Cavernoma PSP protocol

Contents

Purpose

The purpose of this protocol is to set out the aims, objectives and commitments of the Cavernoma Priority Setting Partnership (PSP) and the basic roles and responsibilities of the partners therein.

The Steering Group

The Cavernoma PSP will be led and managed by the following:

Patient representative/s:

  • Dr Ian Stuart (Patient and Founder and Co-ordinator, Cavernoma Alliance UK [CAUK])
  • Dr David White (Carer and Chair of the Trustees of CAUK)
  • Simon Temple (Patient)
  • Kirsten Macpherson / Paula Wheeler (mothers of young sons with cavernoma)

Clinical representative/s:

  • Professor Rustam Al-Shahi Salman (Neurologist)
  • Mr Neil Kitchen (Neurosurgeon)
  • Dr Jenny Thomson (Clinical Geneticist)
  • Dr Vijeya Ganesan (Paediatric neurologist)
  • Mr Connor Malluci (Paediatric neurosurgeon)

Other support organisations:

Angela Collett (Brain & Spine Foundation)

 

The partnership and the priority setting process will be supported and guided by:

David Crowe (The James Lind Alliance [JLA])

Robin Harbour (Information Specialist)

Francesca Howarth (Adminsitrator) 

The Steering Group includes representation of patient/carer groups, clinicians and another Support Organisation (Brain & Spine Foundation).

The Steering Group will agree the resources, including time and expertise that they will be able to contribute to each stage of the process. The JLA will advise on this.

Background to the Cavernoma PSP

The JLA is an organisation which is overseen by the National Institute for Health Research Evaluation, Trials and Studies Coordinating Centre (NETSCC). Its aim is to provide an infrastructure and process to help patients and clinicians work together to agree which are the most important treatment uncertainties affecting their particular interest, in order to influence the prioritisation of future research in that area. The JLA defines an uncertainty as a “known unknown” – in this case relating to the effects of treatment.

Cavernomas are clusters of abnormal, leaky blood vessels that are found in many places in the body. However, the Cavernoma PSP is concerned only with cavernoma found in the brain, also known as cerebral cavernous malformations (CCMs), and spinal cord (the central nervous system) and which may cause a range of neurological symptoms including stroke and epileptic seizure(s). People with CCM present to medical attention at any age, but most frequently during their fourth decade of life with these problems, leaving people affected by their consequences and the risk of their recurrence. Asymptomatic CCMs affect about 1 in 625 people. New CCM diagnoses are made in about 3 in every 500,000 adults each year.

The PSP came together through initial discussions between Dr Ian Stuart, Professor Rustam Al-Shahi Salman and Mr Neil Kitchen following research sponsored by CAUK and Genetic Alliance UK to describe the evidence-base for treatment and management of cavernoma. This was published as “Guidelines for the management of cerebral cavernous malformations in adults”  [1] http://www.cavernoma.org.uk/opus473/final_CCM_guidelines_2.pdf. The conclusion was that the evidence base was very weak, and determining the appropriate research questions via a PSP would be the natural next step.

Definitions

For the purposes of this PSP, we have used the following definitions of certain terms:

Diagnosis: The recognition or identification of a health condition based on symptoms, signs or test results.

Prognosis: The predicted progression and outcome of a condition in an individual.

Treatment: Any preventive, therapeutic, rehabilitative or palliative action intended to improve health or wellbeing for individuals or in communities

Care: The holistic and organised provision for the maintenance and improvement of physical and mental health.

Management: For the purposes of this PSP, ‘management’ is taken to embrace everything to do with cavernoma from first diagnosis through to final outcome

Aims and objectives of the Cavernoma PSP 

The aim of the Cavernoma PSP is to identify the unanswered questions about the management of brain and spine cavernomas affecting adults, children and babies, from patient, carer, family and clinical perspectives and then prioritise these treatment uncertainties.

The objectives of the Cavernoma PSP are to:

  • work with patients, carers, clinicians and others to identify uncertainties about the effects of cavernoma diagnosis, prognosis, treatments and care,
  • 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.

Partners

Organisations and individuals will be invited to take part in the PSP, representing the following groups:

  • people who have or have had personal experience of cavernoma;
  • carers of people who have had cavernoma;
  • medical doctors, nurses and professionals allied to medicine with clinical experience of cavernoma.

