NIH Recommends 88 Drugs Be Tested For Efficacy And Safety For Children

The National Institutes of Health (NIH) is urging the medical, pharmaceutical, and biopharmaceutical industries to focus their studies on a select group of pediatric therapeutic areas and medical products they say are of “elevated need” for kids in 2014.

Released last Monday, the “priority list” features 17 categories of treatments from cardiovascular disease to dermatologic conditions and includes 88 different drugs the agency says are top priority — including sildenafil (Viagra), epogen, fluconazole (Diflucan), and epinephrine.

Every year, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) reviews the prior year’s list of pediatric therapeutics, and based on the review, three therapeutic areas of interest are prioritized for the calendar year. Treatments that make the list are evaluated by six criteria:

• No disqualifying ethical concerns
• Level of evidence available and current gaps
• Potential impact on children, society, and delivery of care
• Consideration of the different populations that may benefit from the research
• Feasibility and availability of the resources needed to conduct the study

Problems can obviously arise when children are treated with medications that have only been tested in adults. 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) said, “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.”

But clinical trials are greatly lacking for medical conditions in which a large number of children are diagnosed. These children could benefit from new medical products if more pediatric trials would be done, according to a 2012 study led by author Dr. Florence Bourgeois.  The research looked at more than 2,400 clinical trials conducted in the previous five years and their respective enrollment of children.

Only 12 percent of trials focused on children and teenagers, even though kids accounted for 60 percent of those suffering with various conditions being studied, according to a Reuters report on the matter.

In response to this problem, the U.S. Congress passed two important pieces of legislation: The Best Pharmaceutical for Children Act (BPCA) and the Pediatric Research Equity Act (PREA). In combination, these laws provide new exclusivity-based incentives to conduct studies (BPCA), and these laws empower the FDA to require companies to conduct pediatric studies as a condition of approval (PREA).

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

BPCA and PREA also offer incentives like expanded market exclusivity for companies who would conduct pediatric testing. However, relatively few companies take advantage of such incentives, either because of a lack of interest, ethical or legal concerns, difficulty enrolling patients, or a lack of profits, RAPS reported.