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Background

When selecting samples for patient preference studies, it may be difficult or impractical to recruit participants who are eligible for a particular treatment decision. However, a general public sample may not be an appropriate proxy.

Objective

This study compares preferences for rheumatoid arthritis (RA) preventive treatments between members of the general public and first-degree relatives (FDRs) of confirmed RA patients to assess whether a sample of the general public can be used as a proxy for FDRs.

Methods

Participants were asked to imagine they were experiencing arthralgia and had screening tests indicating a 60% chance of developing RA within 2 yrs. Using a discrete choice experiment, participants were offered a series of choices between no treatment and 2 unlabeled hypothetical treatments to reduce the risk of RA. To assess data quality, time to complete survey sections and comprehension questions were assessed. A random parameter logit model was used to obtain attribute-level estimates, which were used to calculate relative importance, maximum acceptable risk (MAR), and market shares of hypothetical preventive treatments.

Results

The FDR sample (n = 298) spent more time completing the survey and performed better on comprehension questions compared with the general public sample (n = 982). The relative importance ranking was similar between the general public and FDR participant samples; however, other relative preference measures involving weights including MARs and market share differed between groups, with FDRs having numerically higher MARs.

Conclusion

In the context of RA prevention, the general public (average risk) may be a reasonable proxy for a more at-risk sample (FDRs) for overall relative importance ranking but not weights. The rationale for a proxy sample should be clearly justified.

Highlights

•Participants from the general public were compared to first-degree relatives on their preferences for rheumatoid arthritis (RA) preventive treatments using a discrete choice experiment.

•Preferences were similar between groups in terms of the most important and least important attributes of preventive treatments, with effectiveness being the most important attribute. However, relative weights differed.

•Attention to the survey and predicted market shares of hypothetical RA preventive treatments differed between the general public and first-degree relatives.

•The general public may be a reasonable proxy for an at-risk group for patient preferences ranks but not weights in the disease prevention context; however, care should be taken in sample selection for patient preference studies when choosing nonpatients.

Introduction Amidst growing consensus that stakeholder decision-making during drug development should be informed by an understanding of patient preferences, the Innovative Medicines Initiative project 'Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle' (PREFER) is developing evidence-based recommendations about how and when patient preferences should be integrated into the drug life cycle. This protocol describes a PREFER clinical case study which compares two preference elicitation methodologies across several populations and provides information about benefit-risk trade-offs by those at risk of rheumatoid arthritis (RA) for preventive interventions. Methods and analysis This mixed methods study will be conducted in three countries (UK, Germany, Romania) to assess preferences of (1) first-degree relatives (FDRs) of patients with RA and (2) members of the public. Focus groups using nominal group techniques (UK) and ranking surveys (Germany and Romania) will identify and rank key treatment attributes. Focus group transcripts will be analysed thematically using the framework method and average rank orders calculated. These results will inform the treatment attributes to be assessed in a survey including a discrete choice experiment (DCE) and a probabilistic threshold technique (PTT). The survey will also include measures of sociodemographic variables, health literacy, numeracy, illness perceptions and beliefs about medicines. The survey will be administered to (1) 400 FDRs of patients with RA (UK); (2) 100 FDRs of patients with RA (Germany); and (3) 1000 members of the public in each of UK, Germany and Romania. Logit-based approaches will be used to analyse the DCE and imputation and interval regression for the PTT. Ethics and dissemination This study has been approved by the London-Hampstead Research Ethics Committee (19/LO/0407) and the Ethics Committee of the Friedrich-Alexander-Universitat Erlangen-Nurnberg (92_17 B). The protocol has been approved by the PREFER expert review board. The results will be disseminated widely and will inform the PREFER recommendations.

Objective: Patients have unique insights and are (in-)directly affected by each decision taken throughout the life cycle of medicinal products. Patient preference studies (PPS) assess what matters most to patients, how much, and what trade-offs patients are willing to make. IMI PREFER was a six-year European public-private partnership under the Innovative Medicines Initiative that developed recommendations on how to assess and use PPS in medical product decision-making, including in the regulatory evaluation of medicinal products. This paper aims to summarize findings and recommendations from IMI PREFER regarding i) PPS applications in regulatory evaluation, ii) when and how to consult with regulators on PPS, iii) how to reflect PPS in regulatory communication and iv) barriers and open questions for PPS in regulatory decision-making.

