Orphan drugs

A recent clinical trial showed that a drug used for a small coterie of patients suffering from rare inflammatory disorders can reduce the risks of serious complications in people who have suffered a heart attack.

The FDA has approved the first new treatment for patients with sickle cell disease in almost 20 years.

More than half (53%) of the 111 respondents to an online survey rated orphan drugs as having a major impact on rising drug costs, although there was also fairly wide (if mild) agreement that the 1983 Orphan Drug Act has been successful in creating incentives to develop remedies for rare diseases.
Sales of orphan drugs are forecast to grow 11% over the next five years, to $209 billion. That growth rate is twice as fast as the expected increase in sales of all other prescription medicines. They may account for more than 21% of brand-name prescription drug sales by 2022.
In some respects, the 1983 Orphan Drug Act is a success story. But high prices and allegations that some drug companies have twisted the law to their advantage have made it controversial. Here are some of the main points in the debate.
Sales hit the billion dollar mark as pharmaceutical companies apply creative approaches to increase earnings. For instance, “salami slicing”: Dividing diseases into smaller and smaller categories based on genetic and biomarker differences so that the product can achieve the coveted orphan drug status.

Acting on a request from three influential senators, the Government Accountability Office (GAO) has confirmed that it will investigate potential abuses of the Orphan Drug Act (ODA), according to a report from Kaiser Health News (KHN).

Building on weeks of mounting pressure to address high prescription drug prices, three influential senators have asked the government’s accountability arm to investigate potential abuses of the Orphan Drug Act, according to a report from Kaiser Health News (KHN).

A few weeks ago, Jeffrey Aronin, CEO of Marathon Pharmaceuticals, announced his decision to charge $89,000 per year for deflazacort––a cheap, generic steroid––for patients with Duchenne muscular dystrophy. Now a group of irate senators wants some answers.

Orphan drugs have been in the spotlight recently for reaping hefty profits from tiny patient populations. Some major new prospects are coming down the late-stage pipeline. An article in Endpoint News highlights the top 10 orphan drugs headed for potential FDA approval.