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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.
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.
The FDA’s drug-approval team is short more than 700 people and losing skilled staff members to the drug industry.
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.
A study in Health Affairs concludes that orphan drugs for rare diseases are not having a widespread or deep impact on health care spending.
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