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W-15: Pediatric Development of Molecularly Targeted Oncology Drugs

Poster Presenter

      Shivam Kamlesh Patel

      • Global Regulatory Affairs Fellow
      • University of North Carolina
        United States


To identify pediatric drug development opportunities for oncology products with planned requests for full waivers from pediatric studies in the iPSP’s submitted from 2012, when iPSP submissions at EOP2 were mandated under FDASIA, to 2016.


Initial PSPs for oncology drugs or biological products with plans to request full waivers from pediatric studies were reviewed for potential therapeutic use in children based on clinical experience or nonclinical evidence. In addition, Written Requests (WRs) issued by the FDA were reviewed.


Approximately 100 oncology drugs or biological products were reviewed with about 40 different molecular mechanisms of action identified and considered to search for potential therapeutic use in pediatric patients. Upon evaluation of available nonclinical evidence, approximately one-third of the products were found to have potential therapeutic use in at least one pediatric oncology indication based on molecular mechanism of action, but did not have pediatric studies conducted. Pediatric studies have been conducted in about half of the products reviewed; about 10% of products had no current evidence to support pediatric development as of the time of review (Feb 2017). Of the drug products reviewed, about one-third of the products received a WR, had a Proposed Pediatric Study Request (PPSR) plan, or was discussed at a Pediatric Subcommittee of the Oncologic Drugs Advisory Committee (pedsODAC) meeting. Of the products that did not have a WR or PPSR, one-third was studied by clinical trial networks or investigators in at least one pediatric indication.


Our findings highlight considerations for the early development of products for pediatric oncology indications. PREA has been of limited relevance to pediatric cancer drug development and exemptions to PREA often prevent pediatric patients from receiving early access to emerging investigational agents. The scope of PREA has been suggested to be broadened to require conduct of pediatric studies based on molecular mechanism instead of indication. Under BPCA, the 6-month marketing exclusivity incentive exists if pediatric data is submitted in accordance with an FDA-issued WR. Historically, many WRs were issued late in drug development or long after the approval of a drug for an adult cancer indication. The number of oncology product WRs issued by the FDA has steadily increased in the past few years than in prior years. Although there has been significant progress, our findings show that only one-third of approximately 100 products with planned requests for full waivers had a WR, PPSR, or underwent discussion at a pediatric ODAC meeting and highlights the need for further improvement. Continuous steps have been taken to promote pediatric oncology development including soliciting interest from sponsors and investigators to conduct pediatric studies and creating forums for FDA, investigators, and clinical trial networks to discuss potential therapeutic use of drugs with novel molecular mechanisms of action. Earlier communication and collaboration between the FDA, clinical trial networks and investigators, and sponsors to facilitate the identification and prioritization of emerging drugs of interest for WR consideration and early conduct of pediatric studies is encouraged. In conclusion, this analysis suggests that the decision to conduct pediatric studies should be based on molecular mechanism of action, rather than indication, to accelerate pediatric oncology drug development and improvement of outcomes for childhood cancers.

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