The independent source for health policy research, polling, and news.
Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives but nearly a third of those products aren鈥檛 new or were repurposed multiple times, an investigation shows.
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.
1-20 of 30 results
Patients who depend upon special drugs to treat rare diseases are caught in the crossfire as drugmakers and the FDA battle over regulations that reward companies for developing treatments for relatively small pools of patients.
Josie and George Taylor of Everett, Washington, are two of the first people in the U.S. to recover from novel coronavirus infections after joining a clinical trial for the antiviral drug remdesivir.
Sen. Mike Enzi said he knew of a foundation that would import insulin for patients, but it doesn鈥檛 appear to exist.
A probe by the Government Accountability Office cites breakdowns in the Food and Drug Administration program that approves drugs for rare diseases.
Desperate for help in finding a lifesaving drug for a fatal genetic disease, families banded together to fund early research and then worked with drug companies on clinical trials and marketing. Yet, this small patient advocacy group is stunned by pharma鈥檚 pricing.
An abortion drug invented decades ago is being used to treat Cushing鈥檚 syndrome 鈥 and it鈥檚 bringing in tens of millions of dollars a year.
The FDA鈥檚 Scott Gottlieb says the agency is focused on the big picture, and he wants to know why pharma churns out drugs for some rare diseases but not for others.
Some of the nation鈥檚 most influential scientists recommend eight steps to lower drug prices. KHN takes the political temperature and tells you the chances of Congress acting on them.
Even though congressional Republicans set aside their Obamacare repeal-and-replace efforts this year, here are five major health policy changes that could become law as part of the pending House and Senate proposals.
The House and Senate want to reduce or eliminate federal tax credits for 鈥渙rphan drugs鈥 used to treat rare diseases, but patients are fighting against the plan.
House Republicans want to repeal federal tax credits that have helped spur a boom in orphan drugs for rare diseases.
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 鈥渙rphan drugs鈥 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?
漏 2026 KFF