Background: Huge experiences, more than 20 years, have demonstrated the benefits to involve adult patients in the field of research and clinical trials. Their contributions are positive for the projects in terms of return on investment (reducing time and costs) but above all in terms of return on engagement (quality of life). Advocacy of adult patients or their representatives cover all the different stages of clinical research (from the definition of priorities to communicate the outcomes of a project).
In the case of children the scenario is different. The number or experiences are limited, which are focused basically in the information addressed to young patients involved in clinical trials (patient information sheet and assent document).
Young persons’ advisory groups (YPAGs) are groups of young people interested in the field of research and clinical trials, in science and to become a young advocate. Previous to the participation in any type of research project they receive suitable training to ensure that they have the right knowledge and skills.
The Young Persons’ Advisory Group acts as a scientific council of teenagers founded to improve clinical trials: the YPAG of Sant Joan de Deu Children’s research hospital in Spain is connected with the Clinical Research Unit. The team was created in January, 2015. The training programme lasted six months and it included content and skills about the four topics in which the hospital is performing projects: biomedicine, research, clinical trials and innovation.
After the training program, the team became the Youth Scientific Council of the Hospital. It means that those responsible for the different projects can ask for their consultation and advice with the aim to improve their projects and ensure patient centricity.
The members of the Young Persons’ Advisory Group have regular monthly meetings led by two facilitators of the team: the coordinator of the Clinical Trials Unit and the coordinator of the Patient Engagement in Research Area.
The methodology of every session follows a systematic process addressed to:
Educate and empower the young people in the specific topic of the project for which their help is requested. For example, improve the language, content and format of the assent document.
Dynamic interactivity to collect feedback aiming to improve the project by using the best methods to collect the information. For example, focus groups, questionnaires or surveys, personal interviews, etc.
The content and methods of the sessions are designed specifically for each project. In the first part of the session the principal investigator of the project is the expert responsible to educate the young people on the topic of the session. In the second part of the session, the facilitators perform the practical activity to facilitate the process to discuss delivery and contribution to the project.
In the last three years the Young Persons’ Advisory Group team has been involved in several projects. Below are some examples:
Survey to know children and teenagers’ opinion about drugs and involvement in medical research. The Paediatric Committee (PDCO) is the European Medicines Agency’s (EMA) scientific committee, where the national agencies are taking part to ensure the safety, efficiency and adequacy of medicine specifically for the children population. PDCO prepared an easy survey with eleven questions targeting children and teenagers between 10 and 18 years old, with the goal of knowing their opinion about drugs and their involvement in the medical research (clinical trials with medication). Answers had to be provided from March until May 2015. The goal of this survey was to obtain information about preferences of this population related with pharmaceutical dosage and types, as well as the difficulties they may face when consuming these medications. In addition, the intention was to know their opinion about their possible involvement in research studies with drugs. The Young Persons’ Advisory Group members, in collaboration with their educational institutions, collected more than 500 answers in Spain. Currently, analysed information is in publication phase (scientific publication) with the other data collected across other European countries
European regulation about clinical trials. In 2016, a consultation about ethical aspects of the European Paediatric Clinical Trials Regulation was opened. Young Persons’ Advisory Group members represented by iCAN (International Children’s Advisory Network) Youth Committee joined the public consultation period to review and suggest improvements to the legislation text.
Feedback about the clinical trials studies addressed to pediatric patients. The members of the Young Persons’ Advisory Group reviewed drafts of clinical trials submitted by the pharmaceutical industry, in particular about the treatment of:
The questions that they commented on and that allowed the improvement of the initial study draft proposal were: palatability, frequency of medical follow-ups and number of medical tests, quality of life data, formulation preferences, information for patients and families, etc. At the moment, in collaboration with other groups, the Group is planning to write a “white paper” that will allow to unify working procedures with the pharmaceutical industry and regulators, with the aim of standardising ways of collaboration.
Launch of the European YPAG Network (eYPAGnet). In May of 2017, the eYPAGnet achieved the recognition of EnprEMA and was officially launched. The specific European regulatory environment of paediatric clinical trials and the international and multicentre methodology to perform the studies, encouraged the creation of this network. During the next three years, the SJD Children’s Hospital is going to be the coordinator of the network.
The goals of eYPAGnet are:
Improve the capacity of collaboration with the different agents, who participate in the research process and development of innovative drugs
Gather a variety of experience related with different pathologies
Promote the planning and development of clinical research initiatives for children at the European level
Consolidate the curriculum of capacity-building and empowerment training programs for young patients
Promote and lead the creation of new groups
Empower the selection of professional careers in the scope of science, among the youth
Collaboration with the Ethics Working Group of EnprEMA (European Network of Paediatric Research of EMA). Partly harmonized Informed Consent/Assent template - document for paediatric CTs, was prepared during 2016. The document template was reviewed by the members of Eypagnet about the usefulness and understandable text contents, and it will be finalized after the careful review against the new “Pediatric Ethics Guideline” together with comments of the experts from the European Academy of Paediatrics (EAP). The template will be made publicly available on Enpr-EMA website.
Framework for involvement of patients in the activities of EMA. Young Persons’ Advisory Group was consulted by the EMA in the process to design the “Principles of involvement of patients in the activities of EMA”. In the upcoming months authors are going to help in the process of its implementation.