T O APPRECIATE the dilemmas the Food and Drug Administration (FDA) faces when it reviews new drugs targeting rare diseases, ...

Little-known provisions in the Big Beautiful Bill lobbied by industry will cost Medicare billions in missed savings.

The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug ...

Kedrion is proud to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ...

Last week, President Trump sent letters to 17 major pharmaceutical companies advising them to work with his administration to ...

The FDA awarded orphan drug designation to VT3989 for mesothelioma, supporting the experimental cancer treatment’s ...

In the U.S., drug developers can petition the U.S. Food and Drug Administration (FDA) for an orphan drug designation for any potential therapy that would treat a condition afflicting fewer than ...

The use of orphan drugs for non-orphan diseases is driving up the cost of these already expensive medications. A new report from America’s Health Insurance Plans found that almost half of the ...

Orphan drug policy could unintentionally impede access to orphan-designated COVID-19 drugs by facilitating high prices. We suggest reforms to mitigate price-related barriers to accessing such drugs.

More orphan drugs than non-orphan drugs have been approved by the Food and Drug Administration over the past several years, he said. "My expectation is that will continue this year as well ...

Payers better be prepared for orphan drugs. The Food and Drug Administration (FDA) will evaluate more than 150 new approvals this year, OptumRx officials said in their latest quarterly report on ...

Kedrion Biopharma receives US FDA orphan drug designation for plasma-derived treatment for congenital aceruloplasminemia: Forte Lee, New Jersey Saturday, August 9, 2025, 17:00 Hrs ...