Orphan Drug Designation: When Is It Appropriate, And How Does It Apply To Orphan Subsets?

By Vanessa Atayde

It's a regrettable reality that numerous diseases impact so few patients that developing a drug or biological product to treat them often results in limited returns that can't offset the high development costs. Consequently, the pharmaceutical industry has historically overlooked many rare diseases.

Nonetheless, the 1983 Orphan Drug Act established the Orphan Drug Designation (ODD) program to incentivize pharmaceutical companies to address the unmet needs of rare disease patients. ODD grants sponsors tax credits, waivers from NDA and BLA user fees, and an attractive seven-year market exclusivity period following approval. To qualify for orphan status, a drug or biological product must be intended for the prevention, diagnosis, or treatment of a disease or condition affecting fewer than 200,000 people in the United States.

Given that ODD is frequently denied for orphan subsets, it is crucial for a sponsor to engage the agency with a comprehensive grasp of its expectations and prerequisites. Learn how to develop customized and tailored strategic plans, mitigate regulatory risks, and optimize marketing success by accessing the blog below.