Drug Companies Faulted for Not Testing Drugs in Kids

August 28, 2013

The US Food and Drug Administration (FDA) is faulting 3 pharmaceutical companies for not complying with a federal law that requires them to study their products in children.

The FDA chided the companies in noncompliance letters mailed out this spring. The letters were posted on the FDA Web site August 27, along with responses from the pharmaceutical companies.

In a blog post on the agency's Web site, an administrator in the FDA's Center for Drug Evaluation and Research (CDER) said the letters demonstrate "our ongoing commitment to get these studies done for the benefit of all infants and children."

"We all know that children are not just small adults," said Lynne Yao, MD, associate director of the pediatric and maternal health staff in CDER's Office of New Drugs. "Many changes occur in children as they grow and develop that can affect how a drug works. In fact, some drugs that may work in adults may not work at all in children. There may be different safety concerns compared to when they are used in adults, or they may need to be given in a different dose."

Genyzme received 2 letters regarding its tablet and oral suspension versions of sevelamer carbonate (Renvela), indicated for controlling serum phosphorus in patients with chronic kidney disease who are receiving dialysis, and another letter for doxercalciferol (Hectorol), approved to reduce levels of parathyroid hormone in patients with kidney disease. In addition, the FDA sent a letter to Amedra Pharmaceuticals about its epinephrine injection products (Twinject and Adrenaclick) for severe allergic reactions. The Medicines Company also received a noncompliance letter about clevidipine butyrate injectable emulsion (Cleviprex), indicated for hypertension.

All of the FDA letters state that the companies had missed their deadline to submit an assessment of how their products work in children. In each case, the FDA had allowed the companies to postpone these pediatric studies.

Genzyme replied that its required studies for the tablet and oral suspension forms of sevelamer are "well underway" and promised to deliver them by June 2016. The company said it missed its December 2011 deadline because the FDA had not signed off on study designs until the due date was less than 6 months away. Genzyme blamed the missed deadline for doxercalciferol on communication breakdowns with the FDA but nevertheless vowed to submit the pediatric study by June 2016.

The Medicines Company set August 1, 2016, as the probable delivery date for its pediatric study on clevidipine. It said that a manufacturing hiatus as well as FDA vacillation about the very need for the study ate up most of the 36 months it was given to meet the original deadline of August 1, 2011.

In its letter, Amedra said it has asked the FDA to waive the study regarding epinephrine injection for children weighing less than 15 kg that the agency said was required by law. The redacted version of the Amedra letter posted online does not offer a rationale for the waiver.

2 Laws, More Pediatric Drug Studies

In her blog post, Dr. Yao said the agency issued the noncompliance letters under the authority of the Pediatric Research Equity Act (PREA) of 2003, which mandates drug companies perform pediatric studies of their products in some cases. The FDA can defer these studies in light of the company's circumstances, but "deadlines for deferred studies have often been missed," Dr. Yao wrote.

Last year, when Congress reauthorized PREA, it amended the law to give the FDA new tools to discourage companies from missing their deadlines. If a company fails to submit deferred studies by the final due date, fails to seek or obtain a deferral extension, or fails to ask the agency to approve a required pediatric formulation, the agency can send out the noncompliance letter and post it online.

Before the passage of PREA and a similar 2002 law called Best Pharmaceuticals for Children Act, which financially rewards companies for conducting pediatric studies, more than 80% of drugs approved for adults also were used in children even though their effectiveness and safety in that population had not been determined, according to Dr. Yao.

"Today, that number has been reduced to about 50%," said Dr. Yao.

More information about the FDA's new push to have drugs studied in children is available on the agency's Web site.

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