Orphan Share of FDA Novel Drug Approvals

## What this measures

The share of new medicines approved each year that were designated orphan drugs, meaning they treat a disease affecting fewer than 200,000 people in the United States.

## Why it matters

Orphan designation brings seven years of market exclusivity and tax credits. A rising share means research money is moving toward small, high-priced markets.

## How to read it

Half of 2025's novel approvals carried the designation. Compare decades rather than single years: small annual counts make the share jump.

## What it does not say

The count covers drugs reviewed by FDA's Center for Drug Evaluation and Research. Vaccines, blood products and cell and gene therapies, reviewed by the Center for Biologics Evaluation and Research, are not in it. A count says nothing about how important or how profitable the drugs are. The share counts designations at approval, not whether the drug was later used for common diseases.

## Source

U.S. Food and Drug Administration: "Compilation of CDER NME and New Biologic Approvals 1985–2025" and the "Novel Drug Approvals" page for each later year. FDA website content is in the public domain.