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 an unprecedented collaborative environment for companies developing orphan drugs.
The U.S. Food and Drug Administration (FDA) is introducing new review cycles and initiatives to aid researchers, and regulators in other countries will likely follow suit with similar measures to promote orphan drug development. 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. Sponsors stand to gain by seeking FDA engagement early and often, performing relevant studies