​Pediatric Oncology Trials: Changes on the Horizon
White Paper

PENDING U.S. LEGISLATION

Developers of oncology therapeutics are closely following the Research to Accelerate Cures and Equity for Children Act (also known as the RACE for Children Act), introduced as two identical bills in the House and Senate, H.R.58581 and S.3239,2 respectively, on July 14, 2016.

This legislation would apply to new drug applications (NDAs) and biologic license applications (BLAs) submitted 18 months after enactment, and would impact many therapies in development today. The bills have potential to dramatically alter the pediatric oncology landscape by expanding Pediatric Research Equity Act (PREA) requirements for oncology therapeutics. For drugs being developed for an indication that affects children, PREA requires pediatric trials for new active ingredients, indications, dosage forms, dosing regimens, and routes of administration. For oncology drugs, RACE would remove the requirement that the indication studied in adults affects children; instead, the obligation to conduct pediatric trials would be triggered if the drug or biologic is “directed at a molecular target present in one or more cancers in one or more pediatric populations.” Also, orphan designation would no longer exempt such products from PREA requirements.

One of the provisions of RACE is that in addition to the review comments that are provided within 90 days of submission of the initial Pediatric Study Plan (iPSP), FDA would meet with the sponsor at the end of Phase 1 to discuss preparation of the iPSP for drugs with a molecular target relevant to a pediatric cancer. Within one year of enactment, guidance would be issued on study designs and likely molecular targets. Under RACE, Congress would receive a report on the legislation’s implementation by July 12, 2021.

This insight brief, authored by IQVIA Pediatric and Oncology Center of Excellence experts, provides a summary of the proposed legislation and potential impacts on oncology development programs if adopted. The insight brief also examines specific considerations and planning for pediatric oncology trials, and the changing landscape in pediatric oncology drug development.


PEDIATRIC ONCOLOGY CLINICAL TRIALS: AN OVERVIEW

Some 16,000 children are diagnosed with cancer in the U.S. each year,4 with about 300,000 diagnoses worldwide each year.5 The various forms of leukemia make up almost one-third of all childhood cancers. Classified as rare diseases, childhood cancers share the challenges associated with other rare diseases in terms of identifying, recruiting and retaining patients in clinical trials.


PROGRESS IN PEDIATRIC ONCOLOGY

In recent decades, outcomes have improved substantially in all forms of pediatric cancer. There are many reasons for this including the fact that there is a strong spirit of national and international cooperation among academic investigators, with collaborative groups driving the research agenda. Additionally, there is a mindset of routinely caring for pediatric patients in a clinical trial setting. Most of the progress to date has been gained through incremental adjustments to dosing, combination and scheduling of standard cytotoxic agents.

Relative pediatric cancer survival rates are shown in. Many of these survival rates have now started to plateau. This coincides with an era of discovery that is delivering an improved understanding of the molecular basis of many childhood cancers. The challenge is to understand how to leverage this explosion in scientific knowledge to further improve outcomes.


CHALLENGES TO FURTHER IMPROVING OUTCOMES

The first challenge is the need for targeted agents for pediatric cancers. These will come from repurposed targeted agents developed for adult tumors or from the development of novel agents against novel targets defined in pediatric cancers. Advances in genomic technologies are expanding understanding of how pediatric tumors can be targeted. Potential legislative changes under RACE may also expand the range of adult agents studied in pediatric populations.


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