Case from the Young Persons’ Advisory Group (“YPAG”)
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).
The 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.
YPAG acts as a scientific council of teenagers founded to improve clinical trials: the YPAG of a children’s 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 YPAG have regular monthly meetings lead 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 YPAG 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, were 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. YPAG members, in collaboration with their educational institutions, collected more than 500 answers in Spain. Currently, analyzed information is in publication phase (scientific publication) with the other data collected across other European countries.
- European regulation about clinical trials. In 2016, was opened a consultation about ethical aspects of the European Pediatric Clinical Trials Regulation. YPAG members represented by ICAN Youth Committee joined the public consultation period to review and suggest improvement to the legislation text.
- Feedback about the clinical trials studies addressed to pediatric patients. The members of YPAG 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 the 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 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 the 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 of involvement of patients in the activities of EMA. YPAG 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.
1- Shared purpose
- Planning meetings with the stakeholders (researcher, pharma company, regulator, etc.) who request the scientific advice of YPAG.
- A meeting to evaluate the feedback of YPAG in terms of suggestions and improvements of the assessed project.
- Regular meetings with the most active researchers and clinicians involved in the clinical trials performed in the Hospital.
- Participation in events to introduce what is a YPAG and to inform about the expertise of YPAG.
- Report the outcomes of the feedback session with YPAGs will be sent to the stakeholders and this will analyse in qualitative way the results of the patients’ involvement.
- Maintain at least one annual meeting with the researchers and clinicians involved in the clinical trials performed in our Hospital.
- Participate at least in three events per year to introduce YPAG team.
- In every session, the members of YPAG know in advance the topic, the project and the objectives of the activity. The methodology always has a win-win focus; on one hand, the training of the young people, and on the other hand their contribution to different projects (clinical trials, innovation, etc). The first YPAG in the world was created in 2006 in Liverpool, and there is very close collaboration.Their experience was very useful to grow the team [in Spain].
2- Respect and accessibility
- Involvement of paediatric patients and healthy young people. In the case of patients, they are suffering different type of diseases in order to ensure the diversity of their feedback based on their experience as patients. Their experience in participating in a clinical trial is an important criterion when selected as a member of the group. The number of young people involved in YPAG is stable during a year, and they can belong to the group at least until 18 years. The longer they are involved, the more expertise and capabilities they can offer to different projects through contributing with their “scientific advice”.
- Offer the support that patients with special needs can have to be involved in YPAG: transportation, accessibility, dietary restrictions, etc.
- Inform all the members of the group and their families about the special needs that one member of the group can have. E.g. immunosuppressed.
- To have at least 50% patients as members of YPAG and not more than 2 young people with the same disease (e.g. cancer)
- To have members with different ages in the range between 12 to 18 years old.
- To have boys and girls in an equal distribution.
3- Representativeness of stakeholders
- Young patients are being recruited with the involvement of researchers and clinicians that perform clinical trials in the Hospital.
- Experience as patient participant in a clinical trial will be considered in the recruitment process of patients.
- Scientific interest will be a mandatory feature for all the candidates.
- Good level of English is also mandatory to ensure the involvement of the members of YPAG in international projects.
- The minimum age to be considered in the recruitment process will be 12 years old. At this age in the country where the work is taking place you have to sign the assent document.
- They have members that represent different socioeconomic backgrounds and diseases. Also healthy young people are involved to ensure that they can't have the bias of the disease and ensure that the general feedback that the team can provided is not connected with an specific condition. Related to the young patients they have members with chronic diseases (e.g. diabetes) or patients with complex illness conditions (e.g. cancer).
- They have experience to work with different stakeholders, but for privacy reasons they can not share names or data. Basically they are: pharma companies, research centers that perform non-commercial clinical trials, regulators and ethics committees.
- Annually, a recruitment process is set up, the previous criteria will be considered and also the result of an individual interview with the facilitators of YPAG.