Methods: PREFER performed six literature reviews, 143 interviews and eight focus group discussions with regulators, patient representatives, industry representatives, Health Technology Assessment bodies, payers, academics, and clincians between October 2016 and May 2022.

Results: i) With respect to PPS applications, prior to the conduct of clinical trials of medicinal products, PPS could inform regulators’ understanding of patients’ unmet needs and relevant endpoints during horizon scanning activities and scientific advice. During the evaluation of a marketing authorization application, PPS could inform: a) the assessment of whether a product meets an unmet need, b) whether patient-relevant clinical trial endpoints and outcomes were studied, c) the understanding of patient-relevant effect sizes and acceptable trade-offs, and d) the identification of key (un-)favorable effects and uncertainties. ii) With respect to consulting with regulators on PPS, PPS researchers should ideally have early discussions with regulators (e.g., during scientific advice) on the PPS design and research questions. iii) Regarding external PPS communication, PPS could be reflected in the assessment report and product information (e.g., the European Public Assessment Report and the Summary of Product Characteristics). iv) Barriers relevant to the use of PPS in regulatory evaluation include a lack of PPS use cases and demonstrated impact on regulatory decision-making, and need for (financial) incentives, guidance and quality criteria for implementing PPS results in regulatory decision-making. Open questions concerning regulatory PPS use include: a) should a product independent broad approach to the design of PPS be taken and/or a product-specific one, b) who should optimally be financing, designing, conducting, and coordinating PPS, c) when (within and/or outside clinical trials) to perform PPS, and d) how can PPS use best be operationalized in regulatory decisions.

Conclusion: PPS have high potential to inform regulators on key unmet needs, endpoints, benefits, and risks that matter most to patients and their acceptable trade-offs. Regulatory guidelines, templates and checklists, together with incentives are needed to foster structural and transparent PPS submission and evaluation in regulatory decision-making. More PPS case studies should be conducted and submitted for regulatory assessment to enable regulatory discussion and increase regulators’ experience with PPS implementation and communication in regulatory evaluations.

Background Patient preferences (PP), which are investigated in PP studies using qualitative or quantitative methods, are a growing area of interest to the following stakeholders involved in the medical product lifecycle: academics, health technology assessment bodies, payers, industry, patients, physicians, and regulators. However, the use of PP in decisions along the medical product lifecycle remains limited. As the adoption of PP heavily relies on these stakeholders, knowledge of their perceptions of PP is critical. Objective This study aimed to characterize stakeholders' attitudes, needs, and concerns with respect to PP in decision making along the medical product lifecycle. Methods Semi-structured interviews (n = 143) were conducted with academics (n = 24), health technology assessment/payer representatives (n = 24), industry representatives (n = 24), patients, caregivers and patient representatives (n = 24), physicians (n = 24), and regulators (n = 23) from seven European countries and the USA. Interviews were conducted between April and August 2017. The framework method was used to organize the data and identify themes and key findings in each interviewed stakeholder group. Results Interviewees reported being unfamiliar (43%), moderately familiar (42%), or very familiar (15%) with preference methods and studies. Interviewees across stakeholder groups generally supported the idea of using PP in the medical product lifecycle but expressed mixed opinions about the feasibility and impact of using PP in decision making. Interviewees from all stakeholder groups stressed the importance of increasing stakeholders' understanding of the concept of PP and preference methods and ensuring patients' understanding of the questions asked in PP studies. Key concerns and needs in each interviewed stakeholder group were as follows: (1) academics: investigating the validity, reliability, reproducibility, and generalizability of preference methods; (2) health technology assessment/payer representatives: developing quality criteria for evaluating PP studies and gaining insights into how to weigh them in reimbursement/payer decision making; (3) industry representatives: obtaining guidance on PP studies and recognition on the importance of PP from decision makers; (4) patients, caregivers, and patient representatives: providing an incentive and adequate information towards patients when participating in PP studies; (5) physicians: avoiding bias as a result of commercial agendas in PP studies and clarifying how to deal with subjective and emotional elements when measuring PP; and (6) regulators: avoiding the misuse of PP study results to overrule the traditional efficacy and safety criteria used for marketing authorization and obtaining robust PP study results. Conclusions Despite the interest all interviewed stakeholder groups reported in PP, the effective use of PP in decision making across the medical product lifecycle is currently hampered by a lack of standardization and consensus on how to both measure and use PP.

