orphan drugs

Harvard's Chris Gerry, Ph.D. has previously written about Zolgensma, the world's most expensive drug, and some of the economics behinds its price. Is Zolgensma, the only treatment for spinal muscular atrophy, too expensive, a bargain, or somewhere in between? As Oscar Wilde once said, “the truth is rarely pure and never simple.”
In his second article of a series, Harvard's Dr. Chris Gerry expands the discussion of his first piece, "The Science of Zolgensma, The World’s Most Expensive Medicine" and considers whether Zolgensma merits a price tag of $2.125 million. A look at some of the ethics and complexities of drug pricing.
These smaller population “Orphan Diseases,” defined as those that affect less than 200,000 people across the U.S., collectively impact 25 million Americans. This substantial public health issue warrants Lancet's call-to-action.
Yesterday, the FDA gave the thumbs up to Sanofi s Cerdelga, the first oral treatment for a rare condition called Gaucher s disease. Cerdelga is classified as an orphan drug specialized therapies designed to treat rare diseases in small numbers of patients.
Bacterial resistance is a devastating problem. Over two-million Americans suffer from antibiotic-resistant infections every year, causing about 23,000 deaths. And the World Health Organization warns that a post-antibiotic era may be upon us, when common infections and minor injuries