Matching researchers' needs and patients' contributions: practical tips for meaningful patient engagement from the field of rheumatology.

There is an increasing recognition of the importance of patient engagement and involvement in health research, specifically within the field of rheumatology. In general, researchers in this specialty appreciate the value of patients as partners in research. In practice, however, the majority of researchers does not involve patients on their research teams. Many researchers find it difficult to match their needs for patient engagement and the potential contributions from individuals living with rheumatic disease. In this Viewpoint, we provide researchers and patients practical tips for matching 'supply and demand,' based on our own experiences as patient engagement consultants and trainers in rheumatology research. All authors started as a 'naïve' patient or caregiver, an identity that evolved through a process of 'adversarial growth': positive changes that are experienced as a result of the struggle with highly challenging life circumstances. Here, we introduce four stages of adversarial growth in the context of research. We submit that all types of patients have their own experiences, qualities and skills, and can add specific input to research. The recommendations for engagement are not strict directives. They are meant as starting points for discussion or interview. Regardless of individual qualities and knowledge, we believe that all patients engaged in research have a single goal in common: to contribute to research that ultimately will change the lives of many other patients.

Research agenda setting with children with juvenile idiopathic arthritis: Lessons learned.

AIM: The aim of this qualitative study is to understand the research priorities of Dutch children with juvenile idiopathic arthritis (JIA) as well as researching how children can be involved. BACKGROUND: Several health research agendas have successfully been developed with adults but rarely with children. Children are still seldom recognized as possessing credible knowledge about their own body and life. This research project with focus group discussions and interviews with children with juvenile idiopathic arthritis (JIA) was an innovative addition to a nationwide prioritization of research questions of patients with JIA, their carers and health care professionals, based on the James Lind Alliance (JLA) methodology. RESULTS: Children with JIA appreciated being invited to give their opinion on JIA research prioritization as knowledgeable actors. They have clear views on what topics need most attention. They want more insight on how to medically and socially treat JIA so that they can better fulfil their aspirations at school, later in work and with their relationships. CONCLUSION: We have identified the Top 5 research priorities for children with JIA. Most priorities are unique and differ from the priorities of the adolescents and young adults, parents and healthcare professionals in the main JLA priority setting exercise. Ultimately, two of the children's priorities were included in the final JLA Top 10.

Burden of comorbid conditions in children and young people with juvenile idiopathic arthritis: a collaborative analysis of 3 JIA registries.

OBJECTIVES: Burden of comorbidities are largely unknown in JIA. From 2000, national and international patient registries were established to monitor biologic treatment, disease activity and adverse events in patients with JIA. The aim of this analysis was to investigate in parallel, for the first time, three of the largest JIA registries in Europe/internationally-UK JIA Biologic Registers (BCRD/BSPAR-ETN), German biologic registers (BiKeR/JuMBO), multinational Pharmachild-to quantify the occurrence of selected comorbidities in patients with JIA. METHODS: Information on which data the registers collect were compared. Patient characteristics and levels of comorbidity were presented, focussing on four key conditions: uveitis, MAS, varicella, and history of tuberculosis. Incidence rates of these on MTX/biologic therapy were determined. RESULTS: 8066 patients were registered into the three JIA registers with similar history of the four comorbidities across the studies; however, varicella vaccination coverage was higher in Germany (56%) vs UK/Pharmachild (16%/13%). At final follow-up, prevalence of varicella infection was lower in Germany (15%) vs UK/Pharmachild (37%/50%). Prevalence of TB (0.1-1.8%) and uveitis (15-19%) was similar across all registers. The proportion of systemic-JIA patients who ever had MAS was lower in Germany (6%) vs UK (15%) and Pharmachild (17%). CONCLUSION: This analysis is the first and largest to investigate the occurrence of four important comorbidities in three JIA registries in Europe and the role of anti-rheumatic drugs. Combined, these three registries represent one of the biggest collection of cases of JIA worldwide and offer a unique setting for future JIA outcome studies.

Dutch patients, caregivers and healthcare professionals generate first nationwide research agenda for juvenile idiopathic arthritis.