Background: Multiple sclerosis (MS) is a chronic inflammatory disorder affecting the brain and spinal cord, characterized by immune-mediated myelin damage. Early intervention and detection programs have emerged as promising strategies to improve patient outcomes by identifying and treating MS in its earliest stages.

Objective: This systematic literature review aims to provide an overview of the preferences, attitudes, and opinions of both patients and healthcare professionals regarding early intervention or early detection programs for MS.

Methods: A comprehensive search strategy was employed in March 2023 across multiple databases (MEDLINE, Scopus, PsyInfo, PubMed), from 1990 to 2023. A total of 38 articles were selected for analysis based on predefined inclusion and exclusion criteria.

Results: The majority of articles were published in recent years and represented different methods from case reports to randomized controlled trials, with fewer systematic literature reviews. Data collection approaches included patients, healthcare workers, or mixed samples with varying age ranges and gender ratios, frequently preferring women. These samples represented different preference study methods. The included studies were primarily conducted in the USA and the UK. Thematic analysis revealed several key themes : 1) differences emerged between healthcare professionals' and patients' perspectives 2) interventions for MS outside Disease-Modifying Therapies (DMTs) 3) severe side effects 4) communication, information, and knowledge 5) psychological and emotional aspects.

Conclusions: Understanding these diverse factors and subgroups within the MS population can inform more effective, personalized approaches to MS prevention and treatment.

Background New disease-modifying ways to treat Parkinson’s disease (PD) may soon become a reality with intracerebral transplantation of cell products produced from human embryonic stem cells (hESCs). The aim of this study was to assess what factors influence preferences of patients with PD regarding stem-cell based therapies to treat PD in the future.

Methods Patients with PD were invited to complete a web-based discrete choice experiment to assess the importance of the following attributes: (i) type of treatment, (ii) aim of treatment, (iii) available knowledge of the different types of treatments, (iv) effect on symptoms, and (v) risk for severe side effects. Latent class conditional logistic regression models were used to determine preference estimates and heterogeneity in respondents’ preferences.

Results A substantial difference in respondents’ preferences was observed in three latent preference patterns (classes). “Effect on symptoms” was the most important attribute in class 1, closely followed by “type of treatment,” with medications as preferred to other treatment alternatives. Effect on symptoms was also the most important attribute in class 2, with treatment with hESCs preferred over other treatment alternatives. Likewise for class 3, that mainly focused on “type of treatment” in the decision-making. Respondents’ class membership was influenced by their experience in treatment, side effects, and advanced treatment therapy as well as religious beliefs.

Conclusions Most of the respondents would accept a treatment with products emanating from hESCs, regardless of views on the moral status of embryos. Preferences of patients with PD may provide guidance in clinical decision-making regarding treatments deriving from stem cells.

Aim: Early assessment of patient preferences has the potential to support shared decisions in personalized precision medicine for patients with rheumatoid arthritis (RA). The aim of this study was to assess treatment preferences of patients with RA (< 5 years) with previous experience of inadequate response to first-line monotherapy. Method: Patients were recruited (March–June 2021) via four clinics in Sweden. Potential respondents (N = 933) received an invitation to answer a digital survey. The survey included an introductory part, a discrete choice experiment (DCE) and demographic questions. Each respondent answered 11 hypothetical choice questions as part of the DCE. Patient preferences and preference heterogeneity were estimated using random parameter logit models and latent class analysis models. Results: Patients (n = 182) assessed the most important treatment attributes out of physical functional capacity, psychosocial functional capacity, frequency of mild side effects and likelihood of severe side effects. In general, patients preferred a greater increase in functional capacity and decreased side effects. However, a substantial preference heterogeneity was identified with two underlying preference patterns. The most important attribute in the first pattern was the ‘likelihood of getting a severe side effect’. Physical functional capacity was the most important attribute in the second pattern. Conclusion: Respondents focused their decision-making mainly on increasing their physical functional capacity or decreasing the likelihood of getting a severe side effect. These results are highly relevant from a clinical perspective to strengthen communication in shared decision making by assessing patients’ individual preferences for benefits and risks in treatment discussions.

Introduction: Individualisation of rheumatoid arthritis (RA) treatment needs to take account of individual patients’ preferences to increase patient-centeredness in treatment decisions. The aim of this study was to identify patient-relevant treatment attributes to consider when individualising treatment for patients with RA.

