Challenges and Solutions in Establishing a Pediatric Patient Engagement Initiative

by Maria Cavaller-Bellaubi

Coauthors: Eva Degraeuwe (1,2), Maria Cavaller (1,3), Laura Persijn (1,2), Elke Gasthuys (1), Evelien Snauwaert (1,2), Lieve Nuytinck (1), Ann Raes (1,2), Johan Vande Walle (1,2) Affiliations: (1) Ghent University, departement of Internal Medicine and Pediatrics (2) Ghent University Hospital, Departement of Pediatric Nephrology (3) EURORDIS - Rare Diseases Europe


Background/Aims: For approximately 70% of rare diseases, the onset occurs during childhood but for many diseases, clinical signs of symptoms do not appear in the first days or months following birth. Lack of authorised medicines and consequent off-label use is a significant problem in the paediatric population. After the 2007 European Pediatric Regulation, a vast increase in the amount of pediatric clinical trials has been initiated (1). Many trials were considered unsuccessful, partially related to poor study design, but mostly due to insufficient recruitment rates and low trial retention and therefore its completion (2). However, incorporating patients’ perspectives and insights within the whole drug development process has proven to improve clinical trial design, recruitment and retention rates, and overall resulted in better health outcomes and benefits for all the stakeholders involved (3).
Yet patient engagement (PE) is currently not considered standard practice (4). Initiatives such as European Patient’s Academy on Therapeutic Innovation (EUPATI) and EURORDIS Open Academy provide training courses for patients and researchers (5). Within the IMI2-funded public private project, conect4children, young patients and their families are in the heart of the project and assigns them an active role in the development of the different clinical trials and activities that are going to be conducted by the project. Other peadiatric networks are good examples of communication facilitation between different young patient groups (5), YPAGnet, iCAN, and some countries have founded a well-developed young patient or patient public involvement networks such as France, Spain and Italy(6). However, applying top-level guidelines and advice are not so easily transposable within a new country’s setting.
Method: This study describes the challenges in creating PE groups within a pediatric setting and a new country. As case-study, the start-up of the PE group within pediatric nephrology at Ghent University Hospital will be portrayed.
Results: PE group design and recruitment methodology should be carefully standardized and improved. When involving children and young patients in drug development, the social, cultural, and economic environment of the participant should be considered. For the start-up of our local Belgian PE group, support from the European Young Person’s Advisory Group Network (eYPAGnet) was provided to form a common framework. However, when locally applying this framework, the development was confronted with additional challenges.
An alternative approach to receive funding had to be taken. PE session materials, such as tailored questionnaires and age-appropriate educational materials on clinical trials were locally developed. Collaborations with the children psychology department has proved to be beneficial. Moreover, ethical committee submission process had to be initialized considering the novelty of the PE group design.
Take home messages:
– Submission processes as well as potential funding from stakeholders need to be mobilized to smoothen PE group start-up.
– Efforts from international PE communities should consider difficulty into local translation.
– Standardization of PE material, adapted per age category.