BACKGROUND: Involving the end-users of scientific research (patients, carers and clinicians) in setting research priorities is important to formulate research questions that truly make a difference and are in tune with the needs of patients. We therefore aimed to generate a national research agenda for Juvenile Idiopathic Arthritis (JIA) together with patients, their caregivers and healthcare professionals through conducting a nationwide survey among these stakeholders. METHODS: The James Lind Alliance method was used, tailored with additional focus groups held to involve younger patients. First, research questions were gathered through an online and hardcopy survey. The received questions that were in scope were summarised and a literature search was performed to verify that questions were unanswered. Questions were ranked in the interim survey, and the final top 10 was chosen during a prioritisation workshop. RESULTS: Two hundred and seventy-eight respondents submitted 604 questions, of which 519 were in scope. Of these 604 questions, 81 were generated in the focus groups with younger children. The questions were summarised into 53 summary questions. An evidence checking process verified that all questions were unanswered. A total of 303 respondents prioritised the questions in the interim survey. Focus groups with children generated a top 5 of their most important questions. Combining this top 5 with the top 10s of patients, carers, and clinicians led to a top 21. Out of these, the top 10 research priorities were chosen during a final workshop. Research into pain and fatigue, personalised treatment strategies and aetiology were ranked high in the Top 10. CONCLUSIONS: Through this study, the top 10 research priorities for JIA of patients, their caregivers and clinicians were identified to inform researchers and research funders of the research topics that matter most to them. The top priority involves the treatment and mechanisms behind persisting pain and fatigue when the disease is in remission.

Four normative perspectives on public health policy-making and their preferences for bodies of evidence.

Calls for evidence-informed public health policy-making often ignore that there are multiple, and often competing, bodies of potentially relevant evidence to which policy-makers have recourse in identifying policy priorities and taking decisions. In this paper, we illustrate how policy frames may favour the use of specific bodies of evidence. For the sixth Dutch Public Health Status and Foresight report (2014), possible future trends in population health and healthcare expenditure were used as a starting point for a deliberative dialogue with stakeholders to identify and formulate the most important societal challenges for the Dutch health system. Working with these stakeholders, we expanded these societal challenges into four normative perspectives on public health. These perspectives can be regarded as policy frames. In each of the perspectives, a specific body of evidence is favoured and other types of evidence are neglected. Crucial outcomes in one body may be regarded as irrelevant from other perspectives. Consequently, the results of research from a single body of evidence may not be helpful in the policy-making processes because policy-makers need to account for trade-offs between all competing interests and values. To support these policy processes, researchers need to combine qualitative and quantitative methodologies to address different outcomes from the start of their studies. We feel it is time for the research community to re-politicise the idea of evidence use and for policy-makers to demand research that helps them to account for all health-related policy goals. This is a prerequisite for real evidence-informed policy-making.

Treating juvenile idiopathic arthritis to target: what is the optimal target definition to reach all goals?

In 2018, an international Task Force formulated recommendations for treating Juvenile Idiopathic Arthritis (JIA) to target. The Task Force has not yet resolved three issues. The first issue is the lack of a single "best" target. The Task Force decided not to recommend the use of a specific instrument to assess inactive disease or remission. Recent studies underscore the use of a broad target definition. The second issue is the basic assumption that a treatment aggressively aimed at the target will have 'domino effects' on other treatment goals as well. Thus far, this assumption was not confirmed for pain, fatigue and stiffness. The third issue is shared decision-making, and the role of individual patient targets. Nowadays, patients and parents should have a more active role in choosing targets and their personal treatment goals. In our department the electronic medical records have been restructured in such a way that the patient's personal treatment goals with a target date appears on the front page. The visualization of their specific personal goals helps us to have meaningful discussions on the individualized treatment strategy and to share decisions. In conclusion, a joint treat to target (T2T) strategy is a promising approach for JIA. The Task Force formulated valuable overarching principles and a first version of recommendations. However, implementation of T2T needs to capture more than just inactive disease. Patients and parents should have an active role in choosing personal targets as well.

The European network for care of children with paediatric rheumatic diseases: care across borders.

OBJECTIVES: To provide an overview of the paediatric rheumatology (PR) services in Europe, describe current delivery of care and training, set standards for care, identify unmet needs and inform future specialist service provision. METHODS: An online survey was developed and presented to national coordinating centres of the Paediatric Rheumatology International Trials Organisation (PRINTO) (country survey) and to individual PR centres (centre and disease surveys) as a part of the European Union (EU) Single Hub and Access point for paediatric Rheumatology in Europe project. The survey contained components covering the organization of PR care, composition of teams, education, health care and research facilities and assessment of needs. RESULTS: Response rates were 29/35 (83%) for country surveys and 164/288 (57%) for centre surveys. Across the EU, approximately one paediatric rheumatologist is available per million population. In all EU member states there is good access to specialist care and medications, although biologic drug availability is worse in Eastern European countries. PR education is widely available for physicians but is insufficient for allied health professionals. The ability to participate in clinical trials is generally high. Important gaps were identified, including lack of standardized clinical guidelines/recommendations and insufficient adolescent transition management planning. CONCLUSION: This study provides a comprehensive description of current specialist PR service provision across Europe and did not reveal any major differences between EU member states. Rarity, chronicity and complexity of diseases are major challenges to PR care. Future work should facilitate the development, dissemination and implementation of standards of care, treatment and service recommendations to further improve patient-centred health care across Europe.

