House Republicans want to repeal federal tax credits that have helped spur a boom in orphan drugs for rare diseases.
Following a KHN and NPR investigation, the Food and Drug Administration has moved to speed up approvals of “orphan drugs” while closing a loophole that allowed drugmakers to skip pediatric testing.
Hospital use of two popular heart medicines, nitroprusside and isoproterenol, dramatically dropped after the prices for both soared.
One in 9 Medicare enrollees have COPD and many of them can’t afford the inhalers that keep them out of the emergency room.
A new JAMA study examines how drug rebates can direct money to middlemen and force Medicare patients to cough up more money.
Several public health officials endorse using a federal law to slash hepatitis C drug prices in Louisiana and avoid drug bills that could cripple the state budget.
Amplifying the “patient voice,” those with the rarest afflictions are trained to become powerful advocates for new drugs and legislation that would help the industry.
The Government Accountability Office said it will investigate potential abuses of the orphan drug program, which offers incentives to drugmakers to develop medicines for rare diseases.
Marathon, maker of an expensive treatment for Duchenne muscular dystrophy, sells the drug for $140 million in cash and stock to PTC Therapeutics.
Two Democratic congressmen met with President Trump to seek his support for a bill to expand the government’s ability to negotiate drug prices, but it’s not clear it would have much impact or will gain support.