Tag Archives: price

The most expensive medicine ever treats a rare condition. The price: $2.13 million – Los Angeles Times

Evelyn Villarreal was one of the first children treated, at 8 weeks. Her family, from Centreville, Va., had lost their first child to spinal muscular atrophy at 15 months. Two years later when Evelyn was born a test showed she also had the disease, so the family enrolled her in the gene therapy study at… Read More »

Senate health committee takes on surprise bills, price transparency in legislative package

Dive Brief: The leaders of the Senate HELP Committee on Thursday proposed a wide-ranging and ambitious package of healthcare bills, including measures addressing surprise medical billing, price transparency, interoperability, drug costs and public health. One bill put forward would require commercial insurers to make claims data and expected out-of-pocket costs available to patients through application… Read More »

NICE blames BioMarin for Brineura rejection after price talks break down

BioMarin and England’s cost-effectiveness gatekeeper have been negotiating for a year, but that hasn’t helped the rare-disease specialist secure reimbursement for its Batten disease drug Brineura. Friday, the National Institute for Health and Care Excellence (NICE) published draft guidance sticking with its earlier decision not to endorse Brineura in children with the disease, an inherited… Read More »