Cystic Fibrosis Research Priorities Refresh Protocol

Purpose of the PSP and background

The purpose of this protocol is to clearly set out the aims, objectives and commitments of the Cystic Fibrosis JLA Research Priorities Refresh. The Protocol is a JLA requirement and will be published on the relevant page of the JLA website. The Steering Committee 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. The National Institute for Health Research (NIHR – www.nihr.ac.uk) coordinates the infrastructure of the JLA which is based at the NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC), University of Southampton.

In January 2017 the James Lind Alliance Priority Setting Partnership (JLA PSP) in Cystic Fibrosis (CF), which brought together people with CF, family members and clinical care teams, globally, published its Top 10 health priorities for CF. (See full paper here). Over 600 people took part from more than 30 countries and the majority were from the patient community. The partnership has been influential. The original publication has been cited 80 times and has stimulated both research and research funding, including a recent UK National Institute for Health and Care Research (NIHR) cross programme themed call in CF. Several of the top 10 questions are now being addressed through funded research, both from the NIHR and other funders nationally (e.g. the UK CF Trust) and internationally (US CF Foundation). A summary of some of the research funded in response to the JLA PSP CF “You said, we did: How the Cystic Fibrosis priorities have been addressed since they were agreed in 2017” can be found here.

Aims, objectives and scope of the Cystic Fibrosis JLA Research Priorities Refresh

Whilst considerable progress has been made in addressing many of the health priorities identified by the JLA PSP in CF there is work still to be done. Additionally, the last year has seen some significant changes in CF, such as access to the new, highly effective disease modifying triple therapy Kaftrio^TM, and changes in the provision of care and also delivery of research, due to the COVID pandemic. Many of these changes in the clinical and research landscape are likely to persist beyond the pandemic. The time is therefore right for a refresh of the research priorities for CF. The aim of this refresh, working with people living with CF, their carers and family members, researchers and clinicians is to:

• Define which of the current top 10 priorities still need to be addressed
• Determine if some questions have been answered or have become less relevant
• Decide if questions in the top 11 – 20 are now more important
• Consult with the CF community to see if there are new research priorities as a result in the recent changes
• in treatment, care and delivery of research
• Agree by consensus a refreshed list of research priorities
• Publicise the results of the JLA research priority refresh and the process.

The outputs of this priority refresh exercise will help inform future research strategies and funding decisions
for those who deliver, support and fund CF research in the UK.

The scope of the Cystic Fibrosis JLA Research Priorities Refresh is defined as:

• Treatment options and treatment effects
• Care and management (includes social, emotional and psychological well-being)
• Co-existing conditions

The Cystic Fibrosis JLA Research Priorities Refresh will exclude from its scope questions about:

• Access to services

The Steering Committee is responsible for discussing what implications the scope of the JLA Research Priorities Refresh 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 Committee and Management Team

The Cystic Fibrosis JLA Research Priorities Refresh will be led and managed by a Steering Committee and
supported by a Management Team. The Steering Committee and Management Team includes in their
membership; people living with CF, their carers and family members, researchers, and clinicians, as individuals or representatives from a relevant group.

* clinician

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

Organisations and individuals will be invited to be involved with the Cystic Fibrosis JLA Research Priorities Refresh as partners. Partners are organisations or groups who will commit to supporting the JLA Research Priorities Refresh, 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 JLA Research Priorities Refresh. One of the key principles of the JLA method is that patients, carers and clinicians are involved in the setting of priorities for research. Our proposed refresh methodology will also include the scientific research community as we recognise the value of their expert contributions to the priority setting but also the importance of gaining their buy-in to the delivery of the identified research priorities for the CF community. Partners in the CF JLA Research Priorities Refresh will represent the following groups:

• people living with cystic fibrosis
• carers and/ or family members of people living with cystic fibrosis
• health and social care professionals - with experience of cystic fibrosis
• researchers with appropriate expertise in cystic fibrosis research.

Exclusion criteria:

Some organisations, such as organisations from the life-sciences sector or other industry representation, may be judged by the JLA or the Steering Committee to have conflicts of interest. As this is likely to affect the ultimate findings of the JLA Research Priorities Refresh, 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 Committee considers it may be helpful.

The methods the JLA research priorities refresh will use

This section describes a schedule of proposed steps through which the Cystic Fibrosis JLA Research Priorities Refresh 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 Committee members, guided by the aims and objectives of the Cystic Fibrosis JLA Research Priorities Refresh.