The Public Health Status and Foresight report 2014: Four normative perspectives on a healthier Netherlands in 2040.

Policy-oriented foresight reports aim to inform and advise decision-makers. In value-laden areas such as public health and healthcare, deliberative scenario methods are clearly needed. For the sixth Dutch Public Health Status and Forecasts-report (PHSF-2014), a new approach of co-creation was developed aiming to incorporate different societal norms and values in the description of possible future developments. The major future trends in the Netherlands were used as a starting point for a deliberative dialogue with stakeholders to identify the most important societal challenges for public health and healthcare. Four societal challenges were identified: 1) To keep people healthy as long as possible and cure illness promptly, 2) To support vulnerable people and enable social participation, 3) To promote individual autonomy and freedom of choice, and 4) To keep health care affordable. Working with stakeholders, we expanded these societal challenges into four corresponding normative scenarios. In a survey the normative scenarios were found to be recognizable and sufficiently distinctive. We organized meetings with experts to explore how engagement and policy strategies in each scenario would affect the other three societal challenges. Possible synergies and trade-offs between the four scenarios were identified. Public health foresight based on a business-as-usual scenario and normative scenarios is clearly practicable. The process and the outcomes support and elucidate a wide range of strategic discussions in public health.

Dutch juvenile idiopathic arthritis patients, carers and clinicians create a research agenda together following the James Lind Alliance method: a study protocol.

BACKGROUND: Research on Juvenile Idiopathic Arthritis (JIA) should support patients, caregivers/parents (carers) and clinicians to make important decisions in the consulting room and eventually to improve the lives of patients with JIA. Thus far these end-users of JIA-research have rarely been involved in the prioritisation of future research. MAIN BODY: Dutch organisations of patients, carers and clinicians will collaboratively develop a research agenda for JIA, following the James Lind Alliance (JLA) methodology. In a 'Priority Setting Partnership' (PSP), they will gradually establish a top 10 list of the most important unanswered research questions for JIA. In this process the input from clinicians, patients and their carers will be equally valued. Additionally, focus groups will be organised to involve young people with JIA. The involvement of all contributors will be monitored and evaluated. In this manner, the project will contribute to the growing body of literature on how to involve young people in agenda setting in a meaningful way. CONCLUSION: A JIA research agenda established through the JLA method and thus co-created by patients, carers and clinicians will inform researchers and research funders about the most important research questions for JIA. This will lead to research that really matters.

Patient's experiences with the care for juvenile idiopathic arthritis across Europe.

BACKGROUND: To assess the views of juvenile idiopathic arthritis (JIA) patients and their parents on the care and treatment they receive in referral pediatric rheumatology centers throughout Europe. METHODS: In a collaboration between physicians and patient associations, a questionnaire was developed, covering various domains of JIA care, including demographics, diagnosis, referrals to various health care professionals, access to pain and fatigue management and support groups, information they received about the disease and awareness of and participation in research. The questionnaire was translated and distributed by parent associations and pediatric rheumatologists in 25 countries, 22 of which were European. After completion the replies were entered on the PRINTO website. Replies could either be entered directly by parents on the website or on paper. In these cases, the replies were scanned and emailed by local hospital staff to Utrecht where they were entered by I.R. in the database. RESULTS: The survey was completed by 622 parents in 23 countries. The majority (66.7%) of patients were female, with median age 10-11 years at the completion of the questionnaire. Frequencies of self-reported JIA categories corresponded to literature. Some patients had never been referred to the ophthalmologist (22.8%) or physiotherapist (31.7%). Low rates of referral or access to fatigue (3.5%) or pain management teams (10.0%), age appropriate disease education (11.3%), special rehabilitation (13.7%) and support groups (20.1%) were observed. Many patients indicated they did not have contact details for urgent advice (35.9%) and did not receive information about immunizations (43.2%), research (55.6%) existence of transition of care clinics (89,2%) or financial support (89.7%). While on immunosuppressive drugs, about one half of patients did not receive information about immunizations, travelling, possible infections or how to deal with chickenpox or shingles. CONCLUSIONS: Low rates of referral to health care professionals may be due to children whose illness is well managed and who do not need additional support or information. Improvements are needed, especially in the areas of supportive care and information patients receive. It is also important to improve doctor patient communication between visits. Physicians can be instrumental in the setting up of support groups and increasing patients' awareness of existing support. Suggestions are given to convey crucial pieces of information structurally and repeatedly to ensure, among other things, compliance.