The Steering Group will attempt to invite all organisations that can reach and advocate for these groups to become involved in the PSP. The JLA will take responsibility for ensuring the various stakeholder groups are able to participate equally to the process.

Organisations wishing to participate in the PSP will be required to affiliate to the JLA in order to demonstrate their commitment to the aims and values of the JLA. 

Exclusion Criteria

Some individuals and organisations may be judged by the JLA or the Steering Group to have conflicts of interest. These may be perceived to adversely affect those organisations’ views, causing unacceptable bias. As this is likely to affect the ultimate findings of the PSP, those individuals and 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.

Methods

This section describes a schedule of proposed stages through which the PSP aims to fulfil its objectives. The process is iterative and dependent on the active participation and contribution of different groups. The methods adopted in any stage will be agreed through consultation between the partners, guided by the PSP’s aims and objectives. More details and examples can be found at www.jla.nihr.ac.uk 

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 and through the JLA’s existing register of affiliates. Potential partners will be contacted and informed of the establishment and aims of the Cavernoma PSP and invited to attend and participate in an initial stakeholder meeting.

Step 2: Initial Steering Group meeting

The first Steering Group meeting will have several key objectives:

  • to welcome and introduce potential members of the Cavernoma PSP;
  • to present the proposed plan for the PSP;
  • to initiate discussion, answer questions and address concerns;
  • to identify those potential partner organisations which will commit to the PSP and identify individuals who will be those organisations’ representatives and the PSP’s principal contacts;
  • to establish principles upon which an open, inclusive and transparent mechanism can be based for contributing to, reporting and recording the work and progress of the PSP.

Following the meeting, individuals and organisations which have decided to participate in the PSP will be asked to complete a declaration of interests, including disclosing relationships with the pharmaceutical industry. 

Step 3: Identifying treatment uncertainties

Each partner will identify a method for soliciting from its members questions and uncertainties of practical clinical importance relating to the treatment and management of Cavernoma. A period of around 6-8 weeks will be given to complete this exercise.

The methods may be designed according to the nature and membership of each organisation, but must be as transparent, inclusive and representative as practicable. Methods may include membership meetings, email consultation, postal or web-based questionnaires, internet message boards and focus group work.

The Steering Group will decide how to judge the representativeness of the sample and whether measures are required to adjust for bias in the sample. Such measures may include additional efforts to reach under-represented groups, giving greater weight to uncertainties suggested by under-represented groups, or identifying groups using organised campaigns to lobby for a particular treatment uncertainty.

Existing sources of information about treatment uncertainties for patients 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 (in England) or the Knowledge Network (http://www.knowledge.scot.nhs.uk/home.aspx) in Scotland. 

Step 4: Refining questions and uncertainties

The process of managing the data will be undertaken by Robin Harbour – the JLA can advise on the amount of time likely to be required for its execution. The JLA will participate in this process as an observer, to ensure accountability and transparency.

The consultation process will produce “raw” unanswered questions about diagnosis and the effects of treatments. These raw questions will be assembled and categorised and refined by Robin Harbour into “collated indicative questions” which are clear, addressable by research and understandable to all. Similar or duplicate questions will be combined where appropriate.

The existing literature will be researched by Robin Harbour to see to what extent these refined questions have, or have not, been answered by previous research. Where no systematic review is available, primary studies will be identified (including clinical trials, cohort and case-control studies, or n of 1 designs as appropriate). Where out-of-date systematic reviews are identified, searches for primary studies will be undertaken to establish the value of an updating exercise. Searches of existing research will focus on sources that are most likely to identify relevant material. As a minimum, searches will cover the Cochrane Database of Systematic Reviews, the Cochrane Controlled Trials Register, Embase, Medline, CINAHL, and Psychinfo. Relevant guidelines produced by NICE or SIGN will be checked for uncertainties, along with any other relevant guidelines identified through searches of NHS Evidence (see above) and the National Guidelines Clearinghouse (http://www.guideline.gov/). Additional resources may need to be checked in relation to patient, carer, or family perspectives.

