Pediatric Inflammatory Bowel Disease (Netherlands) PSP Protocol

Purpose of the PSP and background

The purpose of this protocol is to clearly set out the aims, objectives and commitments of Pediatric IBD Priority Setting Partnership (PSP) in line with James Lind Alliance (JLA) principles. The Protocol is a JLA requirement and will be published on the PSP’s page of the JLA website. The Steering Group will review the Protocol regularly and any updated version will be sent to the JLA.

The JLA is a non-profit making initiative, established in 2004. It brings patients, carers and clinicians together in PSPs. These PSPs identify and prioritise the evidence uncertainties, or ‘unanswered questions’, that they agree are the most important for research in their topic area. Traditionally PSPs have focused on uncertainties about the effects of treatments, but some PSPs have chosen to broaden their scope beyond that. The aim of a PSP is to help ensure that those who fund health research are aware of what really matters to patients, carers and clinicians. The National Institute for Health Research (NIHR – www.nihr.ac.uk) coordinates the infrastructure of the JLA to oversee the processes for PSPs, based at the NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC), University of Southampton.

Inflammatory bowel disease (IBD) is a group of chronic disabling disorders of the gastrointestinal tract, including Crohn’s disease (CD) and ulcerative colitis (UC). The incidence of pediatric IBD has increased over the past ten years, and studies forecast a further rise in 2030. In pediatric IBD there are specific considerations to take regarding clinical course, treatment options and psychosocial impact of this disease on the child.

A lot of factors of IBD remain poorly understood. More research is needed to clarify a broad spectrum of uncertainties, such as the etiology of the disease, the ideal diagnostic and therapeutic approach, but also an effective approach to prevent illness and psychosocial burdening. Research agendas are traditionally composed by research groups and funders. As a consequence, evidence uncertainties that are essential to the patients, caregivers and physicians such as clinical dieticians or IBD-specialized nurses could be undervalued and understudied.

The aim of this study is to gather the most important research uncertainties in the management of pediatric IBD from the point of view of pediatric patients, parents/caregivers, and clinicians in the Dutch population using the James Lind Alliance (JLA) approach to investigate priority setting. This shared research agenda will be presented to the parties involved in pediatric IBD research, thus influencing the objectives of future research.

Aims, objectives and scope of the PSP

The aim of the pediatric IBD PSP is to identify the unanswered questions about pediatric IBD from patient, carer and clinical perspectives and then prioritise those that patients, carers and clinicians agree are the most important for research to address.

The objectives of the PSP are to:

• work with patients, carers and clinicians to identify uncertainties about the broad spectrum of the disease, e.g. prevention, treatment impact, psychosocial consequences, diet, etc.
• to agree by consensus a prioritised list of those uncertainties, for research
• to publicise the results of the PSP and process
• to take the results to research commissioning bodies to be considered for funding.

The scope of the pediatric IBD PSP is defined as:

• Causality, potential preventive measures, symptoms, follow-up regimen, treatment strategy (effectiveness, side effects), prognosis (e.g. likelihood of necessary surgery in the future), the effect of the disease on the development of the child (growth, puberty, psychosocial, educational, etc), psychosocial impact (job prospective, relations, depression, anxiety, etc), coping (helping patients in their independence and in taking charge, stimulating shared decision making), ‘choosing wisely’ (https://choosingwisely.org), the effect of decision aids, and the transition from pediatric to adult care. Our study will help researchers and research funders in their decision which study subject in this broad scope should take precedent.
• Pediatric patients are defined as patients up to and including the age of 20 years.
• Submissions that are not in the form of “research questions” will be considered in-scope. It is expected that patients and parents will not always be able to form specific questions, but will be able to express their research question or research topic in, for example, a description of an experience. These submissions will be interpreted, rephrased and presented to the Steering Group. After assessment and approval from the Steering Group, the rephrased submission will be added to the list of in-scope submissions.

The PSP will exclude from its scope:

• Diseases other than pediatric IBD
• Research questions that are unethical to investigate.
• Submissions that are not research questions or research topics, and from which it is not possible to interpret a research question.
• Dutch questionnaires will be provided. As a consequence, non-Dutch respondents will naturally be excluded from the study due to a language barrier.

All out-of-scope submissions will have to be approved by the Steering Group.

The Steering Group is responsible for discussing what implications the scope of the PSP will have for the evidence-checking stage of the process. Resources and expertise will be put in place to do this evidence checking.

The Steering Group

The Steering Group includes membership of patients and carers and clinicians , as individuals or representatives from a relevant group.

