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Molecularly Targeted Therapies in Pediatric Cancer

Molecularly Targeted Therapies in Pediatric Cancer

To access a meeting recap of this archived event, please Click HERE.

Molecularly Targeted Therapies in Pediatric Cancer

Tuesday, February 20, 2018

8:00 AM EST – 1:00 PM EST

JW Marriott
1331 Pennsylvania Ave NW
Salon DE
Washington, DC 20004

Thank you to all attendees! Click HERE for the finalized meeting slides.

Friends of Cancer Research is proud to host a half-day meeting where invited members of the FDA, NIH, industry, academia, and patient advocacy communities will discuss several important considerations that will help identify scientifically-driven approaches for the creation of a list of molecular targets considered substantially relevant in pediatric cancers.

Robust drug development for pediatric cancers is critical to ensuring appropriate access to life-saving drugs and promoting innovation. Title V of the FDA Reauthorization Act (FDARA) of 2017, updates the Pediatric Research Equity Act (PREA), which mandates any sponsor submitting an original application for a cancer product directed at a molecular target that is determined to be substantially relevant to the growth or progression of pediatric cancer, to conduct a molecularly targeted pediatric cancer investigation designed to yield clinically meaningful pediatric study data to inform potential pediatric labeling.

The meeting will facilitate discussion on transparent and scientifically sound processes that address the following provisions:

  1. Developing the molecular target lists: Forming frameworks of factors that may guide the definition of molecular targets as substantially relevant or not relevant to the growth or progression of one or more pediatric cancers
  2. Updating the lists of molecular targets: Defining mechanisms and timelines by which such updates may occur
  3. Applying the molecular target lists: Addressing key considerations in the application of the lists to pediatric cancer drug development

Click below to register and keep scrolling down for the finalized agenda for the meeting.

Finalized Agenda

8:00am – Registration and Breakfast

8:30am – Welcoming Remarks

Ellen Sigal, Friends of Cancer Research

8:35am – FDA Perspective on the Implementation of FDARA: Opportunities for Pediatric Precision Oncology Medicine

Gregory Reaman, U.S. FDA

8:50am – Presentation: Framework to Define Potential Relevance of Molecular Targets

Malcolm Smith, NCI

9:00am – Panel 1 Discussion: Molecular Targets in Pediatric Cancers – Framework and List


Malcolm Smith, NCI (moderator)
Scott Armstrong, Dana-Farber Cancer Institute
Nancy Goodman, Kids V Cancer
Katherine Janeway, Dana-Farber Cancer Institute
Gregory Reaman, U.S. FDA
Martina Uttenreuther-Fischer, Boehringer Ingelheim

10:00am – Q&A

10:30am – Break

10:45am – Presentation: Updating the Molecular Target List – Translating Emerging Science

Peter Ho, Boston Pharmaceuticals

10:55am – Panel 2 Discussion: Processes for Updating the Molecular Target List


Peter Ho, Boston Pharmaceuticals (moderator)
Albert J Allen, Eli Lilly and Company
Martha Donoghue, U.S. FDA
Danielle Leach, St. Baldrick’s Foundation
Rajen Mody, University of Michigan School of Medicine
Nita Seibel, NCI

11:25am – Q&A

11:45am – Presentation: Applying the Molecular Target List to Cancer Drug Development for Pediatrics

Brenda Weigel, University of Minnesota

11:55am – Panel 3 Discussion: Considerations for the Application of a Molecular Target List to Cancer Drug Development for Pediatrics


Brenda Weigel, University of Minnesota (moderator)
Peter Adamson, Children’s Hospital of Philadelphia
Jo Lager, Sanofi
Charles Mullighan, St. Jude Children’s Research Hospital
Susan Weiner, Children’s Cause for Cancer Advocacy
Lynne Yao, U.S. FDA

12:35pm – Q&A

12:55pm –  Closing Remarks

Gregory Reaman, U.S. FDA

Jeff Allen, Friends of Cancer Research