Method: Patients with RA in Sweden were invited to rank the most important treatment attributes in an online survey (April to May 2020). Semi-structured interviews were conducted (October to November 2020) to further identify and frame potential attributes for shared decision-making. The interviews were audio-recorded, transcribed and analysed using thematic framework analysis. Patient research partners and rheumatologists supported the selection and framing of the treatment attributes across the assessment.

Results: The highest ranked attributes (N = 184) were improved functional capacity, reduced inflammation, reduced pain and fatigue and the risk of getting a severe side effect. The framework analysis revealed two overarching themes for further exploration: treatment goals and side effects. ‘Treatment goals’ emerged from functional capacity, revealing two dimensions: physical functional capacity and psychosocial functional capacity. ‘Side effects’ revealed that mild and severe side effects were the most important to discuss in shared decision-making.

Conclusions: Functional capacity (physical and psychosocial) and potential side effects (mild and severe) are important treatment attributes to consider when individualising RA treatment. Future research should assess how patients with RA weigh benefits and risks against each other, in order to increase patient-centeredness early on the treatment trajectory.

BackgroundParkinson’s disease (PD) has been considered to be one of the most promising target diseases for forthcoming cell-based therapy. The aim of this study is to explore the views of individuals with cryopreserved embryos on using human embryonic stem cells for treating PD.

MethodsThe study was performed as a qualitative, semi-structured interview study in June–October 2020. Participants were recruited at a private fertility clinic located in one of the larger Swedish cities. The clinic provides both publicly financed and privately financed IVF-treatments. All interviews were performed by telephone and analyzed using thematic content analysis. Five main categories emerged from 27 sub-categories.

ResultsIn total, 18 interviews were performed with 22 individuals, as either a couple (n = 16) or separately (n = 6). Participants had different views on what a cryopreserved embryo is. Some participants addressed cryopreserved embryos as ‘a lump of cells’, and some in terms of their ‘unborn child’. Conditions for donation of cryopreserved embryos for cell-based treatment in PD were: not losing control of what is happening to the embryo, that donating must be voluntary and based on informed consent with time for reflection, that reimbursement, equality and transparency.

ConclusionsUsing cryopreserved embryos to treat PD is associated with fundamental ethical and practical issues. This study shows that IVF couples with left-over embryos may be supportive but there is a need for future research to assess people’s views on using cryopreserved embryos for cell-based treatment in PD on a more aggregated level.

Objective: Precision medicine in rheumatoid arthritis (RA) creates new opportunities to involve patients in early identification of accurate indicators of health trajectories. The aim of this study was to explore patient perspectives on patient-centredness in precision medicine for RA treatment.

Design: Semistructured interviews were conducted to explore patients' perspectives on a new personalised approach to RA treatment. The interview guide was developed together with patient research partners and health care professionals.

Setting: An invitation to the interviews was sent through a mobile application. The interviews were one-on-one, using an interview guide with open-ended questions. Interviews were conducted digitally (October 2020-February 2021) via Zoom or telephone, depending on each participant's preferences.

Participants: Patients with RA (N=12) were purposively recruited. Patients were eligible if they had an RA diagnosis, were aged 18-80 years, and understood and expressed themselves in Swedish. Participants and researchers did not know each other prior to the interviews.

Results: Participants expressed desires and needs for patients to have an active role in precision medicine by making shared treatment decisions together with a healthcare professional. In order for that to work, patients need information on potential treatment options, an ability to express their preferences, an individual treatment plan and identification of personal treatment goals. Patients also identified two requirements of healthcare professional in precision medicine: a safe environment to express personal matters and two-way communication with healthcare professionals.

Conclusion: Communication between patients and healthcare professionals needs to be more focused on patients' individual treatment preferences and expressed needs, in order to increase patient-centredness in treatment decisions, so shared decision-making can become a reality. More research is needed to design multifaceted implementation strategies to support patients and healthcare professionals to increase patient-centredness throughout treatment personalisation.

Background: Preference assessments of patients with rheumatoid arthritis can support clinical therapeutic decisions for including biologic and targeted synthetic medicines to use. This study assesses patient preferences for attributes of second-line therapies and heterogeneity within these preferences to estimate the relative importance of treatment characteristics and to calculate the minimum benefit levels patients require to accept higher levels of side effects.

Methods: Between November 2018 to August 2019, patients with rheumatoid arthritis were recruited to a survey containing demographic and disease-related questions as well as a discrete choice experiment to measure their preferences for second-line therapies using biologics or Janus kinases inhibitors. Treatment characteristics included were route of administration, frequency of use, probability of mild short-term side effects, probability of side effects changing appearance, probability of psychological side effects, probability of severe side effects and effectiveness of treatment.

