Path towards efficient paediatric formulation development based on partnering with clinical pharmacologists and clinicians, a c4c expert group White paper.

Walsh, Jennifer; Schaufelberger, Daniel; Lurian, Sonia; Klein, Sandra; Batchelor, Hannah; Turner, Roy; Gizurarson, Sveinbjörn; Boltri, L.; Alessandrini, Elisa; Tuleu, Catherine

doi:10.22541/au.161911326.60007239/v1

Cited by 8 publications

(2 citation statements)

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“…Therefore, a strategic workforce has been constructed to appropriately reply to disease burden across childhood, addressing the therapeutic deficit and developing age-appropriate formulations, in order to maximize efficacy and design quality, promote safety, minimize risks, and increase patient adherence to treatments [9,10].…”

Section: Introductionmentioning

confidence: 99%

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies

Domingues,

Jarak,

Veiga

et al. 2023

Pharmaceutics

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The paradigm of pediatric drug development has been evolving in a “carrot-and-stick”-based tactic to address population-specific issues. However, the off-label prescription of adult medicines to pediatric patients remains a feature of clinical practice, which may compromise the age-appropriate evaluation of treatments. Therefore, the United States and the European Pediatric Formulation Initiative have recommended applying nanotechnology-based delivery systems to tackle some of these challenges, particularly applying inorganic, polymeric, and lipid-based nanoparticles. Connected with these, advanced therapy medicinal products (ATMPs) have also been highlighted, with optimistic perspectives for the pediatric population. Despite the results achieved using these innovative therapies, a workforce that congregates pediatric patients and/or caregivers, healthcare stakeholders, drug developers, and physicians continues to be of utmost relevance to promote standardized guidelines for pediatric drug development, enabling a fast lab-to-clinical translation. Therefore, taking into consideration the significance of this topic, this work aims to compile the current landscape of pediatric drug development by (1) outlining the historic regulatory panorama, (2) summarizing the challenges in the development of pediatric drug formulation, and (3) delineating the advantages/disadvantages of using innovative approaches, such as nanomedicines and ATMPs in pediatrics. Moreover, some attention will be given to the role of pharmaceutical technologists and developers in conceiving pediatric medicines.

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Section: Introductionmentioning

confidence: 99%

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies

Domingues,

Jarak,

Veiga

et al. 2023

Pharmaceutics

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“…An extensive overview is presented on the current landscape of paediatric formulation development, and addresses major limitations and challenges, including regulatory. 8 The group presents a paediatric Quality Target Product Profile as an efficient tool to facilitate early planning and decision making across all teams involved in paediatric formulation development.…”

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confidence: 99%

Innovative methodologies in paediatric drug development: A conect4children (c4c) special issue

Wildt

Wong

2022

Brit J Clinical Pharma

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Innovative methodologies in paediatric drug development: A conect4children (c4c) special issue On average, half of all children in Europe take medicines, either for acute or chronic illness. At the same time almost half of these prescriptions are off-label, due to a historical lack of medicines specifically developed for children. 1 For ethical, practical and economic reasons, academic and industry researchers were reluctant to study drugs in children. With the 2007 EU Paediatric Regulation, and similar initiatives across the world, this situation has changed, as companies are now mandated to submit paediatric investigation plans early in the course of drug development. While these legislative efforts have resulted in a huge increase in the initiation of paediatric trials, new paediatric registrations have severely lagged behind. 2 Several causes can be identified, but a major reason for this disappointing result has been the failure to successfully perform paediatric clinical trials, not only in Europe, but across the world. Many studies were started but did not manage to recruit sufficient numbers of patients, and other studies failed to provide adequate answers on efficacy or safety due to suboptimal study design. To overcome these challenges, the European Union with 10 pharmaceutical industry partners allocated, in 2018, €140 million for the conect4children (c4c) project through the public-private Innovative Medicines Initiative 2 (IMI2) funding programme. 3 The aim of this project is to provide better medicines for babies, children and young people through a pan-European clinical trial network. c4c aims to generate a sustainable infrastructure that optimizes the delivery of clinical trials in children through: (a) a single point of contact for all sponsors, sites and investigators; (b) efficient implementation of trials, adopting consistent approaches, aligned quality standards and coordination of sites at national and international level-to set up the network, three academic and at least four industry paediatric proof of viability trials will be run across the European network of over 20 countries and up to 300 sites; (c) collaboration with specialist and national networks; and (d) an education and training platform to shape the future leaders of paediatric drug development. The c4c Academy has been set up and is offering courses for good clinical practice, family and patient involvement, monitoring, trial start-up, paediatric investigation plan, innovative trial design, gene therapy trials, train the trainer, trial specific training, and paediatric drug development. Moreover, c4c

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How to START? Four pillars to optimally begin your orphan drug development

Jonker

Batista

Gabaldo

et al. 2023

Orphanet J Rare Dis

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Drug development is a complex, resource intensive and long process in any disease area, and developing medicines to treat rare diseases presents even more challenges due to the small patient populations, often limited disease knowledge, heterogeneous clinical manifestations, and disease progression. However, common to all drug development programs is the need to gather as much information as possible on both the disease and the patients’ needs ahead of the development path definition. Here, we propose a checklist named START, a tool that provides an overview of the key pillars to be considered when starting an orphan drug development: STakeholder mapping, Available information on the disease, Resources, and Target patient value profile. This tool helps to build solid foundations of a successful patient-centered medicines development program and guides different types of developers through a set of questions to ask for guidance through the starting phase of a rare disease therapeutic pathway.

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Path towards efficient paediatric formulation development based on partnering with clinical pharmacologists and clinicians, a c4c expert group White paper.

Cited by 8 publications

References 0 publications

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies

Innovative methodologies in paediatric drug development: A conect4children (c4c) special issue

How to START? Four pillars to optimally begin your orphan drug development

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