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Expert Insights

New Requirements for Drugs to Treat Cancer for Pediatrics: What The RACE for Children Act Means for Sponsors

New Requirements for Drugs to Treat Cancer for Pediatrics: What The RACE for Children Act Means for Sponsors
08 January 2020

PREA (Pediatric Research Equity Act), initially passed by Congress in 2003 and subsequently made permanent in 2012, is a mandatory program that was enacted to promote development of essential medicines for children. However, products with orphan drug designation (including pediatric studies for cancer drugs directed at molecular targets relevant to children’s cancers), were exempt from the requirements of PREA.

Although the majority of cancers are rare in children, pediatric cancer is the leading cause of death by disease among children past infancy in the United States[1]. While adults with cancer have access to drugs that can potentially save or extend their lives—with almost 900 drugs in the adult cancer pipeline—only a handful are in development for children[2]. Additionally, drugs available to treat pediatric cancer are often several decades old.

Many hope that revisions to PREA will help to change that. On August 18, 2017, the president signed the 2017 FDA Reauthorization Act, which includes Title V, the Research to Accelerate Cures and Equity (RACE) for Children Act. The RACE for Children Act eliminates orphan exemptions from pediatric studies for cancer drugs directed at molecular targets relevant to children’s cancers. 

Complying with The Race for Children Act and PREA – Key Considerations
In August 2020, the RACE for Children Act will go into effect. With the RACE for Children Act eliminating orphan exemptions from pediatric studies for cancer drugs directed at molecular targets relevant to children’s cancers, more drug developers will need to consider the implications of PREA. Since PREA mandates that sponsors who plan to market their drug in the US have an agreed pediatric study plan (PSP) in place at the time of filing, it is important to start now, anticipating at least three to four months for plan development and another seven months to reach agreement with the FDA. 

  • Unless otherwise exempt, sponsors planning to submit a marketing application for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration are subject to PREA.
  • A biosimilar is considered a “new active ingredient” and is subject to PREA.
  • All pediatric populations (from birth up to <17 years of age) must be addressed in the plan; even products not developed for use in adults are subject to PREA.
  • PREA requires that a pediatric study plan must be submitted to the FDA within 60 days after the End-of-Phase (EOP) II meeting. In cases where an EOP2 meeting is not held, the PSP should be submitted before Phase III or combined Phase II/III studies are initiated. In any event, the plan should be submitted at least 210 days before submission of the marketing application to be sure an agreed plan is in place at the time of filing. Not having a plan in place for a product subject to PREA is grounds for a refusal to file.
  • Waivers from conducting pediatric assessments in some (partial) or all (full) can be granted under certain conditions:
    • If studies are impossible or highly impracticable
    • Strong evidence exists to suggest that the treatment would be ineffective or unsafe
    • The drug does not represent a meaningful therapeutic benefit over existing therapies for pediatric patients and is not likely to be used in a substantial number of pediatric patients, or
    • It is not possible to develop an age-appropriate pediatric formulation.

RACE Minimizes PREA Exemptions for Cancer Drugs
With the RACE for Children Act, sponsors developing new molecular therapies to treat adult cancers are now required to also evaluate them in pediatrics if the molecular target is substantially relevant to the growth or progression of pediatric cancer.

As a requirement of PREA, the FDA published, in August 2018, a list of relevant 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.” Currently over 200 possible targets have been identified on this list. This list is being updated continually as new information becomes available.

It has also developed a list of “targets for which there is evidence that they are not associated with the growth or progression of pediatric tumors” for which drugs and biologics directed at these targets may receive waivers. About 5 targets are listed on this waiver list.

How can drug developers prepare for important changes with the RACE for Children Act?

Our VP Pediatric Development (Americas), Lynne Georgopoulos, RN, MSHS, RAC developed a checklist to help drug developers gain insights on important steps to take. Download your copy today

[1] National Cancer Institute
[2] How the RACE for Children Act Will Get Drugs to Kids with Cancer, Cancerletter.com, September 8, 2017.

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