Step 1: Identification and invitation of potential partners

Potential partner organisations will be identified through a process of peer knowledge and consultation, through the Management Team and Steering Group members’ networks, and will also include partner organisations identified through the original JLA PSP. Potential partners will be contacted and informed of the establishment and aims of the Cystic Fibrosis JLA Research Priorities Refresh.

Step 2: Awareness raising

There is a need to raise awareness of the proposed JLA Research Priorities Refresh activity among patient, carer, research and clinical communities, in order to secure support and participation. This will be achieved through a variety of channels and using different approaches for the different audiences but may include social media, newsletter updates, promotion at virtual or face-to-face meetings. A marketing and communications plan will be developed to support this activity. Awareness raising has several key objectives:

• to present the proposed plan for the JLA Research Priorities Refresh
• to generate support for the process
• to encourage participation in the process
• to initiate discussion, answer questions and address concerns

Step 3: Review of the JLA PSP Top 20 priorities for cystic fibrosis

The Management Team will design a survey, distributed to the patient, clinical and research community, which will be accessed through an online platform. This survey will ask the respondent to review each of the top 20 health priorities identified during the original JLA PSP process and indicate which of these they think are still relevant. From that list of the original priorities, they will then be asked to prioritise 3. Once the respondent has reviewed the existing top 20 research priorities and identified their most important 3, they will be asked to suggest up to 2 additional research questions which they feel are priorities for CF. A range of approaches will be used to promote this survey, which will include social media. Consideration will also be given to how to reach groups that may be more difficult to engage with, and targeted communications approaches will be defined which are inclusive of these groups.

Step 4: Refining questions and priorities

The initial survey process will produce a list of priorities identified from the original JLA PSP to still be relevant to the community along with a number of ‘raw’ questions and new research priorities submitted by patients, carers, researchers and clinicians. These outcomes will be categorised and refined by Nicola Rowbotham (Post-doctoral Clinical Researcher) and Sherie Smith (Cochrane Systematic Reviewer) into summary priorities which are clear, addressable by research, and understandable to all. Similar or duplicate questions/ priorities 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 data is being interpreted appropriately and that the summary questions/ priorities 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/ priorities.

The summary questions/ priorities will then be checked against evidence to determine whether they have already been answered by research. This will be done by Nicola Rowbotham and Sherie Smith. The JLA Question Verification Form will be completed, 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 whether the research priority has already been addressed. This will also be supported by undertaking a refresh of a “gaps in the evidence for treatment decisions in CF” review (Rowbotham NJ, Smith S, Prayle AP, et al. Gaps in the evidence for treatment decisions in cystic fibrosis: a systematic review. Thorax 2019;74:229-236). 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 JLA Research Priorities Refresh has decided that its questions are unanswered, and any limitations of this.

Questions/ priorities that are not adequately addressed by previous research will be collated and recorded on a standard JLA template by Nicola Rowbotham and Sherie Smith. This will show the checking undertaken to make sure that the priorities have not already been addressed. 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 JLA Research Priorities Refresh results are publicly available.

The consolidated “long-list” of research priorities to be included in the second prioritisation survey will be made up the highest ranked 15 priorities from the original JLA PSP top 20, together with up to 35 newly submitted research priorities/ questions. 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 JLA Research Priorities Refresh is to prioritise through consensus the identified questions/ priorities about cystic fibrosis. This will involve input from patients, carers, researchers and clinicians. This stage will 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/ priorities 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 where respondents will be asked to select their personal top 10 priorities from the consolidated long-list of up to 50 priorities. With the JLA’s guidance, the Steering Group will agree the method and consider how best to reach and engage patients, carers, researchers and clinicians in the process. The most highly ranked questions (around 15) 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/ priorities are taken forwards to the final prioritisation.
2. The final priority setting stage is generally a workshop facilitated by the JLA. With guidance from the JLA and input from the Steering Group, patients, carers, researchers and clinicians will be invited to participate in an online workshop 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 JLA Research Priorities Refresh, such as researchers, funders, commercial sponsors and the patient and clinical communities. They will need to determine how best to communicate the results and who will take responsibility for this. This will include academic papers, lay reports, infographics, conference presentations and videos for social media.

Agreement of Steering Group

The CF JLA Research Priorities Refresh Steering Group agreed the content and direction of this Protocol on 23rd November 2021.