.
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