Over the last years, a Europe-wide trend toward a patient-focused approach is developing and is influencing the decision-making process related to the clinical research. This new vision aims to draw on patient knowledge and experience in order to deliver benefits for all stakeholders of the drug development process, optimizing the clinical study design. In this context, the “patient empowerment” concept has been developed as an approach encouraging the active participation and self-determination of the patients in the caring procedure. For this reason, in 2016, European Patients’ Academy (EUPATI) launched a public consultation that ended in September 2016 with the release of the EUPATI guidance for patient involvement in the medicine research and development process. Likewise, the recommendations on the “Summaries of Clinical Trial Results for Laypersons” for the Implementation of Regulation (EU) No 536/2014 recommended a clear and comprehensible communication of the clinical trial results to the patients. However, rarely, all these attempts for the patient involvement pay attention to the pediatric population needs. An innovative approach for the patients’ involvement in pediatric clinical research is represented by the Young Persons Advisory Groups, an organization composed of youths, patients, and carers, actively participating in clinical research and advising researchers and their teams.
Part of the book: The Management of Clinical Trials
Drug discovery and development advances in the last decades allowed to find a treatment for many preventable diseases. However, all too often, children are excluded from these progresses since most of the new medicines have been discovered and developed for the adult population. Even if paediatricians routinely give drugs to children ‘off-label’, researchers have demonstrated that children do not respond to medications in the same way as adults. Furthermore, certain specific disorders are unique to children or occur in children differently than in adults. Besides specifically testing medicines in children in proper clinical studies taking into due account the peculiarity of this population, there is a growing recognition of the need to develop paediatric medicines having in mind the specificities of this vulnerable population. In this chapter, we will provide an overview on the drug discovery and development path for children highlighting challenges and new frontiers of each phase from the discovery to the preclinical and clinical development as well as we will provide a slightest hint about paediatric biomarkers discovery, age-appropriate formulation, pregnancy, and perinatal pharmacology and in silico pharmacology. Finally, pricing and reimbursement policies for medicines and new and existing research initiatives in the field will be discussed.
Part of the book: Drug Discovery and Development