Congenital Heart Disease PSP Protocol
Purpose of the PSP and background
The purpose of this protocol is to clearly set out the aims, objectives, and commitments of the Congenital Heart Disease 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 and Care 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.
The Congenital Heart Disease PSP has developed as a natural progression of two other processes. In 2017/18, the congenital heart disease community in the UK came together in a national priority-setting exercise to generate a list of priorities for research focused on single ventricle heart conditions (Cardiol Young 2019;29:303-309) which was conducted outside of the JLA process. More recently, the Adult Cardiac Surgery PSP identified a Top 10 list of priorities in cardiac surgery (BMJ Open 2020;10:e038001) but specifically excluded children, adult congenital heart disease, and transplantation. With the success of this process, the Society for Cardiothoracic Surgery in Great Britain and Ireland (SCTS) Academic and Research Committee encouraged the congenital leads to take forward a PSP in congenital cardiac surgery and after consultation with the community, a broader Congenital Heart Disease PSP encompassing all aspects of management was proposed.
Aims, objectives, and scope of the PSP
The aim of the Congenital Heart Disease PSP is to identify the unanswered questions about congenital heart diseases throughout life, including prior to birth, from patient, carer (primarily parents) 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 all aspects of the management of congenital heart disease
- agree by consensus a prioritised list of those uncertainties, for research
- publicise the results of the PSP and the process
- take the results to research commissioning bodies to be considered for funding.
The scope of the Congenital Heart Disease PSP is defined as: the management of congenital heart disease throughout life, including prior to birth, focusing on:
- Diagnosis, during pregnancy or after birth
- Treatment (medical therapy, catheter intervention, surgery including mechanical support & transplantation, lifestyle, or psychosocial intervention), and
- Outcomes of the conditions and/or treatments and the impact on patients and their families, including the physical, psychological, and social effects of living with congenital heart disease.
The PSP will exclude from its scope questions about:
- Non-management related aspects of congenital heart disease, such as aetiology or non-clinical genetics
- Acquired heart disease, other than occurring in the context of congenital heart disease
- Other co-morbidities, such as non-cardiac aspects of associated syndromes.
The Steering Group is responsible for discussing what implications the scope of the PSP will have for the evidence-checking stage of the process (making sure the questions that go into the prioritisation have not already been answered) and confirming that the resources and expertise have been put in place to undertake it.
The Steering Group
The Steering Group includes members of patients, carers, and clinicians, as individuals or representatives from a relevant group. The Congenital Heart Disease PSP will be led and managed by a Steering Group involving:
Patient and carer representatives:
- Julie Wootton, Parent and Chair of the Children’s Heart Federation
- Michael Cumper, Patient and Vice President, The Somerville Foundation
- Sarah Murray, Parent and Chair of the BHF Clinical Research Collaborative Cardiac Surgery PPI group
- Alex Miskin, Parent of child with congenital heart disease
- Fraser Pender, Patient with congenital heart disease
- Jara Weinkauf, Patient with congenital heart disease
- Sasha Rooprai, Parent of child with congenital heart disease
- Mr Nigel Drury, Clinician Scientist and Consultant in Paediatric Cardiac Surgery, Birmingham Children’s Hospital, Congenital Representative on the SCTS Academic and Research Committee, and PSP Lead
- Prof John Simpson, Consultant Paediatric and Fetal Cardiologist, Evelina London Children’s Hospital, and President, British Congenital Cardiac Association (BCCA)
- Mr Rafael Guerrero, Chief of Congenital Cardiac Surgery, Alder Hey Children’s Hospital, Liverpool, and Chair, Congenital Cardiac Surgery sub-committee, SCTS
- Dr Katherine Brown, Consultant in Cardiac Paediatric Intensive Care, Great Ormond Street Hospital, London
- Dr Louise Coats, Consultant Adult Congenital Cardiologist, Freeman Hospital, Newcastle
- Dr John Thomson, Consultant Interventional Cardiologist, Leeds Teaching Hospitals
- Dr Clare Herd, Information Specialist, Institute of Applied Health Research, University of Birmingham
- Dr Giovanni Biglino, Senior Lecturer in Cardiovascular Bioinformatics, University of Bristol
- Sharmaine Afferion, Administrator, Institute of Cardiovascular Sciences, University of Birmingham
James Lind Alliance:
- Katherine Cowan, Senior JLA Advisor and Chair of the Steering Group
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.