Sometimes, uncertainties are expressed that can in fact be resolved with reference to existing research evidence, i.e. they are "unrecognised knowns" and not uncertainties. If a question about treatment effects can be answered with existing information but this is not known, it suggests that information is not being communicated effectively to those who need it. CAUK will keep a record of these 'answerable questions' and deal with them separately from the 'true uncertainties' considered during the research priority setting process.

Uncertainties which are not adequately addressed by previous research will be collated and prepared for entry into a cavernoma section within the UK Database of Uncertainties about the Effects of Treatments (UK DUETs - www.library.nhs.uk/duets) by Robin Harbour. This will ensure that the uncertainties have been actually checked to be uncertainties. This is the responsibility of the Steering Group, which will need to have agreed personnel and resources to carry this accountability. The data should be entered into UK DUETs on completion of the priority setting exercise, in order to ensure any updates or changes to the data have been incorporated beforehand.

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 relating to the management of cavernoma. This will be carried out by members of the Steering Group and the wider partnership that represents patients, carers and clinicians.

The interim stage, to proceed from a long list of uncertainties to a shorter list (e.g. up to 20), will be carried out over email, whereby organisations consult their membership and ask for a top 15-20 most important uncertainties, ranked or unranked.

The final stage, to reach, for example, 10 prioritised uncertainties, will be conducted in a face-to-face meeting, using group discussions and plenary sessions.

The methods used for this prioritisation process will be determined by consultation with the partner organisations and with the advice of the JLA. Methods which have been identified as potentially useful in this process include: adapted Delphi techniques; expert panels or nominal group techniques; consensus development conference; electronic nominal group and online voting; interactive research agenda setting and focus groups.

The JLA will facilitate this process and ensure transparency, accountability and fairness.

Findings and Research

It is anticipated that the findings of the Cavernoma PSP will be reported to funding and research agenda setting organisations such as the NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC), which includes the HTA Programme, and the MRC, as well as the major research funding charities. Steering Group members and partners are encouraged to develop the prioritised uncertainties into research questions, and to work to establish the research needs of those unanswered questions to use when approaching potential funders, or when allocating funding for research themselves, if applicable.

Steering Group members may comment on the ideal research design required to answer the prioritised uncertainties in the light of the quality of evidence already available, the frequency of the problem, the size of the likely effect of a treatment, and the feasibility of a particular research design.

Publicity

As well as alerting funders, partners and Steering Group members are expected to publish the findings of the Cavernoma PSP using both internal and external communication mechanisms. The JLA may also capture and publicise the results, through descriptive reports of the process itself. This exercise will be distinct from the production of an academic paper. Publication of final results will take precedence over publication of any academic papers.

Signed by the Steering Group

The undersigned agree to follow the Cavernoma Priority Setting Protocol. 

 

[Dr Ian Stuart, CAUK]

 

…………………………………………………………  Date: …………………………

 

[Dr David White, CAUK]

 

…………………………………………………………  Date: …………………………

 

[Simon Temple, CAUK]

 

…………………………………………………………  Date: …………………………

 

[Paula Wheeler, CAUK]

 

…………………………………………………………  Date: …………………………

 

[Angela Collett, Brain & Spine Foundation]

 

…………………………………………………………  Date: …………………………

 

[Professor Rustam Al-Shahi Salman, Centre for Clinical Brain Sciences, University of Edinburgh]

 

…………………………………………………………  Date: …………………………

 

[Mr Neil Kitchen, National Hospital for Neurology and Neurosurgery]

 

…………………………………………………………  Date: …………………………

 

[Dr Jennifer Thomson, Leeds Teaching Hospitals]

 

…………………………………………………………  Date: …………………………

 

 [Dr Vijeya Ganesan, Great Ormond Street Hospital]

 

…………………………………………………………  Date: …………………………

 

[Mr Connor Malluci, Alder Hey Children’s Hospital]

 

…………………………………………………………  Date: …………………………

 

 

 [Robin Harbour, Independent Consultant]

 

…………………………………………………………  Date: …………………………

 

[Francesca Howarth, Independent Administrator]

 

…………………………………………………………  Date: …………………………

 

 [David Crowe, The James Lind Alliance]

 

…………………………………………………………  Date: …………………………