• Skip to primary navigation
  • Skip to main content
  • Skip to primary sidebar
  • Skip to footer

Kids v Cancer

Changing the landscape of pediatric research

DONATE
  • Who We Are
    • About Us
    • About Jacob
    • Mission and Impact
    • Press Kit
    • Contact
  • Policy Development
    • RACE for Children Act / PREA
    • Creating Hope Act / Pediatric Priority Review Vouchers
    • Kids’ Eligibility for Trials
  • For Doctors & Families
    • COMPASSIONATE USE NAVIGATOR
    • Brain Tissue Donation
  • News & publications
    • News
    • Pediatric Cancer Legislative Yearbook 2021
    • Pediatric Cancer Research Consortia 2019
    • Kids v Cancer End of Year Letter 2017
    • Blog
  • Youth Programs
    • All programs
    • Climb the Hill Youth Advocacy Day Sign Up
    • Video and photo gallery
    • Kids v Cancer Youth Board
  • Our stories
  • Get involved
    • Donate
    • Our Parent Council
    • Happy Day Gift
    • Become a Corporate Partner

RACE for Children Act Could Substantially Increase Studies of Pediatric Cancer Drugs (Cancer Therapy Advisor)

March 30, 2020

March 25, 2020

Andrea S. Blevins Primeau, PhD, MBA

Link to the source: Cancer Therapy Advisor

The Research to Accelerate Cures and Equity (RACE) for Children Act has the potential to substantially increase the number of required pediatric studies for novel anticancer drugs, according to an analysis of previously approved agents and RACE criteria.

The RACE for Children Act was enacted in 2017 and will go into effect in 2020, with the goal of stimulating the development of novel targeted therapies for children with cancer. The RACE for Children Act will amend the Pediatric Research Equity Act (known to most as PREA) to require that sponsors must conduct pediatric studies for novel agents intended for the treatment of adult cancers, if the agent’s target is relevant to the growth or progression of pediatric cancers. In addition, it will extend the pediatric study requirement to drugs that receive the orphan drug designation.

Pediatric Trials for Cancer Therapies With Targets Potentially Relevant to Pediatric Cancers
Thomas J Hwang, Liat Orenstein, Steven G DuBois, Katherine A Janeway, Florence T Bourgeois
JNCI: Journal of the National Cancer Institute, Volume 112, Issue 3, 1 March 2020, Pages 224–228, https://doi.org/10.1093/jnci/djz207

“Few targeted therapies have been approved for children in the US under the current regulatory framework,” the authors wrote. However, “the RACE Act has the potential to substantially increase the study of cancer therapies in children.”

The study evaluated novel adult cancer agents approved by the US Food and Drug Administration (FDA) between 2007 and 2017, with a data lock of September 30, 2018. FDA approval letters and dossiers were evaluated for PREA requirements and waivers or exemptions. Drug labels were reviewed for any pediatric labeling. Ongoing pediatric trials were assessed using ClinicalTrials.gov. The authors reviewed the FDA’s Pediatric Molecular Target List published in August 2018 for potential molecular targets in pediatric cancers.

A total of 78 adult cancer drugs were identified. None of the clinical development programs of the 78 novel agents were required to conduct studies in pediatric cancers, 26 were exempt due to waivers obtained because the adult cancer does not occur in children, and 52 were exempt due to an orphan designation for a rare cancer.

At initial FDA approval, 5.1% of the novel drugs included any pediatric information in their labeling. After a median follow-up of 5.1 years, this increased to 21.8% ,and 10.3% received a pediatric indication.

Most of the novel drugs developed for adults also had known targets in pediatric cancers. Of the adult cancer drugs approved previously, 61 (78.2%) had targets on the Pediatric Molecular Target List. Therefore, the Act could increase the number of novel drugs required to be studied in pediatric cancer from 0% to 78.2%.

  • We are in the news
  • Our Publications
  • Our Newsletters
  • Our Op-Eds & Speeches
  • Legislation / Proclamations
  • Videos / Photos
  • RELEVANT NEWS
    • RACE for Children Act
    • Creating Hope Act / Pediatric priority review vouchers
    • Compassionate Use
    • Kids’ eligibility for trials
  • Facebook
  • Twitter

DONATE CONFLICT OF INTEREST SUBSCRIBE

Kids v Cancer · 3500 Macomb Street NW · Washington D.C. 20016 · 646-361-3590 · contact

© 2021 Kids v Cancer All Rights Reserved a 501(c)(3) nonprofit organization EIN 27-1427784 Privacy Policy Terms of Use Sitemap