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.
The independent source for health policy research, polling, and news.
1,081 - 1,100 of 1,290 Results
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.
This immunization may mark a shift among some vaccine makers to higher-priced, “niche” preventives that protect against very specific and sometimes rare illnesses.
After a Kaiser Health News report on an offshore herpes vaccine trial that skirted FDA regulations, St. Kitts and Nevis officials claim they had no knowledge of the testing. An investigation is underway.
The USA's first approved gene therapy — to be used to fight leukemia that resists standard therapies — will cost $475,000 for a one-time treatment.
In this Facebook Live, KHN’s Julie Appleby talks with Stephanie Stapleton and answers readers’ questions about the prescription drug pricing pipeline and the industry stakeholders who have a role in what you pay.
Prominent businessmen and an American university supported offshore testing of an experimental vaccine.
A genetically altered cancer drug, based on CAR T-cell therapies, could be a big success with leukemia patients but at a staggering cost.
As more patients receive hospice care at home, some of the powerful, addictive drugs they’re prescribed are ending up in the wrong hands.
In this episode of “What the Health?” Mary Agnes Carey of Kaiser Health News, Sarah Karlin-Smith of Politico, Margot Sanger-Katz of The New York Times and Julie Appleby of Kaiser Health News discuss the recent extension of cost-sharing subsidies for millions of low-income beneficiaries on the Affordable Care Act’s marketplaces and the state of play on Capitol Hill and in the states concerning initiatives to lower prescription drug costs.
Medicaid spent billions more in 2016 than the year before on decades-old prescription drugs, including many generics, a Kaiser Health News data analysis shows.
Hospital use of two popular heart medicines, nitroprusside and isoproterenol, dramatically dropped after the prices for both soared.
Three years ago, only about a quarter of the nation’s large employers were very confident they would have a health plan in 10 years. That number has now risen to 65 percent.
Some drug courts offer participants a full range of evidence-based treatment, including medication-assisted treatment. Others don't allow addiction medications at all. And some permit just one: Vivitrol.
The new law will help people with chronic conditions that require multiple prescriptions cut down on their shuttles to the drug store and could improve adherence to their drugs.
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.
Embattled opioid seller Mallinckrodt is one of many pharmaceutical companies boosting political contributions and lobbying on Capitol Hill.
The controversial 340B drug discount program for hospitals came under fire at a congressional hearing.
A little-noticed provision in President Donald Trump’s executive order on drug prices may offer a clue to why Big Pharma hasn’t opposed a bill that could bleed their balance sheets of millions of patients.
Report by CDC researchers finds a steady fall in opioid use in recent years, but the rates are still three times higher than in 1999.
© 2026 KFF