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The U.S. Food and Drug Administration (FDA) has published two draft guidance documents focused on pediatric drug development. These guidance documents are intended to provide industry sponsors with information that provides insights on pediatric drug development under the provisions of Pediatric Research Equity Act (PREA) and The Best Pharmaceuticals for Children Act (BPCA) for drugs, biological products, and vaccines used in healthy pediatric populations.
The guidance documents address the range of issues surrounding pediatric drug development, including the need for pediatric studies, the kinds of pediatric studies that may be needed, how to choose study populations and design pediatric studies; how to analyze clinical trial data in children and interpret those analyses; and how to qualify for exclusivity.
The guidance documents include several key takeaways to note:
Specifically, to ensure drug manufacturers study the safety and effectiveness of their products in pediatric populations, the FDA’s Pediatric Research Equity Act (PREA) and Best Pharmaceuticals for Children Act (BPCA) set forth that new drugs must be studied across a variety of age ranges.
The draft guidance helps inform industry sponsors developing drugs intended for pediatric use.
The guidance describes the reasons for developing drugs in the pediatric population, what types of pediatric studies may be necessary, and how to choose appropriate study populations.
It also outlines how to qualify for pediatric exclusivity.
Why Pediatric Drug Development is Important
Drugs need to be developed for children together with any new medicines that they are likely to use to maintain a safe and effective therapeutic arsenal. Kids are not little adults, and their bodies react differently to drugs. Consequently, paediatric studies are often required to ensure drugs are safe and proven effective in children.
Overview of PREA and BPCA, two pediatric drug development incentive laws: If the FDA agrees that there is a high probability of obtaining clinically meaningful information, then PREA mandates that drug manufacturers perform pediatric studies of their products. BPCA gives drug manufacturers up to 6 months of exclusivity for their products if they perform pediatric studies.
The FDA’s Guidance Documents
FDA draft guidance documents contain information of particular interest to sponsors of drugs with a pediatric indication. This guidance outlines the regulatory perspective, including the requirements for submission of a pediatric study plan (PSP) and whether to include this information in a new drug application (NDA), as well as how to qualify for pediatric exclusivity by submitting an additional report. It also provides information on FDA’s enforcement policies with regard to drug development in the pediatric population.
Specific Requirements
It includes more specificity on pediatric studies — the types of which may be needed, how to choose study populations and design and conduct a pediatric study (FDA 2019). Information included will address the procedures for submitting proposed pediatric study plans and reports to the FDA, as well as those for qualifying for pediatric exclusivity.
We have the examples of what type of pediatric studies might be, specifically:
Safety studies: These are aimed at determining how safe a drug is for use in children. They usually consist of tests for the side effects of the drug, as well as its effect on a child’s growth and development.
Efficacy studies: Studies that look at the effectiveness of a given drug with children. Usually, only by comparing the drug with placebo or other treatment.
Pharmacokinetic studies: These are designed to determine how a drug is absorbed, circulated, broken down, and disposed of in children. It is important in calculating the right amount of the drug to give children.
Other FDA guidance documents address important considerations, such as how best to identify populations most likely to benefit from drugs. The FDA encourages drug manufacturers to consider the following when choosing study populations:
The age of the children
Condition under investigation as an indication for treatment
Other treatment options
Advantages of the drug for youngsters
The design and conduct of pediatric studies are also addressed by some FDA guidance documents. The FDA advises that drug manufacturers adhere to good clinical practice (GCP) principles when performing pediatric trials. GCP — It is an international quality standard that is followed during the designing, conducting, recording and reporting of clinical trials.
Conclusion
The release of the two draft guidance documents by the FDA is a step in the right direction for getting safe and effective medicines into children. The guidance documents are informative for industry sponsors developing drugs with pediatric indication. We urge you to read the guidance documents and send your comments to the FDA.
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