Smita Salunke

European Paediatric Formulation Initiative (EuPFI)-Formulating Ideas for Better Medicines for Children.

The European Paediatric Formulation Initiative (EuPFI), founded in 2007, aims to promote and facilitate the preparation of better and safe medicines for children through linking research and information dissemination. It brings together the capabilities of the industry, academics, hospitals, and regulators within a common platform in order to scope the solid understanding of the major issues, which will underpin the progress towards the future of paediatric medicines we want.The EuPFI was formed in parallel to the adoption of regulations within the EU and USA and has served as a community that drives research and dissemination through publications and the organisation of annual conferences. The membership and reach of this group have grown since its inception in 2007 and continue to develop and evolve to meet the continuing needs and ambitions of research into and development of age appropriate medicines. Five diverse workstreams (age-appropriate medicines, Biopharmaceutics, Administration Devices, Excipients and Taste Assessment & Taste Masking (TATM)) direct specific workpackages on behalf of the EuPFI. Furthermore, EuPFI interacts with multiple diverse professional groups across the globe to ensure efficient working in the area of paediatric medicines. Strong commitment and active involvement of all EuPFI stakeholders have proved to be vital to effectively address knowledge gaps related to paediatric medicines, discuss potential areas for further research and identify issues that need more attention and analysis in the future.

Formulating better medicines for children-reflections

Publisher Rights Statement: NOTICE: this is the author’s version of a work that was accepted for publication in International Journal of Pharmaceutics. Changes resulting from the publishing process, such as peer review, editing, corrections, structural formatting, and other quality control mechanisms may not be reflected in this document. Changes may have been made to this work since it was submitted for publication. A definitive version was subsequently published in International Journal of Pharmaceutics, DOI: 10.1016/j.ijpharm.2015.05.017.

Formulating better medicines for children—Still too far to walk

The objective of European Paediatric Formulation Initiative (EuPFI) is to promote collaboration between industry, academia, clinical and regulatory professionals to facilitate preparation of better and safe dosage forms for children. It provides the platform to discuss contemporaneous issues shared by scientific community in developing paediatric medicines. In order to achieve these objectives, EuPFI conferences have been successfully held for six years. Continuing on its mission, in 2015, EuPFI organised the 7 conference on “Formulating better medicines for children” in association with APV (Arbeitsgemeinschaft für Pharmaceutische Verfahrenstechnik). It brought together 140 delegates and keynote speakers from 23 countries on 16-17 September 2016 in Antwerp, Belgium to present the current needs and challenges in paediatric drug development. It highlighted in particular the implications from multiple stakeholder perspectives, including regulators, industry representatives academics and clinicians.

‘Formulating better medicines for children’ – The leap forward

Taking the leap forward towards the awareness and understandings of the paediatric-specific issues in the development of paediatric formulations, the European Paediatric Formulation Initiative (EuPFI) convoked its 5th annual conference in association with the International Association of Pharmaceutical Technology (APV). The conference was held on 18th and 19th September 2013 at the Hotel Porta Fira in Barcelona, Spain. The participation of 162 delegates from industry, academia, regulatory and other organizations from 27 countries with diverse backgrounds and involvement in both formulation development and regulatory submissions, provided a productive setting for the exchange of information and views. Following feedback received from the previous conference, a new format, including pre-conference workshops and innovative showcase, was introduced. The programme for this 2-day conference was participant driven and touched upon a range of topics for product development with the patient’s point of view. In addition, the conference provided the opportunity for practitioners from different disciplines to discuss aspects and issues of formulating paediatric medicines including understanding the research needs for children’s medicines, developing new medicines for children or adapting existing medicines so that they could be used safely and effectively in children and improving ways to encourage children to take their medicines. Two pre-conference workshops led by specialists in each topic area, gave an opportunity for the participants to maximize their learningexperience. The first workshop chaired by Prof. Adolf Valls-iSoler (University of the Basque Country) provided an in-depth review and scientific discussion on how developmental changes may influence the pharmacokinetics and pharmacodynamics of drugs, how pharmacokinetics could be predicted in neonates, infants and children and how it can be linked to pharmacodynamics. The second workshop by Dr. Dirk Mentzer (PDCO, EMA) was an opportunity to learn how the regulatory professionals interpret the paediatric regulation and implement it inpractice. Inparticular, how setting up a paediatric investigation plan links up with the various phases of drug development (designation, protocol assistance, and marketing authorisation). Long standing problems with potential health risks in children have triggered an international response in the form of the European Union (EU)-funded seventh framework programme FP7/2007–13 under grant agreement n 261060 GRiP (global research in paediatrics) project. The keynote plenary lecture by Dr. Kalle Hoppu

CHAPTER 10:Paediatric Pharmaceutics—The Science of Formulating Medicines for Children

Paediatrics (also spelled pediatrics) deals with the medical care of neonates, infants, children, and adolescents, from birth up to 18 years of age. Child health is key to overall human life expectancy, as paediatric diseases may have a lifelong effect on quality of life. On a global scale nearly 5.8 million children under the age of five died in 2015, representing a 52% decline in the number of under-five deaths since 1990. Neonatal deaths fell at a slower pace since 1990, decreasing 42% to 2.6 million; stillbirths declined 47% to 2.1 million. Although life expectancy has improved around the world between 1990 and 2015, it still remains the case that people in more developed countries can look forward to longer and healthier lives than people in less developed countries. One of the factors driving the increases in life expectancy at birth is better health outcomes for young children, implying urgent need for medicines that keep children healthy. Children have the right to access medicines that are appropriate to their unique needs and to have adequate assurance of their quality, safety and efficacy. This has been widely acknowledged on a worldwide platform and prompted global initiatives and legislative changes that have transformed this once niche area into an integral part of the drug development process. This chapter discusses the specific needs of children, how the implementation of paediatric regulations has influenced/promoted the research into this previously neglected population and key attributes to consider during the designing and development of paediatric dosage forms to provide adequate paediatric therapies.

Formulating better medicines for children – collaborate to innovate

Continuing on its mission and conference series to raise awareness and understand paediatric-specific issues in the development of age appropriate dosage forms, the European Paediatric Formulation Initiative (EuPFI) convoked its 8th annual conference with the International Association of Pharmaceutical Technology (APV) on 21 and 22 September 2016 at the Hotel Novotel in Lisbon, Portugal. The participation of 158 delegates from Industry, Academia, Regulatory and other organisations from 26 countries with diverse backgrounds and involvement in both formulation development and regulatory submissions provided a productive setting for the exchange of information and views.