FDA Guidance: The New and Revised Pediatric Study Plans

According to the US Food and Drug Administration (FDA), a majority of prescribed pediatric drugs are not tested. To overcome serious shortage of pediatric drug testing, FDA has issued a final guidance that offers a comprehensive framework for planning and submission of applications for pediatric trials.

These new guidelines by the FDA are going to play a vital role in addressing the issues that occur in the pediatric drug development process, such as the absence of critical information on pediatric use of the medications under certain reported conditions, and the need to amplify the number of pediatric trials conducted for patented products.

The Initial Pediatric Study Plan(iPSP) submission is mandatory for the sponsors who intend to submit a marketing application for the following cases including an API (Active Pharmaceutical Ingredient)/ any new indication except for the one that has orphan status/ a new dosage form/ a new administration mode. According to The Pediatric Research Equity Act (PREA) of 2003, those who wish to file applications for pediatric drugs/biologics are subjected to submit iPSP in the preliminary stage of product development. In addition, with effect from August 18, 2020, an iPSP must be submitted for APIs which come under the PREA’s provision of molecular-based medicine for cancer, regardless of their status of orphan indication.

Sponsors are expected to submit iPSPs within 60 calendar days post the Phase-2 meeting, or as per the agreement between the FDA and the sponsor unless there are exceptional cases. Upon the FDA’s review, the sponsor gets a written response or a meeting request from the Agency, within 90 days of its review period. After responding to FDA's comments within the timeline of 90 days, the sponsor is expected to submit an agreed iPSP before the expiry of its revision deadline.

Similarly, the Agency will review the agreed iPSP within 30 days’ time frame and sends the letter of no agreement if the sponsor fails to fulfill the given criteria. As a result, sponsors will get another 30 days from the date of notification to review the iPSP. Once the amended submission is done, the 210-day cycle of the FDA-sponsor review will be resumed.

The final guide explains the recommended mechanisms with regards to managing non agreed iPSPs that include few recommendations on how to achieve FDA’s agreement and a sample template for iPSP preparation as well.

While the guidance has outlined insightful strategies for the usage of data related to extrapolation and its relevance within the given criteria, it is the responsibility of the applicant to implement them in an accurate way for easy review and approvals. Practice the best Regulatory practices for compliance. Stay informed. Stay compliant.