Developing medications specifically for paediatric patients involves navigating complex regulatory pathways. Due to developmental differences compared to adults, children require dedicated clinical trials and dosing guidelines tailored to their needs. Successfully bringing paediatric treatments to market requires a thorough understanding of the drug development and approval process.

Paediatric drug development is a complex and challenging process, central for ensuring the safety and effectiveness of new drugs for children. Failure to meet the FDA's paediatric study regulations can lead to significant delays or even the rejection of your marketing application. The Paediatric Study Plan (PSP) is an important document that outlines the sponsor's comprehensive plans for paediatric drug development. A well-written PSP can help you navigate the complexities of paediatric drug development and increase the likelihood of a successful outcome.

Paediatric drug development is an important component of healthcare, as children have unique physiological and pharmacological needs compared to adults. However, conducting clinical trials in paediatric populations can be challenging due to ethical, practical, and scientific considerations. To address these concerns, the FDA has implemented various regulations and incentives to promote paediatric drug development.

The Study Plan

An initial paediatric study plan (iPSP) is a document that outlines the paediatric studies a sponsor plans to conduct. It helps the FDA and the sponsor agree on appropriate studies, timelines, and the regulatory pathway for receiving a potential paediatric indication. Biotech companies developing complex treatments in oncology, rare diseases, or advanced technologies should incorporate paediatric data to ensure their treatments are safe and effective for all age groups, fulfilling regulatory requirements and enhancing market potential.

The iPSP must be submitted to the FDA within 60 days after the End-of-Phase 2 meeting or, if such a meeting does not take place, 210 days before the sponsor submits a marketing application. This allows for a 120-day review period by the FDA, ensuring that any issues or concerns are addressed before the marketing application is submitted. A paediatric study plan outlines the specific paediatric studies that a sponsor intends to conduct. It includes elements such as study objectives, design, age groups, relevant endpoints, and the statistical approach.

Supporting documentation and any other information required by the FDA should also be included. By outlining these details, biotech companies can ensure clarity and compliance throughout the paediatric drug development process.

Drug Specific Waivers and Deferrals

The FDA provides sponsors with the option to request waivers or deferrals of paediatric assessments or reports for molecularly targeted paediatric cancer investigations. Waivers may be granted under specific circumstances, such as when conducting the necessary studies is impossible or highly impracticable, or when the drug does not provide a meaningful therapeutic benefit to the paediatric patients.

Partial waivers may be considered for subsets of the pediatric population or when reasonable attempts have been made to develop age-appropriate formulations. Understanding these waiver and deferral options enables the biotech companies to navigate the regulatory landscape more effectively.

PSP Timelines

Once the iPSP is submitted, the FDA has 90 days to respond in writing or meet with the sponsor. The review process involves consultations with the FDA's internal Paediatric Review Committee (PeRC). During the subsequent 90 days, sponsors have the opportunity to review FDA comments and negotiate the details of the iPSP. An agreed iPSP must be submitted within this period.

The FDA then has 30 days to review and issue correspondence confirming or expressing disagreement. It's worth noting that engaging in open communication and addressing any concerns raised by the FDA during the iPSP process can help the company to provide safe and effective treatments for all age groups.


The inclusion of paediatric populations in drug development remains an ongoing effort that requires diligence from all parties. By carefully crafting and submitting comprehensive paediatric study plans, biotech companies can comply with regulations, ensure compliance, and enhance the safety and efficacy profile of their drugs. To achieve success in paediatric drug development, it is important to learn the fundamentals of paediatric study plans, waivers, deferrals, and the submission process. By adhering to these ground rules and addressing potential challenges, you can submit a complete and regulation-compliant plan.

Ensure compliance and guide your development process with experts from NDA who provide comprehensive regulatory guidance tailored to your needs. Contact us by using the form below to discuss personalized support for navigating complex requirements and exceeding FDA expectations.

Stay up to date

Contact us

Your drug development needs are unique

Contact us to learn how our experts can support you to meet all regulatory, quality and safety requirements and ensure the best path to approval.

Let's Bring Medicines to the World

  • This field is for validation purposes and should be left unchanged.