• Mission
• Pediatric Molecular Target List
• Common Commentary for EMA and FDA Scientific Review of Pediatric Cancer Drug Development Plans

Mission

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

• Relevant Pediatric Molecular Target List without references
• Relevant Pediatric Molecular Target List with references

Non-relevant Molecular Targets That Warrant Waiver from Required Evaluation

• Non-Relevant Molecular Target List without references
• Non-Relevant Molecular Target List with references

Common Commentary for EMA and FDA Scientific Review of Pediatric Cancer Drug Development Plans

The evolving regulatory landscape surrounding pediatric cancer drug development in the U.S. and EU has more closely aligned the timelines for required submission of plans for pediatric development of appropriate new cancer drugs and biologics developed for adults with cancer. This has created an opportunity for sponsors to seek preliminary scientific advice from both the EMA and the FDA on Pediatric Investigation Plans (PIPs) and initial Pediatric Study Plans (iPSPs) through the Pediatric Cluster Calls coordinated by the FDA’s Office of Pediatric Therapeutics.

Given the global nature of cancer drug development and the relative rarity of childhood cancer, which impacts study populations for clinical trials, the demand for international collaboration in study design and conduct has intensified. We have recommended that new PIPs and iPSPs for new cancer products be submitted to their respective agencies simultaneously to promote global coordination and international research collaboration (Reaman et al. J Clin Oncology, 2020).

This Common Commentary template is provided to demonstrate to sponsors the structure of our scientific discussions and review in the context of regulatory agency positions on selected aspects of pediatric drug development.

Contact Information

Email: OCEPeRC@fda.hhs.gov