Orphan drugs

Research and development for prescription drugs is thriving

Pills in a hand
Photo: John Tlumacki/The Boston Globe via Getty Images

In 2018, a record 59 new drugs were launched — a portfolio that makes it clear that development is increasingly geared toward specialty drugs, according to a new IQVIA report.

By the numbers: Almost half launched with orphan drug status for the use that was being approved, meaning they were designed to serve small patient populations.

Orphan drug approvals have skyrocketed under Trump

llustration of medication bottle, pills, dollar, syringe, and price arrow
Illustration: Sarah Grillo/Axios

The past 2 years have seen a surge in the number of prescription drugs approved through a special process that was designed to help foster new treatments for rare diseases.

Why it matters: Rapid scientific advances have helped fuel this rise. But the sheer volume of rare-disease approvals is also sparking a debate about whether they're too easy to obtain, or whether they give drugmakers a way to game the system.