FDA Approves Beta-Thalassemia Gene Treatment Costing $2.8M Per Patient
The treatment is made by Bluebird Bio, and is reported to represent a potential cure for the rare blood disorder. The Wall Street Journal notes it will be one of the most expensive drugs in the U.S. The treatment, called Zynteglo, had been approved in Europe but its costs were deemed prohibitive.
The Wall Street Journal:
FDA Approves Bluebird’s $2.8 Million Gene Therapy For Rare Blood Disease
The Food and Drug Administration approved a gene therapy from Bluebird Bio Inc. that represents a potential cure for a rare blood disorder at a price of $2.8 million per patient, making it one of the nation’s most expensive drugs. The FDA on Wednesday cleared the treatment, Zynteglo, for patients who rely on regular blood transfusions because of a genetic disease called beta-thalassemia, which interferes with the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. (Walker, 8/17)
The Boston Globe:
Bluebird Bio’s First Gene Therapy For Rare Blood Disease Wins FDA Approval
Bluebird won approval for Zynteglo in Europe in 2019 but eventually shuttered its commercial operations there when payers refused to cover its previous $1.8 million price tag. The therapy treats the severe form of a disease called beta thalassemia, where a genetic mutation in the beta-globin gene impairs the ability of blood cells to carry oxygen throughout the body. People with the condition require regular blood transfusions once or twice a month, and chelation therapies to reduce high levels of iron caused by the transfusions. (Cross, 8/17)
In other research —
The New York Times:
New Method Improves Speed And Cost Of Birth Defect Testing
After 10 years of effort, medical researchers at Columbia University have developed a very fast and cheap way to detect the extra or missing chromosomes that most often cause miscarriages or severe birth defects. The method, described Wednesday in the New England Journal of Medicine, takes less than two hours using a palm-size device and costs $200 per use. With current testing procedures, women can end up paying $1,000 to $2,000, often out of pocket. (Kolata, 8/17)
Axios:
Some NIH-Funded Trials Never Reported Results
The National Institutes of Health failed to sanction institutions and labs running government-funded clinical trials for not reporting their results despite requirements to do so, according to a new report from a federal watchdog. (Dreher, 8/17)
On hearing aids —
The Washington Post:
Over-The-Counter Hearing Aids Will Soon Be Available. Here's What To Know.
The new class of devices will be designed to help people with self-perceived mild to moderate hearing loss, according to Barbara Kelley, executive director of the Hearing Loss Association of America, which has put together a tip sheet to guide consumers who stand to benefit. (Sellers, 8/17)
NPR:
Over-The-Counter Hearing Aids Will Bring Relief, But With Some Confusion
"Medicare doesn't cover hearing aids or exams for fitting hearing aids," the government program's website states. "You pay 100% of the cost for hearing aids and exams." Many Americans might be surprised that the federal insurance program doesn't offer help to people who are most likely to need hearing aids. But it's by design. (Chappell, 8/17)
