A federal drug program blocks rural hospitals from getting discounts on rare-disease drugs, forcing staff to cut back on supplies of lifesaving medicines.
Following a KHN 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.
The controversial 340B drug discount program for hospitals came under fire at a congressional hearing.
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.
Two companies that faced criticism for high-priced drugs, Marathon and Mallinckrodt, have dropped out of the PhRMA trade association.
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.
Amid an uproar over high drug prices, three GOP senators are asking the Government Accountability Office to investigate whether the Orphan Drug Act is being abused.
A drug from Marathon Pharmaceuticals has ignited a firestorm on Capitol Hill and beyond. What makes it different than the $750,000 drug that came before it?
Former FDA Commissioner Dr. Robert Califf shares his views about drug approvals, regulations and safety concerns after stepping down from the giant agency.
After hearing complaints about its high price, Marathon Pharmaceuticals is pausing the launch of an $89,000 drug for a rare disease.
Citing a Kaiser Health News investigation, Senate Judiciary Committee Chairman Chuck Grassley vows to examine the orphan drug program and possible fixes.
The designation, which is made by the Food and Drug Administration, allows drugmakers to claim seven years of market exclusivity.
Orphan drugs for rare diseases have helped or saved hundreds of thousands of patients like 2-year-old Luke Whitbeck, but families and insurers are picking up the astronomical cost.
Una investigación de Kaiser Health News analiza las acciones de compañias farmacéuticas para manipular los precios de medicamentos huérfanos, utilizados para tratar enfermedades raras.