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.
The FDA granted approval for Spinraza in late December for use on children and adults with spinal muscular atrophy. Insurance coverage is mostly focused on infants and children.
The high cost of Spinraza, a new and promising treatment for spinal muscular atrophy, highlights how the cost-benefit analysis insurers use to make drug coverage decisions plays out in human terms.
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.
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?
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.
Luke Whitbeck’s life was saved by a rare disease drug, but it costs $300,000 a year.
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.
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.
Follow the twists and turns of the orphan drug industry over the past three decades.
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.
The former Congressman championed the Orphan Drug Act decades ago but now he fears it’s being manipulated to make money.
A voucher awarded to companies that find treatments for rare childhood diseases can be sold to the highest bidder — and then used to speed up approvals for much more common drugs.