Results: A total of 358 patients were included in the analysis. A latent class analysis revealed three preference patterns: (1) treatment effectiveness as the single most important attribute, (2) route of administration as the most important attribute, closely followed by frequency of use and psychological side effects and (3) severe side effects as the most important attribute followed by psychological side effects. In addition, disease duration and mild side effects influenced the patients’ choices.

Conclusion: Respondents found either effectiveness, route of administration or severe side effects as the most important attribute. Patients noting effectiveness as most important were more willing than other patients to accept higher risks of side effects. 

Background

A more digitalised world comes with the promise to improve people’s lives. Therefore, it is essential that policymakers also align digital interventions with initiatives to empower citizens and strengthen their digital health literacy. The aim of this study was to explore the views of Swedish policymakers regarding the potential and barriers of a European strategy to strengthen digital health literacy. 

Method

Representatives from Swedish governmental agencies and regions were purposively approached by email to ask them to participate in online workshops to discuss the potential and barriers of developing a European strategy to strengthen digital health literacy. 

Results

The results highlight the need for a national strategy to strengthen digital health literacy. The findings point to critical areas for improvement, ethical and social considerations, and the importance of inclusive and accessible health information online. Participants identified vulnerable groups requiring targeted support to enhance their digi- tal health literacy, particularly those at risk of digital exclusion. Participants emphasised the importance of consider- ing various combinations of conditions or problems that people may have, urging policymakers to adopt a nuanced approach to enhance digital literacy. 

Conclusions

There is a critical need for policymakers to strengthen digital health literacy in the population to ensure health equity in relation to digitalisation opportunities. Policymakers advocate for a dedicated national strategy, sup- porting policymakers to prioritize digital health literacy. Tailoring information, enhancing digital support for preven- tion, and considering ethical implications are reported as important aspects to improve digital health literacy.

Objectives: There is an increased interest in patient preferences informing the development and authorisation of medical products. A requirement for robust and meaningful results of such studies is that patients adequately understand the risks and benefits associated with treatments for which their preferences are elicited. This study aims to determine the influence of an educational tool, compared with traditional written information on patient preferences elicited in a discrete choice experiment (DCE).

Methods: Treatment preferences of Swedish patients with rheumatoid arthritis (RA) were assessed using a DCE. Patients were recruited via clinics, a research panel, and the Swedish Rheumatism Association. Respondents received training materials either as plain written text or as an online educational tool. The educational tool was designed to enhance understanding of the written text by using graphics, pictograms, icon arrays, spoken text, and click-on functions. Data were analysed using random parameter logit models.

Results: 675 patients with RA were included in the analysis. The patients received either a written information (n = 358) or information via an educational tool (n = 317). Respondents receiving the educational tool placed relatively more importance on all included side effects in their decision making, compared to respondents receiving the written text, who placed greater importance on treatment effectiveness and administration methods.

Conclusion: Compared to the respondents receiving the written text, the decisions of respondents receiving the educational tool were more influenced by medication side effects. Further research is needed to provide guidance on how and when to use educational tools to inform and elicit patients’ preferences.

Practice implications: The ways in which attributes are presented to patients significantly impacts preferences measured in a DCE.

Background

There is increasing interest in involving patient preferences for benefits and risks in regulatory decision making. Therefore, it is essential to identify patient perspectives regarding the value of patient preference information (PPI).

Objectives

The aim of this study was to explore how patients with rheumatoid arthritis (RA) value the use of PPI in regulatory decision making regarding medical products.

Methods

Regulators and patients with RA were interviewed to gather initial insights into opinions on the use of PPI in regulatory decisions regarding medical products. The interviews were used to draft and validate the interview guide for focus groups with patients with RA. Participants were purposively sampled in collaboration with the Swedish Rheumatism Association in Stockholm and Uppsala. Each focus group consisted of three to six patients (18 in total). All interviews were audio-recorded, transcribed verbatim, and analysed using content analysis.

Results

According to the participants, PPI could lead to regulators considering patients’ needs, lifestyles and well-being when making decisions. PPI was important in all stages of the medical product lifecycle. Participants reported that, when participating in a preference study, it is important to be well-informed about the use of the study and the development, components, administration, and risks related to the medical products.

