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.
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.
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.