In some cases, it has been suggested that researchers are represented on the Steering Group, to advise on the shaping of research questions. However, researchers cannot participate in the prioritisation exercise. This is to ensure that the final prioritised research questions are those agreed by patients, carers, and clinicians only, in line with the JLA’s mission.
Organisations and individuals will be invited to be involved with the PSP as 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:
- people who have congenital heart disease, both children and adults
- carers of people who have congenital heart disease, including parents and other family members
- health and social care professionals with experience of the management of congenital heart disease.
Agreed key partner organisations are:
- Children’s Heart Federation
- The Somerville Foundation
- British Congenital Cardiac Association (BCCA)
- Society for Cardiothoracic Surgery in Great Britain and Ireland (SCTS)
Other confirmed partners include Little Hearts Matter, Tiny Tickers, Young at Heart, Max Appeal, the Paediatric Critical Care Society (PCCS), the Congenital Cardiac Anaesthesia Network (CCAA), Paediatricians with Expertise in Cardiology Specialist Interest Group (PECSIG), the Society of Clinical Perfusion Scientists (SCPS), the Congenital Cardiac Nurses Association (CCNA), the British Adult Congenital Cardiac Nurse Association (BACCNA), and the Centre for BME Health in Leicester.
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 and 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 PSP.
Step 2: Awareness raising
The PSP will raise awareness of the proposed activity among patient, carer, and clinician communities, to secure support and participation. This will be achieved primarily by email and social media, through engagement with partner organisations to reach a wide range of stakeholders from across the breadth of congenital heart disease. 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 Congenital Heart Disease PSP will carry out a consultation to gather uncertainties from patients, carers, and clinicians. A period of three months will be given to complete this exercise, which may be revised by the Steering Group if required. The Steering Group recognises that South Asian communities are disproportionately affected by congenital heart disease and experience worse outcomes from interventions whilst being underrepresented in consultations. To address this need, the committee sought advice and input from the Centre for BME Health in Leicester and have included a parent of South Asian ethnic origin on the committee. The initial survey will be translated into Bengali, Urdu, Gujarati, and Hindi through the Centre to increase participation from within these communities, and also into Polish and Welsh to further widen access amongst non-English speakers.
The Steering Group will use the following methods to reach the target groups:
- Online survey, using REDCap through the University of Birmingham licence, in English only
- Paper survey, to be available as a PDF for printing, including in other languages, with a Freepost address
- Social media, such as twitter, Facebook, Instagram and TikTok, to promote the survey (although additional questions obtained via social media responses will be included in the survey)
- Consider supplemental activities, such as discussion groups or interviews to engage with young people, or tailored communications to increase responses from groups identified as poor responders to the survey.
Existing sources of information about treatment uncertainties for patients, carers, and clinicians will be searched. These can include question-answering services for patients and carers and for clinicians; research recommendations in systematic reviews and clinical guidelines; protocols for systematic reviews being prepared and registers of ongoing research.
The starting point for identifying sources of uncertainties and research recommendations is NHS Evidence: www.evidence.nhs.uk. Other sources of uncertainties for research recommendations may include:
- Cochrane database
- NHS Evidence – UK DUETs
- NICE Guidance and NICE Research recommendations database
- BMJ Clinical Evidence
- Relevant treatment guidelines published by American Heart Association. American College of Cardiology, European Society of Cardiology, and others.
Step 4: Refining questions and uncertainties
The consultation process will produce ‘raw’ questions and comments indicating the areas of uncertainty identified by patients, carers, and clinicians. These raw questions will be categorised and refined by the Information Specialist(s) 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 by the Information Specialist(s), to determine whether they have already been answered by research. The PSP will complete the JLA Question Verification Form, which 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 and 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.
Questions that are not adequately addressed by previous research will be collated and recorded on a standard JLA template by the Information Specialist(s). 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, considering any changes made at the final workshop, 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 congenital heart disease in children and adults. The Congenital Heart Disease PSP therefore will split into two parallel tracks focusing on the fetus/child and the adult, respectively, for the prioritisation stages; uncertainties concerning transfer of care could be included in either track, at the discretion of the Steering Group. These processes will involve input from patients, carers, and clinicians. The JLA encourages PSPs in general 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.
- 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) in each track will be taken forward to the two final priority setting workshops. If 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.
- Ideally the final priority setting stage will be two one-day workshops, one fetal/paediatric and one adult, 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 each workshop for 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 PSP 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 the PSP Lead.
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 email@example.com.
Agreement of the Steering Group
The PSP Steering Committee agreed the content and direction of this Protocol on 21st July 2021.