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  1. Oncology Center of Excellence

Pediatric Oncology

The mission of the Oncology Center of Excellence Pediatric Oncology Program is to promote the development of safe and effective new drugs and biologics to treat cancer in children. To do this, we attempt to maximize the authority available through the Best Pharmaceuticals for Children Act (BPCA) to increase the number of Written Requests for pediatric studies of appropriate new drugs developed for adult cancers much earlier in the development timeline.

Recent amendments (FDARA 2017) to the Pediatric Research Equity Act (PREA) provide enhanced opportunities to extend the promise of precision medicine to children with cancer. The FDA, with input from the National Cancer Institute and the pediatric cancer research community, developed a Pediatric Molecular Target List to comply with the amended provisions of PREA to provide some guidance to industry in planning for new drug and biologic submissions.

The Pediatric Oncology Program holds Pediatric Oncology Product Development Early Advice Meetings with sponsors to discuss pediatric development plans. We also hold various meetings with stakeholders, including the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee, the Oncology Subcommittee of the Pediatric Review Committee (PeRC), as well as other public outreach meetings.

Pediatric Molecular Target List

This list includes 1) molecular targets for which existing evidence and/or biologic rationale exist to determine their potential relevance to the growth or progression of one or more pediatric cancers and 2) those targets for which there is evidence that they are not associated with the growth or progression of pediatric tumors for which requirement for early pediatric evaluation of drugs and biologics which are directed at these targets would be waived.

The list fulfills the statutory requirements imposed by FDARA and is expected to provide some guidance to industry in planning for initial Pediatric Study Plan submissions for new drug and/or biologic products in development for cancer in accordance with the amended provisions of the Pediatric Research Equity Act.

Relevant Molecular Targets

Non-relevant Molecular Targets That Warrant Waiver from Required Evaluation

Contact Information

For more information, please contact OCEPeRC@fda.hhs.gov

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