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.

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 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 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.

[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.

[Cost-effectiveness: euros per QALY is not enough]. / Kosteneffectiviteit: euro's per QALY is niet genoeg.

The number of economic evaluations being published in scientific literature each year is increasing exponentially. Cost-effectiveness seems to be playing an increasing role in decisions about reimbursement or the implementation of curative or preventive interventions. A cost-effectiveness ratio represents the fraction between the costs and effects that are associated with an intervention. What costs are included in the numerator of the fraction? What assumptions on effectiveness and reach are made to express the denominator? Various examples have shown that small differences in assumptions can have significant consequences for the cost-effectiveness ratio. This holds true for presumptions on the long-term effects of an intervention as well as the willingness to participate in an intervention. Such assumptions and choices can sometimes highly determine the outcome of a cost-effectiveness analysis. For this reason, caution in interpreting results of cost-effectiveness analyses is warranted.

[Diabetes mellitus in the Netherlands: estimate of the current disease burden and prognosis for 2025]. / Diabetes mellitus in Nederland: schatting van de huidige ziektelast en prognose voor 2025.

OBJECTIVE: To estimate the number of people with diagnosed diabetes mellitus in the Netherlands in 2007 using a new method; to describe trends in the past; to predict the situation in 2025. DESIGN: Model calculations. METHODS: Based on five general practice records (Nijmegen Continuous Morbidity Registration [CMR], Netherlands Information Network of General Practice [LINH], Limburg Family Practice Registration Network [RNH-Limburg], Registration Network University Family Practices, Leiden and its environs [RNUH-LEO], and the transition project) the prevalence and incidence of diagnosed diabetes in the Netherlands in 2007 was estimated. Trends in the prevalence of diagnosed diabetes were estimated from the five records over the period 2000-2007. The prevalence of diagnosed diabetes in 2025 was estimated using the Dutch Chronic Diseases Model, which takes into account demographic developments and a further increase in obesity in the Netherlands in the future. RESULTS: In 2007, 740,000 persons (95% CI: 665,000-824,000) with diabetes were undergoing care. The incidence of new diabetes during 2007 was 71,000 (95% CI: 57,000-90,000). The prevalence of diagnosed diabetes increased by almost 80% in 2000-2007. The model projection resulted in an estimate of 1.3 million people with diagnosed diabetes in 2025, i.e. 8% of the Dutch population. There is a high level of uncertainty about these estimates. CONCLUSION: The increase in the number of diabetes patients in 2025 has consequences for care and will require measures to be taken in coming years in the areas of prevalence and care organisation.