$89,000 Orphan Drug Gets A New Owner — And Likely A New Price
Marathon, maker of an expensive treatment for Duchenne muscular dystrophy, sells the drug for $140 million in cash and stock to PTC Therapeutics.
Trump, Dems Look For Common Ground On Drug Prices
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.
Three Key Senators Ask GAO To Investigate Possible Abuses Of The Orphan Drug Act
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.
5 Reasons Why An $89,000 Drug Has Congress Fuming
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 Chief Cites 5 Things To Watch On Drug Approvals And Keeping Drugs Safe
Former FDA Commissioner Dr. Robert Califf shares his views about drug approvals, regulations and safety concerns after stepping down from the giant agency.
Drugmaker Marathon ‘Pausing’ Delivery Of $89,000-A-Year Muscular Dystrophy Drug
After hearing complaints about its high price, Marathon Pharmaceuticals is pausing the launch of an $89,000 drug for a rare disease.
Grassley Launches Inquiry Into Orphan Drugs After KHN Investigation
Citing a Kaiser Health News investigation, Senate Judiciary Committee Chairman Chuck Grassley vows to examine the orphan drug program and possible fixes.
KHN Video: Orphan Drugs Creating Gold Rush For Pharmaceutical Firms
The designation, which is made by the Food and Drug Administration, allows drugmakers to claim seven years of market exclusivity.
Sky-High Prices For Orphan Drugs Slam American Families And Insurers
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 Manipulate Orphan Drug Rules To Create Prized Monopolies
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.
FDA’s Drug Approval Team Copes With 700 Unfilled Jobs As Industry Lures Staff
The FDA’s drug-approval team is short more than 700 people and losing skilled staff members to the drug industry.
A Golden Ticket That Fast-Tracks A Drug Through The FDA
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.
Study Says Concerns About Orphan Drug Spending Are Unjustified
A study in Health Affairs concludes that orphan drugs for rare diseases are not having a widespread or deep impact on health care spending.
