NCHR Testifies at the Pediatric Oncology Subcommittee Meeting

May 22, 2024

We thank you for the opportunity to offer public commentary on Section 504 of the 2017 FDA Reauthorization Act (FDARA) to Section 505B of the Food, Drug, and Cosmetic Act, which allowed original applications (submitted on or after August 18, 2020) involving pediatric investigations of certain targeted cancer drugs with new active ingredients to be assessed based on their molecular target rather than clinical indication.

The National Center for Health Research (NCHR) is a nonprofit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with a particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.

FDARA and the Research to Accelerate Cures and Equity (RACE) for Children act were developed to facilitate the pediatric assessment of molecular targeted cancer drugs and ultimately, expedite the development of new, safe and effective therapies for pediatric oncology patients.

However, based on the FDA’s updated analysis of initial pediatric study plans for molecular targeted drugs and the Government Accountability Office’s audit findings, data show that while the RACE for Children act has helped increase the number of planned studies for pediatric cancer patients, it has not demonstrated an increase in the number of drugs approved to treat pediatric cancers. Further, there is little data to suggest that the new drugs in the pipeline will be safe or any more effective than what is currently available, as a majority of these applications received full waivers for pediatric study requirements or have deferred their clinical investigation.

To fully maximize the potential of the FDARA and RACE for Children legislation and ensure timely investigations of molecularly targeted drugs that significantly improve the standard of care for pediatric oncology patients, changes are needed.

To aid in the development of pediatric oncology treatments that are both safe and effective, sponsors should prioritize engaging with key stakeholders. This includes initiating discussions with regulatory agencies such as the EMA Paediatric Committee, FDA Pediatric Review Committee, FDA Oncology Subcommittee of the Pediatric Review Committee, and Oncology Center of Excellence. It is advisable for sponsors to initiate these discussions early on in the drug development process. Conducting joint meetings with both the FDA and EMA to facilitate international cooperation will help ensure that regulatory guidance is being followed and help to create effective pediatric development plans. Early engagement with key scientists and academic leaders is also necessary to assist in robust drug development and the design of pediatric investigations that yield results that are both scientifically and clinically meaningful. Furthermore, it is crucial to involve physician scientists with expertise in the incorporation of pediatric-specific patient centered outcomes into study designs, as collecting such data will be imperative to fully understand the risks and benefits of treatments among pediatric oncology patients.

Under FDARA and RACE for Children many new molecular targeted therapies for pediatric patients with cancer have and will continue to receive full or partial waivers for pediatric study requirements. While the end goal of these policies is to ultimately accelerate the pathway for finding novel cures and improving the overall survival of pediatric oncology patients, this legislation must be met with caution. The approval of new molecular drug therapies that are non-superior to other treatments, or that are too toxic to achieve treatment adherence will not achieve this end goal. Thus, we strongly encourage the use of independent Pediatric Expert Groups (PEGs) and Patient and Caregiver Engagement Panels throughout the drug development and investigative process. Involving independent PEGs early on and throughout the process can help shift the ownership and responsibility of the scientific process to those who have scientific expertise and interests remain to the scientific process itself, rather than being driven by monetary incentives. The use of Patient and Caregiver Engagement Panels help guide study outcomes and improve how study results directly relate to patients and their loved ones. These panels also help build rapport and enhance study design and participation, with higher rates of patient enrollment and retention.

We appreciate the opportunity to provide public commentary and hope these recommendations prove useful in advancing molecularly targeted drugs that significantly improve the standard of care of pediatric oncology patients.

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