Improving Regulatory and Operational Performance in Orphan Drug Development

Presented by

Nach Davé and Kimberly Glen

About this talk

Today more than ever, regulatory bodies are providing a progressive platform for rare disease drug development. Extensions to existing regulations and incentives and attempts to reduce orphan drug designation applications provide a collaborative environment for companies developing orphan drugs. Understanding the regulatory landscape in orphan drug development — specifically the various pathways, incentives, and engagement opportunities — with regulators can help optimize the process and bring therapies to market faster. The U.S. Food and Drug Administration is introducing new review cycles and initiatives to aid researchers, and regulators in other countries will likely follow with similar measures to promote orphan drug development. Sponsors stand to gain by seeking FDA engagement early and often, performing relevant studies in rare populations and using federal grants and other available programs. All of these measures can increase the value of their companies and contribute to a growing database within the rare disease space. People attending this webinar will better understand: - The regulatory and operational nuances of orphan drug development - The benefits of early planning and engagement - How to operationalize their regulatory strategy We will examine strategic considerations in the regulatory and operational nuances of rare disease drug development, including: - Changes in the regulatory landscape that provide a favorable environment for development of orphan drugs - The need for early regulatory engagement when developing and operationalizing plans for drug development - Operationalizing clinical trials within rare disease to support orphan drug designation, including: Patient identification, engagement, and retention Investigative site selection and site team engagement - Involving patient groups, obtaining increased funding, and taking advantage of significant government reforms that are focusing much-needed attention on treating rare diseases
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