Sen. Mike Enzi said he knew of a foundation that would import insulin for patients, but it doesn’t 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’s pricing.
An abortion drug invented decades ago is being used to treat Cushing’s syndrome — and it’s bringing in tens of millions of dollars a year.
The FDA’s 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’s 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 “orphan drugs” 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.
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.