Orphan drug designation for non-profits: integrating early regulatory strategy to derisk rare disease development

Rare diseases collectively affect an estimated 25–30 million people in the United States alone. Yet only a small fraction of the more than 7000 conditions with identified molecular causes have approved therapies.¹

For non-profit organisations working to close these gaps in the US market, limited funding, small teams and restricted regulatory capacity often determine how far a programme can realistically advance.

The cost of clinical trials, FDA user fees and the complexity of navigating the regulatory process without dedicated internal expertise can exacerbate these constraints. 

Orphan drug designation (ODD) can make development more feasible for non-profits, reports H. Greg Thomas (pictured), Vice President of R&D at Kiel Laboratories.