Pulmonary Hypertension (Canada) Protocol
Purpose of the PSP and background
The purpose of this protocol is to clearly set out the aims, objectives and commitments of the Pulmonary Hypertension Priority Setting Partnership (PSP) in line with James Lind Alliance (JLA) principles and the basic roles and responsibilities of the partners. 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.
Our collaboration began in the context of the recently launched Canadian Pulmonary Hypertension Registry (CPHR) and its aspiration to meaningfully engage patients and their representatives to identify and prioritize new, patient-relevant research questions. Using a validated approach to prioritization, we will identify the Top 10 uncertainties for future Pulmonary Hypertension research.
Pulmonary Hypertension (PH) is defined by high blood pressure in the lungs due to narrowing, obstruction, and dysfunction of the pulmonary arteries . While considered a rare disease, thousands of Canadians are affected by PH and the prevalence is increasing . When untreated, PH patients only live 2.5 years, on average . Despite advances and development of effective treatments, approximately 10% of patients still die every year [4-6], with an annual risk of death that is 7-13 times higher than the general Canadian population . In addition to being life threatening, PH is a progressive, disabling condition that affects all age groups and has devastating implications for patients and families . There are considerable economic and social consequences of living with PH [8, 9]. Quality of life is reduced in PH to a similar extent as in severe chronic obstructive pulmonary disease, end-stage kidney failure on dialysis and comparable to that of patients with advanced cancer.
PH is highly clinically relevant given the inordinately increased risks of morbidity and mortality, as well as engendering significant health care and human costs. In contrast to other chronic diseases, the specific research priorities according to PH patients and other stakeholders have not been defined. Several priority workshops and statements for future PH research have been published, none of which involved patients or their representatives during the process. Similarly, PH research has largely been driven by pharmaceutical companies and individual researchers. PH is underrepresented in terms of its public awareness profile compared to other cardiovascular and respiratory diseases and is also under-represented in terms of public research funding. Patient engagement and incorporating their perspectives in clinical research design and outcome measures were emphasized in the recent 2018 World Symposium on Pulmonary Hypertension . Identification of research uncertainties and patient priorities during this project will influence the research agenda in Canada and around the world for years to come by defining the most important and impactful health care gaps for our community to tackle. We will also establish sustainable capacity for future patient-oriented research as well as patient & caregiver input in the design and conduct of clinical research within the CPHR and within broader, multinational initiatives.
We expect that many of the identified priorities will be able to be studied directly within the CPHR framework and infrastructure.
The rigorously identified uncertainties resulting from this project will form the foundation for grant applications and catalyze projects that can be addressed within the existing CPHR infrastructure or elsewhere. This project will influence the national and international PH research agenda. Additionally, we will foster new partnerships with patients within the CPHR Steering Committee and establish new capacity for patient-oriented research in Canada in the PH field.
Aims, objectives and scope of the PSP
The aim of the Pulmonary Hypertension PSP is to identify the most important unanswered questions and uncertainties about the diagnosis, treatment, and prognosis of pulmonary hypertension from the perspective of patients, caregivers, as well as policy-makers and clinicians involved in pulmonary hypertension care 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 engage patients, caregivers, and clinicians in identifying uncertainties about the diagnosis, treatment and prognosis of PH.
- to agree by consensus and obtain a prioritized list of 10 uncertainties for future PH research.
- to disseminate the results of this project and its process.
- to measure the impact and value of this initiative to patients & caregivers, clinicians, researchers.
- to take the results to research commissioning bodies to be considered for funding. OR
- to share the results with the Canadian Institutes of Health Research and other researchers so that these priorities will be considered when undertaking research projects in the future.
The scope of the Pulmonary Hypertension PSP is defined as:
- diagnosis, treatment and prognosis of pulmonary hypertension
- causes, risk factors and aggravating factors of pulmonary hypertension
- management of the co-existing conditions with pulmonary hypertension
- physical or emotional impact of pulmonary hypertension
- public (patient and carer) responses inclusive of all provinces in Canada
- clinician responses inclusive of all provinces in Canada to ensure diversity of responses and an adequate sample size
The PSP will exclude from its scope questions about:
- paediatric pulmonary hypertension
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 Pulmonary Hypertension PSP will be led and managed by a Steering Group involving the following:
Patient and carer representative/s:
Nicole Dempsey, Pulmonary Hypertension patient – Pulmonary Hypertension Association of Canada (PHAC) board member
Jennifer Howard, Pulmonary Hypertension patient from Calgary
Jason Weatherald, Pulmonologist, University of Calgary
Steeve Provencher, Pulmonologist, Université Laval
Lisa Mielniczuk, Cardiologist, University of Ottawa
John Swiston, Pulmonologist, University of British Columbia
Lena Legkaia, Vancouver General Hospital
Hina Iqbal, University of Calgary
James Lind Alliance Adviser and Chair of the Steering Group:
Tamara Rader, JLA
The Steering Group will agree on 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 pulmonary hypertension
- carers of people who have had pulmonary hypertension
- patient advocacy groups (PHAC and Fondation Hypertension Artérielle Pulmonaire Québec [HTAPQ]).
