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FDA Releases New Guidance on Pediatric Drug Development

Updated: Feb 16



The U.S. Food and Drug Administration (FDA) has released two draft guidance documents on pediatric drug development. The guidance documents, "Pediatric Drug Development Under PREA and BPCA Scientific Considerations" and "Pediatric Drug Development Complying with PREA and BPCA Regulatory Considerations," provide important information for industry sponsors who are developing drugs, biological products, and vaccines for pediatric use.

The U.S. Food and Drug Administration (FDA) has released two draft guidance documents on pediatric drug development. The guidance documents, "Pediatric Drug Development Under PREA and BPCA Scientific Considerations" and "Pediatric Drug Development Complying with PREA and BPCA Regulatory Considerations," provide important information for industry sponsors who are developing drugs, biological products, and vaccines for pediatric use.


The two guidance documents discuss a variety of topics, including the rationale for pediatric drug development, the types of pediatric studies that may be required, the selection of appropriate study populations, the design and conduct of pediatric studies, the analysis and interpretation of pediatric study data, and the process for qualifying for pediatric exclusivity.


Key takeaways from the guidance documents include:

  • The FDA's Pediatric Research Equity Act (PREA) and Best Pharmaceuticals for Children Act (BPCA) require drug manufacturers to study the safety and effectiveness of their products in pediatric populations.

  • The FDA's draft guidance provides important information for industry sponsors who are developing drugs for pediatric use.

  • The guidance discusses the rationale for pediatric drug development, the types of pediatric studies that may be required, and the selection of appropriate study populations.

  • The guidance also provides information on the process for qualifying for pediatric exclusivity.

The Importance of Pediatric Drug Development

Pediatric drug development is essential to ensure that children have access to safe and effective medicines. Children are not simply small adults, and their bodies respond differently to drugs. As a result, it is often necessary to conduct pediatric studies to determine the safety and effectiveness of drugs in children.


The FDA's PREA and BPCA laws are designed to encourage pediatric drug development. PREA requires drug manufacturers to conduct pediatric studies of their products if the FDA determines that such studies are likely to yield clinically meaningful information. BPCA provides a marketing exclusivity period of up to 6 months for drug manufacturers who conduct pediatric studies of their products.


The FDA's Guidance Documents

The FDA's draft guidance documents provide important information for industry sponsors who are developing drugs for pediatric use. The guidance discusses the regulatory framework for pediatric drug development, the process for submitting pediatric study plans and reports to the FDA, and the process for qualifying for pediatric exclusivity. The guidance also provides information on the FDA's enforcement policies for pediatric drug development.


Specific Requirements

The FDA's guidance documents also provide more specific information about the types of pediatric studies that may be required, the selection of appropriate study populations, and the design and conduct of pediatric studies. The guidance also provides information on the process for submitting pediatric study plans and reports to the FDA, and the process for qualifying for pediatric exclusivity.


Here are some specific examples of the types of pediatric studies that may be required:

  • Safety studies: These studies are designed to assess the safety of a drug in children. They typically involve measuring the drug's side effects and its effects on a child's growth and development.

  • Efficacy studies: These studies are designed to assess the effectiveness of a drug in children. They typically involve comparing the drug to placebo or another treatment option.

  • Pharmacokinetic studies: These studies are designed to assess how a drug is absorbed, distributed, metabolized, and excreted in children. This information is important for determining the appropriate dose of the drug for children.

The FDA's guidance documents also discuss the selection of appropriate study populations. The FDA recommends that drug manufacturers consider the following factors when selecting study populations:

  • The age of the children

  • The medical condition that the drug is being studied for

  • The availability of alternative treatment options

  • The potential benefits of the drug for children

The FDA's guidance documents also provide information on the design and conduct of pediatric studies. The FDA recommends that drug manufacturers follow the principles of good clinical practice (GCP) when conducting pediatric studies. GCP is a set of international standards for the design, conduct, recording, and reporting of clinical trials.


Conclusion

The FDA's release of the two draft guidance documents is an important step in ensuring that children have access to safe and effective medicines. The guidance documents provide valuable information for industry sponsors who are developing drugs for pediatric use. The FDA is accepting comments on the draft guidance documents until July 17, 2023. We encourage you to review the guidance documents and submit your comments to the FDA.

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