The pediatric IBD PSP will be led and managed by a Steering Group involving the following:

Patient and carer representative/s:

Patients: Isabella, Yanniek, Dana en Stijn
Parents/carers: Simone van Harten, Kees/Ella Mouthaan, Hannie/Bas Schoonderwoert and Germa van der Weide

Clinical representative/s:

• Pediatric gastro-enterologists: Tim de Meij (Amsterdam UMC) and Patrick van Rheenen (UMC Groningen)
• Gastro-enterologist: Nanne de Boer (Amsterdam UMC)
• IBD-nurse practitioner: Pamela Reimering-Hartgerink (Medisch Spectrum Twente) and Heidi van de Wielen (Jeroen Bosch Ziekenhuis, Den Bosch)
• Dietician: Esther van Steenbergen (Erasmus MC, Rotterdam)
• Psychologists: Anne-Marije Goutbeek (Amsterdam UMC) and Alice van Dijk-Lokkart (Amsterdam UMC)

Project coordinators: J. Jagt and S Thoma

James Lind Alliance Adviser: Jonathan Gower, JLA

Chair of the Steering Group: Menne Scherpenzeel

The Steering Group will agree the resources, including time and expertise that they will be able to contribute to each stage of the process, with input and advice from the JLA.

Partners

Partners are organisations or groups who will commit to supporting the PSP, promoting the process and encouraging their represented groups or members to participate. Organisations which can reach and advocate for these groups will be invited to become involved in the PSP. Partners represent the following groups:

• Children/adolescents who have pediatric IBD
• Carers of children/adolescents who have pediatric IBD
• Health and social care professionals - with experience of pediatric IBD

Partners in this PSP are:

- Patients’ association “Crohn & Colitis NL”
- KiCC (Kids Crohn’s and colitis) working group for research

This protocol will be updated when other partners have been approached by the members of the Steering Group.

Exclusion criteria

Some organisations may be judged by the JLA or the Steering Group to have conflicts of interest. These may be perceived to potentially cause unacceptable bias as a member of the Steering Group. As this is likely to affect the ultimate findings of the PSP, those organisations will not be invited to participate. It is possible, however, that interested parties may participate in a purely observational capacity when the Steering Group considers it may be helpful.

The methods the PSP will use

This section describes a schedule of proposed steps through which the PSP aims to meet its objectives. The process is iterative and dependent on the active participation and contribution of different groups. The methods used in any step will be agreed through consultation between the Steering Group members, guided by the PSP’s aims and objectives. More details of the method are in the Guidebook section of the JLA website at www.jla.nihr.ac.uk where examples of the work of other JLA PSPs can be seen.

Step 1: Identification and invitation of potential partners

Potential partner organisations will be identified through a process of peer knowledge and consultation, through the Steering Group members’ networks. Potential partners will be contacted and informed of the establishment and aims of the pediatric IBD PSP.

Step 2: Awareness raising

PSPs will need to raise awareness of their proposed activity among their patient, carer and clinician communities, in order to secure support and participation. Depending on budget, this may be done by a face-to-face meeting, or there may be other ways in which the process can be launched, e.g. via social media. It may be carried out as part of steps 1 and/or 3. The Steering Group should advise on when to do this. Awareness raising has several key objectives:

• to present the proposed plan for the PSP
• to generate support for the process
• to encourage participation in the process
• to initiate discussion, answer questions and address concerns.

Step 3: Identifying evidence uncertainties

The pediatric IBD PSP will carry out a consultation to gather uncertainties from patients, carers and clinicians. A period of 3 weeks will be given to complete this exercise (which may be revised by the Steering Group if required).

The pediatric IBD PSP recognises that the following groups may require additional consideration.

- Patients in the lower income class. To reach this group, we will distribute surveys on paper as well as online. This way, potential participants that can’t afford a computer at home, will be able to fill in the surveys.
- Since it isn’t possible for the youngest children to fill in a questionnaire, we will conduct 1-on-1 interviews with children in the age category of 6-8 years. Additionally, we will ask parents from children younger then 8 years to think about their needs. Lastly, we will ask older children, that were diagnosed at such a young age, to think back. We will ask them what, in retrospect, they would have wanted to know when they were diagnosed.
- To include the young adults of 18-20 years old and their parents/caregivers/physicians, we will have to recruit through separate channels, since they aren’t a part of the regular pediatric field. To reach this group, we will contact the gastro-enterologists and nurses that specifically work with this group.
- We will monitor demographics like age, treatment center, educational attainment, and specialty (doctor, specialized nurse, dietician, psychologist, etc), to ensure representation of all groups, and intervene if needed.