- health system administrators (Dr. Sid Viner, Alberta Health Services) and health care professionals (Gail Nicholson, RN, Pulmonary
- Hypertension Professionals Network) – both with experience in pulmonary hypertension.
Individuals and organizations wishing to participate in the PSP will be required to demonstrate their commitment to the aims and values of the process. The underlying principles of the project are:
- transparency of process
- balanced inclusion of patient, family member, and health care provider interests and perspectives
- exclusion of non-clinician researchers for voting purposes (but they may be involved in other aspects of the process)
- exclusion of groups/organizations that have significant competing interests (e.g. pharmaceutical companies and device manufacturers)
- an audit trail of the original submitted uncertainties to the final prioritized list
- recognition that making priority decisions does not create new knowledge, but reviews existing evidence of uncertainty.
Details about the commitment procedure can be found at: www.lindalliance.org.
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 Pulmonary Hypertension PSP and invited to participate. This will include groundwork with PHAC and HTAPQ to prepare to broadly engage patients across Canada.
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. This will be done via the PHAC and HTAPQ websites, social media accounts and within PH specialty clinics across the country. The Steering Group will 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 Pulmonary Hypertension PSP will carry out a consultation to gather uncertainties from patients, carers and clinicians. A period of 4 months will be given to complete this exercise (which may be revised by the Steering Group if required).
The Pulmonary Hypertension PSP recognises that the following groups may require additional consideration. In order to include indigenous groups, we will attempt trips to at least 3 tribal communities across Canada in order to discuss the partnership and determine interest in participation.
The Steering Group will use the following methods to reach the target groups
- online surveys
- email invitations to complete the online survey
- outpatient PH clinics (Vancouver, Calgary, London, Ottawa and Quebec City) to complete the online survey
The key deliverables will be the development, piloting and distribution of an online survey. The initial survey will propose an open-ended question and ask for the basic demographic characteristics of the survey participants in English and French. We will pilot the surveys by administering them to 20 patients in the Calgary and Quebec City PH clinics in English and French, respectively to ensure the purpose and questions are clear and easily understood. Each respondent can propose up to 10 questions or uncertainties. We will use a secure REDCap platform based at the University of Calgary to capture and house the online survey data. A link to the online survey will be distributed on the PHA Canada (www.phacanada.ca) and HTAPQ (www.htapquebec.ca) websites and via e-mail invitations to their members. We will also approach consecutive patients and their families/caregivers in five (5) outpatient PH clinics in Vancouver, Calgary, London, Ottawa, and Quebec City to complete the online survey using iPads over a 4-month period. Other clinics across Canada may also administer paper surveys and send them by post to the coordinating centre. Most PH patients are followed up every 3-4 months in clinic, which will help ensure the survey can be proposed to the majority of the patients in these clinics.
The background information (to be included in the survey form) as well as the wording of the survey items will be designed to be clear and easy to understand by all participants. In addition, the following identifiable demographic data will be requested in the survey: age, gender, geographic location (province), ethnic group, and whether the participant is a patient, caregiver or family member, health professional, administrator or professional in a health care system organization or a member of a partner organization that represents people with pulmonary hypertension. These data will be collected in an effort to determine whether the perspectives of our stakeholder groups of interest have been successfully captured, and to study whether research priorities are influenced by these demographic factors.
Existing sources of evidence uncertainties may also be searched. A literature search and review will be performed to identify additional gaps, uncertainties and research priorities identified in current national and international PH guidelines.