Conclusions

Patients thought PPI could be valuable to consider in regulatory decisions. It is essential for patients to be well-informed when asked for their preferences. Research on information materials to inform patients in preference studies is needed to increase the value of PPI in regulatory decision making.

Objective Some immunomodulatory drugs have been shown to delay the onset of, or lower the risk of developing, rheumatoid arthritis (RA), if given to individuals at risk. Several trials are ongoing in this area; however, little evidence is currently available about the views of those at risk of RA regarding preventive treatment. Method Three focus groups and three interviews explored factors that are relevant to first degree relatives (FDRs) of RA patients and members of the general public when considering taking preventive treatment for RA. The semi-structured qualitative interview prompts explored participant responses to hypothetical attributes of preventive RA medicines. Transcripts of focus group/interview proceedings were inductively coded and analysed using a framework approach. Results Twenty-one individuals (five FDRs, 16 members of the general public) took part in the study. Ten broad themes were identified describing factors that participants felt would influence their decisions about whether to take preventive treatment if they were at increased risk of RA. These related either directly to features of the specific treatment or to other factors, including personal characteristics, attitude towards taking medication, and an individual's actual risk of developing RA. Conclusion This research highlights the importance of non-treatment factors in the decision-making process around preventive treatments, and will inform recruitment to clinical trials as well as information to support shared decision making by those considering preventive treatment. Studies of treatment preferences in individuals with a confirmed high risk of RA would further inform clinical trial design.

Treatments used for rheumatoid arthritis (RA) are under investigation for their efficacy to prevent RA in at risk groups. It is therefore important to understand treatment preferences of those at risk. We systematically reviewed quantitative preference studies of drugs to treat, or prevent RA, to inform the design of further studies and trials of RA prevention. Stated preference studies for RA treatment or prevention were identified through a search of five databases. Study characteristics and results were extracted, and the relative importance of different types of treatment attributes was compared across populations. Twenty three studies were included 20 of RA treatments (18 of patients; 2 of the general public) and 3 prevention studies with first-degree relatives (FDRs). Benefits, risks, administration method and cost (when included) were important determinants of treatment choice. A benefit was more important than a risk attribute in half of the studies of RA treatment that included a benefit attribute and 2/3 studies of RA prevention. There was variability in the relative importance of attributes across the few prevention studies. In studies with non-patient participants, attributes describing confidence in treatment effectiveness/safety were more important determinants of choice than in studies with patients. Most preference studies relating to RA are of treatments for established RA. Few studies examine preferences for treatments to prevent RA. Given intense research focus on RA prevention, additional preference studies in this context are needed. Variation in treatment preferences across different populations is not well understood and direct comparisons are needed.

OBJECTIVE: To quantify preferences for preventive therapies for rheumatoid arthritis (RA) across three countries.

METHODS: A web-based survey including a discrete choice experiment was administered to adults recruited via survey panels in the UK, Germany and Romania. Participants were asked to assume they were experiencing arthralgia and had a 60% chance of developing RA in the next 2 years and completed 15 choices between no treatment and two hypothetical preventive treatments. Treatments were defined by six attributes (effectiveness, risks and frequency/route of administration) with varying levels. Participants also completed a choice task with fixed profiles reflecting subjective estimates of candidate preventive treatments. Latent class models (LCMs) were conducted and the relative importance of attributes, benefit-risk trade-offs and predicted treatment uptake was subsequently calculated.

RESULTS: Completed surveys from 2959 participants were included in the analysis. Most participants preferred treatment over no treatment and valued treatment effectiveness to reduce risk more than other attributes. A five-class LCM best fitted the data. Country, perceived risk of RA, health literacy and numeracy predicted class membership probability. Overall, the maximum acceptable risk for a 40% reduction in the chance of getting RA (60% to 20%) was 21.7%, 19.1% and 2.2% for mild side effects, serious infection and serious side effects, respectively. Predicted uptake of profiles reflecting candidate prevention therapies differed across classes.

CONCLUSION: Effective preventive pharmacological treatments for RA were acceptable to most participants. The relative importance of treatment attributes and likely uptake of fixed treatment profiles were predicted by participant characteristics.

Objectives: To investigate stakeholder perspectives on how patient preference studies (PPS) should be designed and conducted to allow for inclusion of patient preferences in decision-making along the medical product life cycle (MPLC), and how patient preferences can be used in such decision-making.

