November’s Regulatory Focus: Global development for pediatric products

Feature ArticlesFeature Articles | 06 December 2021 | Citation

Feature articles during November included articles on strategies for pediatric clinical trials; the US Food and Drug Administration’s (FDA’s) rare pediatric disease priority review voucher program; challenges, solutions, and opportunities in global pediatric development; and recent FDA approvals of drugs and biologics for children.
Clinical trials and the PRV program
Clinical trials for demonstrating pediatric effectiveness are difficult to design, get approved, and conduct. In Strategies for pediatric clinical trials and drug development, Susanne Schmidt presents a
pediatrician’s perspective on addressing some of the challenges in designing and conducting pediatric effectiveness trials. Possible solutions include closer consideration of pediatric subpopulations and issues related to age, pharmacokinetic and pharmacodynamic correlations, and study endpoints. Schmidt also emphasizes the importance of a patient- and family-centric approach to studies within an ethical framework.
The FDA’s priority review voucher (PRV) program was expanded in 2012 to include rare pediatric diseases. The program has stimulated development of therapeutics for unmet medical needs in the pediatric population, according to research; however, it also has been criticized, particularly regarding pharmacoeconomic impact and placing unanticipated burdens on the FDA. In Effectiveness of the rare pediatric disease priority review voucher program, Rahul Chandrasekhar traces the history of the program, outlines the criteria for receiving a PRV, and explains its key features. The author examines the program’s efficacy and addresses the previously mentioned shortcomings around pharmacoeconomic impact and burden on the FDA. The rare pediatric disease PRV program was originally set to expire on 30 September 2020 but has been extended to 30 September 2024.
Challenges, opportunities, and new approvals
In Global pediatric development: Challenges, potential solutions, and opportunities, Melodi McNeil focuses on two key challenges in pediatric drug development – small patient numbers and divergent regulatory requirements from the FDA and European Medicines Agency (EMA). Small patient numbers can potentially be addressed by using additional sources of evidence (e.g., real-world data and evidence), a model-informed drug development approach, innovative trial design, and/or collaborating with clinical trial networks and consortia. Regarding the differences between FDA and EMA requirements, McNeil emphasizes the importance of early and frequent communication with the agencies, along with periodic “cluster calls” with agency representatives, using the FDA-EMA parallel scientific advice program for concurrent exchange of information, and having an EMA observer present during an FDA Type F meeting.
In The RACE is on: Opportunities in pediatric oncology product development, Lisa Crose summarizes the regulatory changes associated with the Research Acceleration for Cure and Equity (RACE) Act, part of the FDA Reauthorization Act of 2017, and describes how to incorporate the changes into pediatric product development programs. As with most things regulatory, early strategizing is crucial, as are regular Type F meetings with the FDA’s Oncology Center of Excellence Pediatric Oncology.
Priyal J. Dave’s Recent FDA approvals of drugs and biologics for children is a roundup of prescription products that have recently become available in the US. The article also includes examples of therapeutic products previously approved for adults and which are now available for expanded use in children.
Upcoming in Regulatory Focus
What’s coming in December?
Articles during December will examine APL and the Role of Social Media. Look for this topic and more throughout December at Regulatory Focus.
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For the December issue, Regulatory Focus will cover Leadership in regulatory affairs. The submission due date for articles has been extended to 7 January 2022. To contribute, email
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February 2022 issue
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Also see 2022 Editorial Calendar.
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