Regulatory Focus™ > News Articles > Focusing on Pediatric Study Commitments, FDA Prepares to Name and Shame Noncompliant Companies

Focusing on Pediatric Study Commitments, FDA Prepares to Name and Shame Noncompliant Companies

Posted 26 August 2013 | By Alexander Gaffney, RAC

Pharmaceutical and biopharmaceutical companies, consider yourselves on notice-very, very public notice. In an announcement made on 26 August 2013 through its FDA Voice blog, the US Food and Drug Administration (FDA) said it will this week begin to publish letters of non-compliance for companies it has found to have missed deadlines to conduct pediatric studies required by federal law.


Historically, many companies seeking product approval in the US avoided clinical studies involving children, wary of the ethical problems, the lack of incentives, and the potential consequences if the testing brought up new adverse problems. As a result, many products lacked adequate dosing or safety information for children.

This, as regulators will tell you, is a problem. "We all know that children are not just small adults," wrote Lynne Yao, associate director of Pediatric and Maternal Health Staff in FDA's Center for Drug Evaluation and Research's (CDER) Office of New Drugs (OND). Some drugs may be super-potent in children relative to adults, while others may not work at all. In either case, there are inherent safety risks.

To remedy this situation, the US Congress passed into law two critical pieces of legislation: The Best Pharmaceutical for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), which combined had the effect of providing new exclusivity-based incentives to conduct studies (additional six months for pediatric written requests by FDA), as well as the ability for FDA to require companies to conduct pediatric studies as a condition of approval.

The effects of this legislation, Yao continued, have been substantial. "Before BPCA and PREA became law, more than 80% of the drugs approved for adult use were being used in children, even though the safety and effectiveness had not been established in children. Today that number has been reduced to about 50%."

A New Tactic: Name and Shame

But while many medicines now have information available for children as a result of these requirements-and we should note that not all drugs are intended to be used in children, and thus don't have pediatric requirements for testing-the FDA Voice posting by Yao makes clear that some companies aren't holding up their end of the bargain, and FDA is about to start calling them out in a very public way.

"If a sponsor has failed to seek or obtain a deferral extension, has failed to submit deferred pediatric studies by the final due date agreed to with FDA, or has failed to request approval for a required pediatric formulation, FDA can send a non-compliance letter to the sponsor and publish the letters on the web," Yao explained.

And those letters, Yao added, are going to be regularly published to an FDA webpage starting this week along with the sponsor's responses. "We believe this important step demonstrates our ongoing commitment to getting these studies done for the benefit of all infants and children," Yao said.

Variety of Reasons for Delays

Already, FDA has published four non-compliance letters: three from rare disease drug manufacturer Genzyme, one from Amedra Pharmaceuticals and one from The Medicines Company. The response letters indicate a range of reasons for delays.

Genzyme, for example, lists a long history of trying to get its studies off the ground, and notes that it has 40 patients enrolled awaiting the initiation of a trial for two drugs, and has been waiting since December 2010 to hear back from FDA on another proposed study.

Amedra, by contrast, had acquired the drug in question from another company, and said it is of the opinion that it needs a waiver due to unspecified reasons (contained in the letter but redacted, presumably to protect trade secrets). The Medicines Company, however, said safety issues and drug shortages had delayed its attempts to fulfill its pediatric study requirements, and is now seeking a deferral from FDA.

FDA Voice Posting

Pediatric Noncompliance Letters

Tags: Pediatric

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