Methods: Two literature reviews and semi-structured interviews (n = 143) with healthcare stakeholders in Europe and the US were conducted; results of these informed the design of focus group guides. Eight focus groups were conducted with European patients, industry representatives and regulators, and with US regulators and European/Canadian health technology assessment (HTA) representatives. Focus groups were analyzed thematically using NVivo.

Results: Stakeholder perspectives on how PPS should be designed and conducted were as follows: 1) study design should be informed by the research questions and patient population; 2) preferred treatment attributes and levels, as well as trade-offs among attributes and levels should be investigated; 3) the patient sample and method should match the MPLC phase; 4) different stakeholders should collaborate; and 5) results from PPS should be shared with relevant stakeholders. The value of patient preferences in decision-making was found to increase with the level of patient preference sensitivity of decisions on medical products. Stakeholders mentioned that patient preferences are hardly used in current decision-making. Potential applications for patient preferences across industry, regulatory and HTA processes were identified. Four applications seemed most promising for systematic integration of patient preferences: 1) benefit-risk assessment by industry and regulators at the marketing-authorization phase; 2) assessment of major contribution to patient care by European regulators; 3) cost-effectiveness analysis; and 4) multi criteria decision analysis in HTA.

Conclusions: The value of patient preferences for decision-making depends on the level of collaboration across stakeholders; the match between the research question, MPLC phase, sample, and preference method used in PPS; and the sensitivity of the decision regarding a medical product to patient preferences. Promising applications for patient preferences should be further explored with stakeholders to optimize their inclusion in decision-making.

Objective

We aimed to empirically compare maximum acceptable risk results estimated using both a discrete choice experiment (DCE) and a probabilistic threshold technique (PTT).

Methods

Members of the UK general public (n = 982) completed an online survey including a DCE and a PTT (in random order) measuring their preferences for preventative treatment for rheumatoid arthritis. For the DCE, a Bayesian D-efficient design consisting of four blocks of 15 choice tasks was constructed including six attributes with varying levels. The PTT used identical risk and benefit attributes. For the DCE, a panel mixed-logit model was conducted, both mean and individual estimates were used to calculate maximum acceptable risk. For the PTT, interval regression was used to calculate maximum acceptable risk. Perceived complexity of the choice tasks and preference heterogeneity were investigated for both methods.

Results

Maximum acceptable risk confidence intervals of both methods overlapped for serious infection and serious side effects but not for mild side effects (maximum acceptable risk was 32.7 percent-points lower in the PTT). Although, both DCE and PTT tasks overall were considered easy or very easy to understand and answer, significantly more respondents rated the DCE choice tasks as easier to understand compared with those who rated the PTT as easier (7-percentage point difference; p < 0.05).

Conclusions

Maximum acceptable risk estimate confidence intervals based on a DCE and a PTT overlapped for two out of the three included risk attributes. More respondents rated the DCE as easier to understand. This may suggest that the DCE is better suited in studies estimating maximum acceptable risk for multiple risk attributes of differing severity, while the PTT may be better suited when measuring heterogeneity in maximum acceptable risk estimates or when investigating one or more serious adverse events.

 Objectives:  Over the last few decades, there have been significant improvements in the treatment of rheumatoid arthritis (RA), with the development of new treatments and guidelines for teamwork and patient self-care and access to digital tools. This study aimed to explore the experiences of individuals with RA interacting with healthcare. It also looked at how a self-care application, an educational programme called the 'healthcare encounter', improved patient-doctor communication.

 Design:  Semistructured interviews were conducted, and qualitative content analysis was performed.

 Setting:  The potential participants, individuals with established, or under investigation for, RA diagnosis at rheumatology clinics in Sweden, were asked to participate in the study via a digital self-care application called the Elsa Science Self-care app.

 Participants:  Ten interviews were performed with participants from nine clinics following a meeting with the rheumatologist or other healthcare personnel between September 2022 and October 2022. Phrases, sentences or paragraphs referring to experiences from healthcare meetings and opinions about the digital programme were identified and coded. Codes that reflected similar concepts were grouped; subcategories were formulated, and categories were connected to their experiences and opinions.

 Results:  Among our participants, three main categories emerged: the availability of healthcare, individual efforts to have a healthier life and personal interaction with healthcare. Participants described that the 'healthcare encounter' educational programme can be a source of information, which confirms, supports and creates a sense of control.

 Conclusion:  The participants valued being seen and taking part in a dialogue when they had prepared themselves (observed symptoms over time and prepared questions). The implementation of digital self-care applications might need to be incorporated into the healthcare setting, so that both the patients and the healthcare personnel have a shared understanding. Collaboration is essential in this context.

