Informed Participation and Patient Empowerment: A Patient- Centered Approach to Improve Pediatric Clinical Research

Abstract

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.

Medical Device Development for Children and Young People—Reviewing the Challenges and Opportunities

“…Globally, groups of healthy children and communities of pediatric patients have been established to contribute to research, on the basis of the recognition that they are "experts in their own lives" [82], as well as the fact that they provide valuable insights into service experience, research participation, and their own health and care needs [83]. Promoting the participation of young people in their healthcare produces positive outcomes, including a reduction in vulnerability and anxiety, respect, confidence, and cooperation; thus, active participation in research development and delivery should in turn engender the same results (every child has a right to be heard, UNICEF [84]). In relation to MD development for children, structured groups have emerged, including Young Persons Advisory Groups (YPAGs) [18], including the YPAG of the National Institute for Health Research (NIHR) Children and Young People Medtech Cooperative [85].…”