A Bitter Battle Over the ‘Orphan Drug’ Program Leaves Patients’ Pocketbooks at Risk
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.
‘You Pray That You Got The Drug.’ Ailing Couple Gambles On Trial For COVID-19 Cure
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.
False Lead: Senator’s Offer To Help Patient Import Cheap Insulin Goes Nowhere
Sen. Mike Enzi said he knew of a foundation that would import insulin for patients, but it doesn’t appear to exist.
Government Investigation Finds Flaws In the FDA’s Orphan Drug Program
A probe by the Government Accountability Office cites breakdowns in the Food and Drug Administration program that approves drugs for rare diseases.
The High Cost Of Hope: When The Parallel Interests Of Pharma And Families Collide
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.
How A Drugmaker Turned The Abortion Pill Into A Rare-Disease Profit Machine
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.
FDA Chief Says He’s Open To Rethinking Incentives On Orphan Drugs
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.
Experts Tell Congress How To Cut Drug Prices. We Give You Some Odds.
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.
Congress Isn’t Really Done With Health Care — Just Look At What’s In The Tax Bills
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.
Patients With Rare Diseases And Congress Square Off Over Orphan Drug Tax Credits
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 Aim To Yank Tax Credits For Orphan Drugs
House Republicans want to repeal federal tax credits that have helped spur a boom in orphan drugs for rare diseases.
Without Price Breaks, Rural Hospitals Struggle To Stock Costly, Lifesaving Drugs
A federal drug program blocks rural hospitals from getting discounts on rare-disease drugs, forcing staff to cut back on supplies of lifesaving medicines.
FDA Moves To Guard Against Abuse Of ‘Orphan Drug’ Program
Following a KHN 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.
Denial, Appeal, Approval … An Adult’s Thorny Path To Spinraza Coverage
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.
Drug Puts A $750,000 ‘Price Tag On Life’
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.
Drugmakers Help Turn Patients With Rare Diseases Into D.C. Lobbyists
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.
GAO To Launch Investigation Of FDA’s Orphan Drug Program
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.
$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.
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?
