Purpose of the PSP and background
The purpose of this protocol is to clearly set out the aims, objectives and commitments of the Spinal Sarcoma 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 (every 2-3 months) 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.
Sarcomas are a rare group of cancers that can arise in any part of the body, including bone, tendons, blood vessels and fatty tissues, making them prone to late diagnosis. In other malignancies early diagnosis has an impact on the complexity of therapeutic procedures, quality of life and survival rates. Over 5000 patients are diagnosed with sarcoma in England each year. Over 600 of these diagnoses arise in bone and approximately one tenth of these, equating to around 60 patients annually, occur in the spine. Unlike bony sarcomas that arise in the extremities, spinal sarcomas have a relatively poor prognosis, the reasons for which are unclear. Spinal sarcomas, which tend to be smaller in size than those found in the extremities, may present late in the disease course when patients develop neurological deficits. These neurological symptoms often mimic other benign spinal, neurological or musculoskeletal conditions and this in turn, may contribute to a late diagnosis. Treatment tends to involve a combination of surgery, chemotherapy and radiotherapy. As with many malignancies, early diagnosis is essential and surgery is potentially less complex with fewer complications and risks, adjuvant therapies such as chemotherapy and radiotherapy may be more effective and ultimately the prognosis may be improved.
Research to date has involved collaboration between clinicians and researchers without fully considering patients and their carers. Similarly, the development and testing of pharmaceuticals and technological advances in this area have not always considered the needs of patients and clinicians.
A PSP is urgently required, to increase awareness of these unusual cancers, promote early investigation and diagnosis and also to bring together the needs of patients and clinicians when addressing and organising areas of research in the future. Ongoing research will identify the best and most effective treatments resulting in more favourable outcomes and increased survivorship. Our primary aim is to bring together the spectrum of clinicians, patients, carers and charities from each step in the patient pathway, using the JLA process, to determine the top 10 research priorities in Spinal Sarcoma.
Aims, objectives and scope of the PSP
The aim of the spinal sarcoma PSP is to identify the unanswered questions about spinal sarcomas 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, their families, carers and clinicians to identify uncertainties about symptom recognition, early diagnosis, treatment options, long-term rehabilitation and support (including mental health and wellbeing).
- 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 spinal sarcoma PSP is defined as:
- patients living with spinal sarcoma identified through the five main clinical centres treating this condition in the UK including Birmingham, London, Manchester/Oswestry, Newcastle and Oxford
- carers of either patients who are currently living with sarcoma and its long-term effects or those who may have lost a relative or friend to a spinal sarcoma diagnosis
- clinicians including oncologists, surgeons, radiologists, Allied Health Professionals (AHPs eg physiotherapists, occupational therapists etc), psychologists, play therapists, pathologists who can be identified via the aforementioned treatment centres.
- charities and patient-led support groups involved either in sarcoma cancer research or who provide support to patients and their families and friends
The scope of this PSP will aim to bring together clinicians, patients and carers to identify and prioritise gaps in evidence in the assessment, treatment and care of patients with spinal sarcoma based in the UK.
It is recognised that international participants may respond to the surveys in which case their priorities will be reported separated and shared with their relevant overseas groups.
The Steering Group
The Steering Group includes membership of patients and carers and clinicians , as individuals or representatives from a relevant group.
The spinal sarcoma PSP will be led and managed by a Steering Group involving the following:
Patient and carer representative/s:
Mr James and Mrs Vicki Woodall – parents of paediatric patient
Mrs Tina Lafford – mother of adult patient
Mr Andrew Ohagwa- RNOH patient
Other members to be confirmed
Mr Marcin Czyz, Consultant Spinal Surgeon, Robert Jones and Agnes Hospital, Oswestry
Dr Sorrel Bickley, Director of Research, Policy and Support, Sarcoma UK
Ms Sandra McAllister, Sarcoma MDM Quality Improvement Lead. Consultant Plastic Surgeon. NI
Ms Ioanna Nixon, Clinical Oncologist and Scottish Quality and Safety Fellow, Glasgow
Prof Adrienne Flanagan, Professor Musculoskeletal Pathology, Royal National Orthopaedic Hospital/ UCL
Dr Beatrice Seddon, Consultant Clinical Oncologist, UCL
Abby Mccarthy, Orthopaedic Oncology Physiotherapist, Royal National Orthopaedic Hospital
Dr Richard Roope, Primary Care Adviser for Cancer Research UK and the Clinical Adviser for cancer for the RCGP
Hanny Anwar and David Baxter
James Lind Alliance Adviser and Chair of the Steering Group:
Maryrose Tarpey, JLA
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.
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 had spinal sarcoma
- family members and carers of people who have had spinal sarcoma
- health care professionals - with experience of spinal sarcoma.
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 spinal sarcoma PSP.
We recognise that patient and clinician collaboration is a vital component of a PSP and have planned patient involvement from the outset. We have direct access through our clinical centres to patients with Spinal Sarcoma. In addition, the leading Sarcoma patient charities in the UK, Sarcoma UK and Bone Cancer Research Trust, support us and have agreed to use their existing patient networks to ensure broad participation.
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.
We are aware that many sarcoma patients are young people and recognise the need to communicate via multiple different modalities, including direct communication, the telephone, post, and electronic communication, to guarantee equality of access. We will use multilingual communications, where appropriate.
Step 3: Identifying evidence uncertainties
The spinal sarcoma PSP will carry out a consultation to gather uncertainties from patients, carers and clinicians. A period of one year will be given to complete this exercise (which may be revised by the Steering Group if required).
Existing sources of evidence uncertainties will be searched. In addition to the traditional search for systematic reviews and meta-analysis, this PSP will need to consider expert opinion and current best practice guidelines because of the small number of cases of spinal sarcoma. As far as we know, in the past ten years, there have been five meta-analyses and two systematic reviews in the spinal sarcoma field. These have focused on prognostication, staging of the disease using genetic markers and treatment strategies. There has not yet been a Cochrane review, and we hope that as part of this PSP working with Robin Grant, the Cochrane Group will include spinal sarcoma in an “Orphan Tumours” review.
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 the information specialist 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 the information specialist. 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 the information specialist. 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 spinal sarcoma. 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.
- 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 18 - 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.
- 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 Hanny Anwar and David Baxter.
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 Spinal Sarcoma PSP Steering Group agreed the content and direction of this Protocol on 17 November 2021.