By: Lynne Yao, M.D.
We all know that children are not just small adults. Many changes occur in children as they grow and develop that can affect how a drug works. In fact, some drugs that work in adults may not work at all in children. There may be different safety concerns compared to when they are used by adults, or they may need to be given in a different dose. That’s why products that are used in children must be studied in children.
Congress enacted two laws that will increase the study of drugs in children: The Best Pharmaceuticals for Children Act (BPCA) provides an incentive for drug companies to conduct FDA-requested pediatric studies by granting an additional six months of marketing exclusivity. The Pediatric Research Equity Act (PREA) requires drug companies to study their products in children under certain circumstances. When pediatric studies are required, they must be conducted with the same drug and for the same use for which they were approved in adults.
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%.
Under PREA, FDA can waive studies in children if the studies are not necessary. For example, if the disease for which the drug is being used in adults does not exist in children, such as prostate cancer, FDA would waive studies for children. In some cases, FDA has allowed sponsors to defer pediatric studies, depending on the circumstances. However, deadlines for deferred studies have often been missed.
Fortunately, FDA now has tools to discourage companies from missing deadlines for deferred pediatric studies. When Congress reauthorized PREA last year as part of the Food and Drug Administration Safety and Innovation Act, or FDASIA, it gave FDA new authorities. FDA can grant extensions for deferred pediatric studies at a sponsor’s request if there is good cause for a delay in completing the studies. For example, if the sponsor has diligently attempted to recruit patients, but is having difficulty recruiting enough pediatric patients to complete the study, FDA can grant a deferral extension. However, 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.
This week, FDA is publishing the first of these non-compliance letters and the sponsors’ responses. They will be posted to a public FDA web page on an ongoing basis. We believe this important step demonstrates our ongoing commitment to getting these studies done for the benefit of all infants and children.
Lynne Yao, M.D., is Associate Director, Pediatric and Maternal Health Staff, in FDA’s Center for Drug Evaluation and Research’s Office of New Drugs