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.
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?
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.
Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives but nearly a third of those products aren’t new or were repurposed multiple times, an investigation shows.
Check out all the drugs the FDA has approved to treat rare diseases. You can search by brand name, or by disease, and see familiar names that were first sold on the mass market or all the drugs that won FDA approval to treat more than one rare disease.
Follow the twists and turns of the orphan drug industry over the past three decades.