How, Why, And When To Apply For An Orphan Drug Application

By Hema Balasubramanian and Mamta Puri-Lechner

In the fast-paced world of medical innovation, securing a special designation from the U.S. Food and Drug Administration (FDA) is a significant milestone for any life science company. These designations are designed to accelerate the development and approval of drugs that address unmet medical needs for serious or life-threatening conditions. They are particularly crucial for companies on the brink of groundbreaking discoveries, especially when backed by promising clinical data, at a critical stage in development, or with new funding to scale operations quickly. One such special designation is the Orphan Drug Designation (ODD), offered by the FDA and other global health authorities.

But when should a clinical trial sponsor consider applying for orphan disease designation? This is a common question among our clients and industry peers. If you are in the early stages of drug development with strong nonclinical proof of concept, or have promising early-phase clinical trial data that suggests your candidate could improve an orphan disease, this question is likely on your mind.

Alternatively, if you've just completed a Phase I clinical trial (perhaps in oncology) and have biomarker data indicating efficacy in a rare tumor type, you may be contemplating an orphan indication but are uncertain about the application process, timing, and potential benefits or pitfalls of this pathway.

This article provides strategic insights for clinical trial sponsors on targeting an orphan drug designation and the logistical considerations involved in pursuing this path from the outset.