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.
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.
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.