The Steering Group will use the following methods to reach the target groups:

- Recruitment of the patients and their parents/caregivers will be targeted through the pediatric IBD consultation hours in the participating hospitals. The participating hospitals are the ones that are involved in the Kids with Crohn's, Colitis (KiCC) working group for collaborative pediatric IBD research in the Netherlands. The treating physicians and IBD-specialized nurses will inform the patients and their caregivers of this study. They will give the eligible candidates a patient information letter in which the purpose and execution of the study are clearly explained. This letter will provide a link to the questionnaire.
- For the recruitment of the clinicians, we will target physicians such as pediatricians, IBD-specialized nurses, dieticians, and psychologists. They will be approached via meetings of the KiCC and via the partners.
- Additionally, we will upload the questionnaires on our website. This website will be promoted by the patients’ association for Crohn’s & Colitis through their network, and via social media (e.g. Instagram and Facebook).
- The questionnaires will circulate until we reach our target number of inclusions.

Existing sources of evidence uncertainties may also be searched. For example, we will include the research uncertainties named in the guideline from the NVK and research recommendations from systematic reviews.

Step 4: Refining questions and uncertainties

The consultation process will produce ‘raw’ questions and comments indicating patients’, carers’ and clinicians’ areas of uncertainty. These raw questions will be categorised and refined by S. Thoma/J.Jagt into summary questions which are clear, addressable by research, and understandable to all. Similar or duplicate questions will be combined where appropriate. Out-of-scope and ‘answered’ submissions will be compiled separately. The Steering Group will have oversight of this process to ensure that the raw data is being interpreted appropriately and that the summary questions are being worded in a way that is understandable to all audiences. The JLA Adviser will observe to ensure accountability and transparency.

This will result in a long list of in-scope summary questions. These are not research questions and to try and word them as such may make them too technical for a non-research audience. They will be framed as researchable questions that capture the themes and topics that people have suggested.

The summary questions will then be checked against evidence to determine whether they have already been answered by research. This will be done by S. Thoma/J.Jagt. The PSP will complete the JLA Question Verification Form, which clearly describes the process used to verify the uncertainty of the questions, before starting prioritisation. The Question Verification Form includes details of the types and sources of evidence used to check uncertainty. The Question Verification Form should be published on the JLA website as soon as it has been agreed to enable researchers and other stakeholders to understand how the PSP has decided that its questions are unanswered, and any limitations of this.

Questions that are not adequately addressed by previous research will be collated and recorded on a standard JLA template by S.Thoma/J.Jagt. This will show the checking undertaken to make sure that the uncertainties have not already been answered. The data should be submitted to the JLA for publication on its website on completion of the priority setting exercise, taking into account any changes made at the final workshop, in order to ensure that PSP results are publicly available.

The Steering Group will also consider how it will deal with submitted questions that have been answered, and questions that are out of scope.

Step 5: Prioritisation – interim and final stages

The aim of the final stage of the priority setting process is to prioritise through consensus the identified uncertainties about pediatric IBD. This will involve input from patients, carers and clinicians. The JLA encourages PSPs to involve as wide a range of people as possible, including those who did and did not contribute to the first consultation. There are usually two stages of prioritisation.

1. Interim prioritisation is the stage where the long list of questions is reduced to a shorter list that can be taken to the final priority setting workshop. This is aimed at a wide audience, and is done using similar methods to the first consultation. With the JLA’s guidance, the Steering Group will agree the method and consider how best to reach and engage patients, carers and clinicians in the process. The most highly ranked questions (around 25) will be taken to a final priority setting workshop. Where the interim prioritisation does not produce a clear ranking or cut off point, the Steering Group will decide which questions are taken forwards to the final prioritisation.

2. The final priority setting stage is generally a one-day workshop facilitated by the JLA. With guidance from the JLA and input from the Steering Group, up to 30 patients, carers and clinicians will be recruited to participate in a day of discussion and ranking, to determine the top 10 questions for research. All participants will declare their interests. The Steering Group will advise on any adaptations needed to ensure that the process is inclusive and accessible.

Dissemination of results

The Steering Group will identify audiences with which it wants to engage when disseminating the results of the priority setting process, such as researchers, funders and the patient and clinical communities. They will need to determine how best to communicate the results and who will take responsibility for this. Previous PSPs’ outputs have included academic papers, lay reports, infographics, conference presentations and videos for social media.

It should be noted that the priorities are not worded as research questions. The Steering Group should discuss how they will work with researchers and funders to establish how to address the priorities and to work out what the research questions are that will address the issues that people have prioritised. The dissemination of the results of the PSP will be led by dr. T. de Meij and Menne Scherpenzeel/Daniëlle van der Horst.

The JLA encourages PSPs to report back about any activities that have come about because of the PSP, including funded research. Please send any details to jla@soton.ac.uk.

Agreement of the Steering Group

The pediatric IBD PSP Steering Group agreed the content and direction of this Protocol on June 28th, 2021.