Historically, many drugs have been prescribed to children even though this patient population have largely been excluded from clinical trials. Authorities worldwide have, therefore, implemented regulations to address the gap in drug research involving children and to promote efforts that can lead to increased knowledge of pediatric pharmaceutical use.

There is an obvious logic. If medicines are to be used in children, they need to be studied in pediatric populations to ensure they are safe and effective. Here, we share important considerations for your pediatric development plan, including the US pediatric study plan (PSP) and the EU pediatric investigation plan (PIP).

When do sponsors need to conduct pediatric studies and when are they exempt?

Whether you need to include children in your clinical studies will partly depend on which disease you are targeting and what type of medicine you are studying. If you have a drug that targets a condition that does not affect children, such as Alzheimer’s disease, you will be granted a waiver. A waiver may also be given for specific age groups based on safety or lack of efficacy, the condition not occurring in the specific age group or other specific age-related reasons. Sometimes, a deferral from the requirement to study the drug in the pediatric population may be granted which means that the studies can be postponed until after you have shown that the drug is safe and effective in adults. However, outlining a PIP/PSP for your drug is mandatory, regardless of whether you expect to receive a waiver or deferral for the pediatric studies.

The challenge of harmonizing across national borders

Harmonizing pediatric study plans for different parts of the world is a complex task due to authorities in different regions having varying recommendations about when to initiate the development of pediatric study plans and what they should include. For example, in the EU, it’s preferred to submit a PIP early in the development process, when pharmacokinetic data are available, whereas in the US, the FDA requests a PSP after the completion of Phase II trials. These differences in timing make it challenging to coordinate pediatric studies globally.  To manage this effectively, the best practice is to set a strategy for the global pediatric plan early in the development process. Without this proactive approach, the pediatric plans could delay the entire development project.

The contents of a PSP or PIP

The purpose of a PIP/PSP is to gather comprehensive information about the use of a drug in pediatric populations. Below are examples of what it should contain:

• An overview of the disease, diagnosis, and treatment, highlighting differences between children and adults.
• An assessment of the need for the drug in children across all age groups from birth to adolescence.
• A summary of available chemical, preclinical, and clinical data on the drug.
• A proposed strategy for any required preclinical studies and measures to adapt the drug’s formulation for use in children.
• A proposed plan for potential clinical studies in children, including the timing of these studies in relation to those conducted in adults.

Financial benefits of conducting pediatric studies

Conducting pediatric studies not only ensures the safety and efficacy of a medicine in children but may also introduce new market opportunities in the pediatric population. In addition, following your pediatric plan can yield significant financial benefits in the form of a six-month patent extension (additional protection). It may seem short, but a six-month extension provides valuable exclusivity on the market and helps developers maximize the commercial lifespan of their product.

Regulatory incentives for pediatric oncology drugs: The RACE for Children Act

The Research to Accelerate Cures and Equity (RACE) for Children Act, passed by the U.S. Congress in 2017 and implemented in August 2020, significantly reformed the landscape of pediatric oncology drug development. The Act mandates that new cancer drugs developed for adults must also be evaluated for pediatric use if the molecular target of the drug is relevant to pediatric cancers. This requirement includes drugs with orphan drug designation, previously exempt from such studies. Prior to the RACE Act, pharmaceutical companies were not obligated to conduct pediatric studies for oncology drugs developed for adult cancers, leading to a significant gap in treatment options for children.

Early findings are promising, showing a clear rise in the number of oncology drugs being studied for pediatric use. Between August 2020 and August 2022, 32 initial pediatric study plans were submitted to the FDA due to the RACE Act, indicating a promising shift towards more inclusive drug development practices. [1]

Key Takeaways

Integrating pediatric patients into clinical trials can help ensure the safe and effective use of medicines for children. This is emphasized by global regulatory requirements and incentivized initiatives. However, navigating diverse sets of regulatory guidelines across countries and regions presents challenges in harmonizing and coordinating pediatric development plans on a global scale. With careful planning and considerations of the key factors outlined here, sponsors can minimize delays and expedite the approval process, ensuring timely access to safe and effective drugs for both adults and children.

Have questions? Get in touch with our experts: Erika Spens, Director Regulatory, Affairs; Sofie Broberg, Senior Consultant, Regulatory Affairs; Anna Törner, VP, Strategic Regulatory Affairs; and Linda Nord, Senior Consultant Regulatory Affairs: Contact Our Strategic Consulting Team

Notes

[1] Children’s Cancer Cause. (2023, February 8). First Two Years of the RACE Act Evaluated in New GAO Report. https://www.childrenscancercause.org/blog/race-act-gao-report

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