Seminar 5 - Diary by William Chin
Freiburg, May 23, 2014 - Seminar 5 was held on the 8 – 10 May 2014 and addressed the entire spectrum of development and authorization of medicinal product for children. In the EU, the submission of an approved PIP is mandatory for marketing authorization of new substances or for new indications for products that are still protected by patent. Therefore, the objective of this seminar was to provide an understanding of the requirements and principles of authorization of medicinal products for use in children.
The line-up of speakers in this seminar was great; all speakers have extensive experience in their field of expertise. My personal highlight of the meeting was the presentation by Dr. Daniel Brasseur, the former Chairman of the Paediatric Committee (PDCO). Who could give a better lecture on this very topic other than the man who spearheaded the PDCO for 6 years since its inception in 2007! Dr. Brasseur is now a member of the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP). In his presentation, he spoke about the role and function of PDCO, its interaction with EMA and the industry, and how decision-making is performed in PDCO. Definition of the “condition” and “indication”, key binding element and extrapolation between subsets of population in pediatric studies was thoroughly explained. The concept of multiple PIPs for one product was also described, as this approach is use in order not to delay the authorization of medicines for use in adults or for sponsors who has the contingency need to claim the pediatric rewards. Dr. Avril Mankel (Santen, GmbH) explained the technicality of a PIP preparation, including a detail explanation of its format, submission timeline PIP, modification of PIP and its compliance check. She reminded that PIP now is an integral part of drug development program and it is important to check for internal consistency of the PIP because any development changes would affect the timeline of both submission of the adult MA and compliance check of PIP. Dr. Ashley Strougo (Astellas Pharma Global Development Europe) gave an exciting talk on simulation tools that can be used in pediatric studies and the use of different PK modeling approaches such allometric scaling, physiologically based pharmacokinetics and the mechanistic approach. She is also an advocate for transparency of clinical data because published information on paediatric studies may be helpful in terms of modeling and simulation (M&S) of a starting point for paediatric plan. Dr. Strougo concluded that regulators are starting to embrace the use of M&S. Genevieve Michaux (Counsel at Hunton & Williams) gave a presentation on the legality aspects of PIP, its obligation and the rewards that comes with it. Dr. David M. Cross (Cross Pharma Consulting) presented on the topic of juvenile animal studies design considerations followed by case histories. Although juvenile toxicity studies could support paediatric clinical trials and labeling, it should not be a routine part of development. Justification and its relevance needed to be understood in the context of different cross functions such as DMPK and clinical pharmacology. A recorded lecture of Dr. Jörg Breitkreutz (Heinrich-Heine-University Düsseldorf) entitled “Requirements on paediatric drug formulations” was presented in this seminar. Dr. Koen Crombex (MSD Europe) spoke about MSD’s experience with PIPs. Overall it is continuous learning experience with PDCO and the average time needed for a PIP is approximately 1 year. The take home message from this seminar is that the Pediatric Regulation exists to encourage the development of safe pediatric medicines in the EU market. To comply with this regulation, or any other regulations, involves careful planning that includes effort from multidisciplinary team.
William Chin is currently undertaking Master’s in Regulatory Affairs forBiopharmaceuticals at EUCRAF and previously obtained a Ph.D. in Biomedical Science from the Department of Pharmacy, National University of Singapore.