4- Roles and responsibilities
- YPAG has two coordinators with experience in the field of clinical trials and education. They are the facilitators of the feedback sessions and the liaison with the different stakeholders. Any request of the stakeholders is going to be addressed to them by email or phone. A common email account was created in order to facilitate the interaction between the two facilitators and the stakeholders.
- The members of YPAG have also internal responsibilities in the group: president, secretary, education committee, means of communication committee, etc. (they are elected for 3 years).
- Facilitate interactions with the stakeholders.
- Provide always feedback in “real” projects (protocols, assent documents, etc).
- Stable involvement of the facilitators/coordinator at all the time, with independence of the project in which the team is involved. In any case, the role of professionals with expertise in the field of clinical trials and education will be needed.
- Annual approval of the internal role and responsibilities of the young people involved in YPAG. The minimum roles will be: president, secretary, coordinator of the education committee and coordinator of the means of communication committee.
- Participate in meetings with the stakeholders to introduce the methodology of patients’ involvement that is behind the YPAGs.
- Use official documents and projects to provide scientific feedback.
5- Capacity and capability for engagement
- A common training process will be offered to all the new members of YPAG. The training programme will be focused in four areas of expertise: biomedicine, clinical research, innovation and clinical trials. These four fields are related to the different research projects in what the members of YPAG can be involved.
- During the scientific advice sessions a representative of the stakeholder involved in the project (researcher, pharma company, regulator, etc.) is going to participate in the activity. This is to ensure that the content, terminology and goals of the project are understandable for the members of YPAG.
- Annually they perform a survey with the members of their team to know the evaluation of the training program and the projects in which they were involved. The biggest benefit for them is to know that they are helping other sick kids. Second, the access to a rigorous and innovative scientific knowledge, and last but not least, the development of personal/professional skills. The older members of the group help with the training process of the new members.
- A test is mandatory at the end of every training module to measure if the young people has achieved the educative goals.
- Specific training content is going to be offered to the young advocates related to the project in what they will be involved. This is going to be offered by a representative of the stakeholder participant.
6- Transparency in communication and documentation
- A private agreement will be signed by the parties (stakeholder and the Children’s Hospital) to ensure that the rules about documentation and confidentiality are aligned. When involving minors this is mandatory to ensure that the ethical principles are respected.
- The members of YPAG sign an agreement as volunteers of the project. In this document the rules about confidentiality, transparency and ethics are detailed.
- With the agreement of the stakeholders they are going to spread the word about the main outcomes of the different projects in what the young people have been involved.
- Signing an agreement is mandatory to start any type of project collaboration by all stakeholder.
- Signing an agreement as a volunteer of YPAG will be mandatory to all the members of YPAG.
- The dissemination of the projects in what the members of YPAG have been involved at least is going to be done in the website of YPAG.
7- Continuity and sustainability
- In the private agreement with the stakeholders the economic value of the scientific advice provided by YPAG is detailed. It can be translated into an economic income to ensure the sustainability of the group or in an in-kind contribution to improve the skills and capabilities of the young members (e.g. develop a new educational resource).
- All the projects and good practices are going to be collected in the annual report of YPAG.
- A detailed mapping database of stakeholders will be updated.
- All the relationship with the stakeholders will be detailed in the private agreement.
- The annual report is going to be sent to all the stakeholders involved in the year’s project and to the contacts of the mapping stakeholders database.
- Quarterly update of the database to include new contacts.
Positive impact for specific medicines development phases:
- Identification of young patients’ preferences about medicines that can impact directly in the adherence to the treatment (E.g. taste, formulation, dosis, etc).
- Suggestions related to protocol design
- Improvement of the information related to Patient Reported Outcome Measures (PROMs)
At this moment authors don't have standardized templates to be used in all the studies they are involved in. Currently, the limited experience with young patients advocacy requires a collective work with all the stakeholders to standardise procedures. For this reason authors are leading the European YPAG network (eYPAGnet). One of the activities that they will perform next year is the development of a guideline about the young patients involvement in drug development. They hope to have common templates and tools to be used across Europe.