FDA Awards Funding to Support Pediatric Clinical Trials Research

By: Susan McCune, M.D.

Despite many efforts over the past two decades to increase the number of FDA-approved therapies for children, some 60 percent of drugs that are used in children are not approved for pediatric use, and the percentage of FDA-approved options is even smaller for neonates, with 90 percent of drugs used in this population unapproved for neonatal use.

Susan McCune, M.D.FDA is taking steps to address this problem.

These drugs have not been approved for use in children because they have not been successfully tested in pediatric clinical trials. In fact, a recent study found a failure rate of up to 42% for trials that were done under the 2002 Best Pharmaceuticals for Children Act (BPCA), which grants an additional 6 months of marketing exclusivity for companies that study the drug in children.

It is not clear whether these failures mean the drugs do not work in children or whether investigators did not select the right drug, identify the right patient population, choose the right dose, use the right trial design, or identify the right endpoints for their trials. Many sponsors also said they were unable to complete their studies because they couldn’t recruit subjects for the trials.

FDA believes it is critical to support the development and maintenance of a scientific and organizational infrastructure that can plan, start up, conduct, and close out pediatric clinical investigations. That’s why we decided to issue awards to facilitate pediatric clinical trials and pediatric trial-related research to the Institute for Advanced Clinical Trials for Children (IACT for Children) and Duke University.  Each awardee will receive $1 million for this year under the Global Pediatric Clinical Trials Network Cooperative Agreement.

To optimize the potential for successful pediatric clinical trials, the scientific community must accomplish a number of goals:

  • Leverage basic science research on the causes of diseases to support the use of extrapolation from adult data
  • Understand the maturation of metabolic pathways to be able to use modeling and simulation to optimize dosing strategies in pediatric patients
  • Develop innovative trial designs, including protocols that are standardized and can study multiple therapies at one time, thus optimizing the use of data from multiple sources
  • Leverage and standardize all sources of information including clinical trials, registries, natural history studies, and electronic health records
  • Develop multiple types of biomarkers for use in pediatric trials
  • Develop clinically meaningful short- and long-term efficacy and safety endpoints for pediatric trials

Bringing stakeholders into a network that can provide best practices, innovative approaches, and streamlined conduct of regulatory quality clinical trials can increase the efficiency of pediatric studies, better ensuring the quality and efficiency of pediatric therapeutic development. Successful pediatric trials will result in data to support up-to-date labeling that informs use in children.

Innovations in trial design and new technologies could contribute substantially to increasing pediatric trial successes. For example, in a recent article, Janet Woodcock, CDER Center Director and Lisa LaVange, Director of CDER’s Office of Biostatistics, noted that building a common trial network and associated infrastructure can afford considerable advantages in both efficiency and quality during drug development. Having a network of experienced clinical centers to serve as sites to study multiple interventions makes sense as compared with establishing study centers one trial at a time. And the use of a single system for clinical data management will enable shorter start-up times as the protocol is expanded to incorporate new investigations, they said.

Through the FDA grants, we expect IACT for Children and Duke University will be able to help academia and industry-based institutions overcome some of the historic challenges posed by pediatric clinical trials. They can help optimize pediatric study designs, protocols, best practices, and training, and they can engage stakeholders. They can provide expert advice to investigators, streamline and improve processes and systems to enhance trial quality, and reduce administrative burdens with the ultimate goal of establishing pre-qualified, trial-ready sites. Through the Global Pediatric Clinical Trials Network Cooperative Agreement, FDA will work with IACT for Children and Duke University to make a meaningful contribution to advances in pediatric clinical research, resulting in scientifically validated information to support the labeling of many more medical products for safe and effective use in children.

Susan McCune, M.D., is FDA’s Director, Office of Pediatric Therapeutics

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