Introduction: Research on young children’s occupational aspirations and the factors shaping them is still limited, especially in early interventions addressing gender disparities in high-status fields like STEM.

Methods: This is the first study in Sweden utilizing the Drawing the Future method, surveyed 1,832 children (aged 5–13) from 28 schools in Skåne region of southern, asking them to draw their dream jobs. This exercise was conducted in a classroom setting and facilitated by their class teacher.

Results: Significant gender differences emerged, revealing distinct stereotypical patterns in children’s future occupational aspirations and influencing factors. Only three occupations—footballer, doctor, and police officer—were popular among both genders. Girls preferred people- or animal-centered roles, while boys leaned toward jobs involving “things” (p < 0.001). Girls felt they could pursue similar careers as boys, but boys showed more skepticism (p < 0.001). Influence patterns also varied by gender: 25% of girls were inspired by mothers, while 45% of boys were inspired by fathers (p = 0.02). Beyond immediate family, girls often sought career information from acquaintances, while boys turned to media (p < 0.001). STEM interest was limited, with “game developer” being the only STEM job on boys’ lists. Additionally, a larger proportion of boys ranked STEM subjects among their top 10 favorite school subjects, while girls preferred crafts, art, and English (p < 0.001).

Discussion: These findings highlight the need for early, unbiased, evidence-based career interventions and policies to broaden children’s awareness of diverse job options and opportunities in the labor market.

<h1>Background</h1><p>Patient preference (PP) information is not effectively integrated in decision-making throughout the medical product lifecycle (MPLC), despite having the potential to improve patients’ healthcare options. A first step requires an understanding of existing processes and decision-points to know how to incorporate PP information in order to improve patient-centric decision-making.</p><h1>Objectives</h1><p>The aims were to: 1) identify the decision-making processes and decision-points throughout the MPLC for industry, regulatory authorities, and reimbursement/HTA, and 2) determine which decision-points can potentially include PP information.</p><h1>Methods</h1><p>A scoping literature review was conducted using five scientific databases. Semi-structured interviews were conducted with representatives from seven European countries and the US, including industry (n = 24), regulatory authorities (n = 23), reimbursement/HTA (n = 23). Finally, validation meetings with key stakeholders (n = 11) were conducted.</p><h1>Results</h1><p>Six critical decision-points were identified for <em>industry decision-making,</em> three for <em>regulatory decision-making</em>, and six for <em>reimbursement/HTA decision-making.</em> Stakeholder groups agreed that PP information is not systematically integrated, either as obligatory information or pre-set criteria, but would benefit all the listed decision-points in the future.</p><h1>Conclusion</h1><p>Currently, PP information is not considered as obligatory information to submit for any of the MPLC decision-points. However, PP information is considered an important component by most stakeholders to inform future decision-making across the MPLC. The integration of PP information into 15 identified decision-points needs continued discussion and collaboration between stakeholders.</p>

Objectives: Patient preference information (PPI) is gaining recognition among the pharmaceutical industry, regulatory authorities, and health technology assessment (HTA) bodies/payers for use in assessments and decision-making along the medical product lifecycle (MPLC). This study aimed to identify factors and situations that influence the value of patient preference studies (PPS) in decision-making along the MPLC according to different stakeholders.

Methods: Semi-structured interviews (n = 143) were conducted with six different stakeholder groups (physicians, academics, industry representatives, regulators, HTA/payer representatives, and a combined group of patients, caregivers, and patient representatives) from seven European countries (the United Kingdom, Sweden, Italy, Romania, Germany, France, and the Netherlands) and the United States. Framework analysis was performed using NVivo 11 software.

Results: Fifteen factors affecting the value of PPS in the MPLC were identified. These are related to: study organization (expertise, financial resources, study duration, ethics and good practices, patient centeredness), study design (examining patient and/or other preferences, ensuring representativeness, matching method to research question, matching method to MPLC stage, validity and reliability, cognitive burden, patient education, attribute development), and study conduct (patients’ ability/willingness to participate and preference heterogeneity). Three types of situations affecting the use of PPS results were identified (stakeholder acceptance, market situations, and clinical situations).

Conclusion: The factors and situation types affecting the value of PPS, as identified in this study, need to be considered when designing and conducting PPS in order to promote the integration of PPI into decision-making along the MPLC.