Biotech Values

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Eteplirsen is considered an orphan drug by the FDA, which means it treats a disease that affects fewer than 200,000 patients. With less than 15,000 children living with DMD in the U.S., Eteplirsen actually falls into an even rarer category: it’s called an “ultra orphan drug” because the mechanism by which it works will have an effect on less than 2,000 children with the disease.

Historically, the FDA has used its ability to accelerate approval sparingly. In 2011, just 10% of 30 new drugs qualified under the agency’s accelerated approval program; in 2012, only four made the cut. The agency is under heavy scrutiny to make sure that drugs are safe before they’re made widely available. The pressure may be even more intense when it comes to drugs for children, whose developing bodies are still developing. “There are special protections in place for children who participate in research,” wrote Sandy Walsh, team leader for medical products and tobacco in the FDA Office of Public Affairs, in an email response to questions about the process.

In July, Congress passed new legislation called the FDA Safety and Innovation Act; it included a new designation known as “breakthrough” therapy that joins three other programs to speed the development and availability of “potentially really beneficial” drugs, says Erica Jefferson, deputy director of the FDA Office of Public Affairs.

The new legislation is not very different from existing laws that allow for accelerated approval, which were applied to bring AIDS drugs to dying patients as quickly as possible. Accelerated approval grants a conditional thumbs-up contingent upon further studies. Since 1993, accelerated approvals have shaved an average of six to seven months off the traditional processing time.

“We don’t want to introduce anything that is not going to work and does more harm than good,” says Jefferson, who is prohibited from discussing the specifics of any drug under review. “But this is an acknowledgment that there are treatments out there that can be potential game-changers

Read more: http://healthland.time.com/2013/02/07/both-my-sons-deserve-to-live-a-mothers-plea-for-quicker-action-from-the-fda/#ixzz2KekxxW22