[Patient preferences versus evidence-based medicine: did the pioneers of evidence-based medicine take the patient's preferences into account?]. / Patiëntvoorkeur versus evidencebased medicine: Hadden de pioniers van EBM oog voor wat de patiënt wil?

A patient's values and preferences are one of the three 'pillars' of evidence-based medicine (EBM). How can we explain that this one pillar has hardly been elaborated in the EBM-literature?? Were the EBM pioneers really committed to the patient's preferences, were they not ready yet, or were they not committed at all? In key international EBM publications dated between 1985 and 2000, we only found sympathetic, yet vague, statements lacking concrete content. In the Netherlands, a Health Council report set the tone with a sense of fear for 'consumer medicine'. In addition to an overly optimistic view of the past, in 2014 Greenhalgh sketched a vision of the future of EBM in which the sympathetic comments about patient preferences are finally made concrete. The EBM movement has already successfully adapted to social developments in the past; therefore, there is reason for optimism.

[The forgotten capitulation of evidence-based medicine]. / De vergeten capitulatie van evidencebased medicine.

In 1992, the Canadian physician Gordon Guyatt wrote an article that is generally regarded as the starting point of evidence-based medicine (EBM). He described the ideas behind the McMaster residency programme for 'evidence-based practitioners', founded by David Sackett. Eight years later, in 2000, Guyatt concluded that this programme was too ambitious. In a new publication he described most doctors as 'evidence-users'. This editorial marks the transition from an individual to a collective form of EBM, emphasizing the use of evidence-based guidelines. The starting point of this collective form of EBM is not the well-known 1992 paper, but the forgotten editorial in 2000, which was described by Guyatt's colleagues as the capitulation of EBM.

[The future of guidelines: an exploration based on the four perspectives of the PHSF-2014]. / De toekomst van richtlijnen: een verkenning aan de hand van de vier perspectieven van de VTV-2014.

In the Dutch Public Health Status and Foresight report (PHSF), the Dutch National Institute for Public Health and the Environment explores the future of public health and health care in the Netherlands up to 2040. In the first article of a short series on the PHSF 2014, we briefly explained the study and four normative perspectives on public health. In the second article, we described the most important future trends up to 2030. In this, the final article on the PHSF 2014, we use the four normative perspectives as a foresight of future guidelines. We make some recommendations in which normative diversity is taken into account. Our ultimate goal is to see our results and analyses also being put to use in other strategic discussions and determinations.

[A trend scenario and four perspectives for 2040: the Dutch Public Health Status and Forecast 2014]. / Een trendscenario en vier perspectieven voor 2040: de Volksgezondheid Toekomst Verkenning 2014.

In the Dutch Public Health Status and Forecast report (PHSF), the Dutch National Institute for Public Health and the Environment explores the future of public health and health care in the Netherlands until 2040. The PHSF-2014 will consist of four parts: (a) a description of public health in the Netherlands now and in the past; (b) future trends, based on model calculations and expert opinions; (c) future scenarios based on four normative perspectives on public health; and (d) opportunities and dilemmas in the prevention of future problems. In this first article of a short series on the PHSF-2014, we briefly explain these four parts of the study. In the next article, we will describe the most important future trends until 2030. In the final article, we will summarize the conclusions of the PHSF-2014.

[How much should a gained life-year cost? Study on the assessment of a QALY]. / Hoeveel mag een gewonnen levensjaar kosten? Onderzoek naar de waardering van de QALY.

Increasing healthcare costs force policy makers to make difficult choices in the insurance package. In order to make rational choices, there must be an understanding of the healthcare costs as well as the value of the health that the care provides. Health economists have in recent years carried out extensive research into the value that people attribute to health. Health is of great social value, and is difficult to express in concrete monetary terms. One extra life-year in good health ('quality-adjusted life year', QALY) seems to be valued on average at no less than 50,000 euros by people. Methodology needs to be developed in this area so that study results are more uniform and can be better compared. Physicians and policy makers will thus gain more insight into the value of health.