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 Steering Group in collaboration with 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 in discussion with the Steering Group. Out-of-scope and ‘answered’ submissions will be compiled and tracked 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 members of the Steering Group. 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 Steering Group. 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 Pulmonary Hypertension. 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 on the method and consider how best to reach and engage patients, carers and clinicians in the process. The list of indicative questions will be reduced to a smaller list by piloting an interim prioritization survey with a small focus group of patients, carers and other stakeholders in Calgary to ensure the instructions and process are clear. This survey will be disseminated again to clinicians and PHA Canada/HTAPQ networks and respondents from the initial survey who provided an email address. Questions on this interim online prioritization survey ask respondents to choose their 10 most important questions and rank them on a scale of 1 (not important) to 10 (most important). The rankings will be combined to obtain the Top 20-30 uncertainties. These will then be verified as true uncertainties using a rapid review methodology employed in a previous priority setting partnership for depression. This process consists of a PubMed (MEDLINE) search filtering the available evidence based on hierarchies of evidence and relevance of the study design to the research question. The search results will be limited to systematic reviews, guidelines, and randomized controlled trials. Abstracts will be screened by the research assistant to remove out-of-scope items and duplicates. Individual articles will then be reviewed in detail by clinicians on the Steering Group to confirm if each potential Top 20-30 uncertainty has not been resolved. This process will involve multiple Steering Group members and assistance from Alberta SPOR Support Unit. Lastly, www.clinicaltrials.gov will be searched to determine if these uncertainties are already being addressed in ongoing clinical studies. The reduced list of most highly ranked questions (around 20-30) 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.
Patients, caregivers, and clinicians/researchers will be invited to participate in a full-day workshop in Calgary, Alberta. Workshop participants will be selected by the Steering Group in order to ensure a broad representation in terms of sex/gender, ethnicity, geographic/provincial location, and educational background. Aboriginal patient or caregiver representation in the workshop will be ensured. Invitations for the workshop will be extended at least 3 months before the date to allow adequate preparation time and to arrange travel. Reminders will be sent to participants along with key documents, including an overview of the Workshop, itinerary, and the short list from the interim prioritisation. The James Lind Alliance guidebook recommends between 12-30 participants for the priority setting workshop. Our workshop will include 26 participants, with an additional 3 facilitators, 3 Steering Group members and an information specialist to record data during the workshop. Our 26 participants will be comprised of 13 patients or caregivers and 13 clinicians/ researchers/ policymakers identified through the initial survey. One patient/caregiver and one clinician/researcher/policymaker will represent each of the 13 Canadian PH centres. To encourage additional participation of patients/caregivers who live in remote settings and who may be unable to travel because of their underlying medical condition (e.g. some patients with PH cannot fly due to high oxygen requirements) or logistically difficult travel (e.g. patients who live in Northern or remote settings), the possibility of virtual participation for up to 3 individuals will be included using the Skype for Business application. Clinicians may be a PH physician, PH nurse, or other health care professional from each PH centre. The aim is to include a similar number of patients and caregivers given the enormous impact of PH on carers and family members.
Small group sessions (Two groups with 8 and one group with 9 participants, with balanced mixes of patients, caregivers and clinicians) will discuss the top 20-30 uncertainties from the interim prioritisation list and rank them. Three (3) Steering Group members will attend in non-participatory roles to observe each group discussion. Three (3) facilitators certified in the James Lind Alliance process will be present to guide the workshop and each small group. After the small group sessions, a whole group review will enter each small group’s ranking into a spreadsheet and a combined ranking will be obtained. A second small group session with 3 new groups of 10 follows, with discussion and ranking of the combined rank list. Then another whole group review will occur in order to reach a consensus Top 10 list.
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. Jason Weatherald.
For post-grant Knowledge Translation (KT) activities, a KT report will be prepared and publicized with the Top 10 List on the PHA Canada and HTAPQ websites and in their newsletters. The KT platform from the Alberta SPOR Support Unit has also committed to assist in this phase. The proceedings of this PSP will be submitted as a manuscript to a peer-reviewed journal in the cardiovascular or respiratory medicine field (target journals include the American Journal of Respiratory and Critical Care Medicine, Thorax, and CHEST). Drs. Mehta, Provencher, and Weatherald will engage and facilitate KT through the Canadian Thoracic Society. Dr. Mielniczuk is an executive council member for the Canadian Cardiovascular Society who will facilitate KT through them. Dr. Viner’s role and experience in health systems administration will help translate and implement our findings within the largest single health care system in Canada (Alberta Health Services).
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 Pulmonary Hypertension PSP Steering Group agreed the content and direction of this Protocol on March 27, 2020.
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