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Background Research about the decision to participate in a clinical study has tended to be limited to single indications and has focused on narrow sets of study and participant characteristics. This study applied stated preference methods to understand the clinical trial design attributes that most influence willingness to participate and how this varied with participant characteristics. Methods Adults residing in the US, China, or Poland with a self-reported diagnosis of cancer, heart disease, migraine, rheumatoid arthritis, or multiple sclerosis completed an online survey. Participants were asked whether they would participate in clinical studies defined by seventeen attributes within five categories (payment/support, administration/procedures, treatment-related, study location/time commitment, and data collection/feedback). Participants saw six different hypothetical clinical study profiles. Depending on their participation decision to an initial clinical study profile, the subsequent five questions had one design attribute (randomly selected per question) consecutively improved or deteriorated to elicit preferences. A logistic regression was used to determine which participant characteristics influenced participation decisions. A latent class logit model was used to identify how the influence of study design features varied between participants and whether groups of participants with similar preferences could be identified. Results The survey was completed by 487 participants (32% China, 35% Poland, 33% US; 8%–19% per indication). Willingness to participate was found to be a function of participant age, certain elements of quality of life, and previous treatment experience, in particular number of lines of treatment received and experience of adverse events. Willingness to participate was influenced by study design features such as payment, study duration, and time commitment – both the overall time and whether the time was at home or away from home, with the latter being particularly relevant to participants experiencing fatigue due to their disease. Conclusions This study quantifies how study designs influence willingness to participate and how this varies with participant types. These findings suggest that it is how an indication influences quality of life and treatment experience, rather than the indication alone, that impacts participation rates, opening the way for insights that are transferrable across indications, which may be particularly useful when considering rare diseases.

ObjectivesWe aimed to quantify patient preferences for efficacy, safety and convenience features of atopic dermatitis (AD) treatments.Design and settingOnline discrete choice experiment survey.ParticipantsAdults in the UK, France and Spain who had used AD treatments during the past 2 years.Primary and secondary outcome measuresPreferences for attributes were analysed using a multinomial logit model. Willingness to make trade-offs was expressed as the maximum acceptable decrease (MAD) in the probability of achieving clear/almost clear skin at week 16.ResultsThe survey was completed by 404 patients (44.1±12.0 years; 65% women; 64% moderate/severe eczema). Most patients (68%) had no prior experience of using self-injectable treatments for AD or any other illness. Participants most valued increasing the chance of achieving a meaningful reduction in itch at week 16 from 20% to 50%, followed by reducing the risks of serious infections from 6% to 0% and of eye inflammation from 20% to 0%. Participants were willing to accept a decrease in the possibility of achieving clear/almost clear skin to obtain a treatment that can be paused (MAD=24.1%), requires occasional check-ups (MAD=16.1%) or no check-ups (MAD=20.9%) over frequent check-ups, is administered as a one time per day or two times per day oral pill versus a subcutaneous injection every 2 weeks (MAD=16.6%), has a 2-day over 2-week onset of action (MAD=11.3%), and can be used for flare management (MAD=5.8%).ConclusionsAlthough patients with AD most valued treatment benefits and risks, they were willing to tolerate reduced efficacy to obtain a rapid onset, oral administration, less frequent monitoring and a treatment that can be paused. Understanding patients’ preferences for AD therapies, including new targeted therapies, can aid shared decision-making between clinicians and patients and support health technology assessments.

Background Patients with follicular lymphoma (FL) often relapse or become refractory to treatment (R/R). While the R/R FL treatment landscape evolves, little is known about the priorities of patients and physicians. This discrete‐choice experiment (DCE) study assessed patients' and physicians' treatment preferences, and the trade‐offs they would be willing to make between efficacy, tolerability, and administration. Methods An online survey was conducted in US‐based patients (≥18 years) with R/R FL and FL‐treating physicians. The DCE was informed by a targeted literature review, clinical data, expert oncologist input, and pilot interviews. Participants completed eight experimental choice tasks where they chose between two hypothetical treatment profiles defined by six attributes: progression‐free survival (PFS), administration/monitoring, risks of laboratory abnormalities requiring intervention, severe infections, diarrhea, and cytokine release syndrome (CRS). Relative attribute importance (RAI) and willingness to trade‐off between PFS and other attributes were estimated. Results Two‐hundred patients (mean age 63.5 years; median three prior lines of therapy) and 151 FL‐treating physicians participated. Increasing PFS was most important for both groups, although it was relatively less important to patients than physicians (RAI 35.2% vs. 45.7%). Administration/monitoring was three times more important to patients than physicians (RAI 28.8% vs. 9.5%); patients preferred oral treatment and would be willing to tolerate a significant reduction in PFS for oral administration over weekly intravenous infusions. Avoiding CRS was less important to patients than to physicians (RAI 7.7% vs. 15.8%). Both groups would accept shorter PFS for reduced risks of side effects (especially of laboratory abnormalities for patients and of CRS for physicians). Conclusion Although PFS was the most important attribute to patients and physicians, both would tolerate lower PFS for reduced side effects. Patients would also accept a substantial reduction in PFS for oral administration. Differences between the preferences/priorities of patients and physicians highlight the importance of shared decision‐making.

Background Poor distribution of already inadequate numbers of health professionals seriously constrains equitable access to health services in low- and middle-income countries. The Senegalese Government is currently developing policy to encourage health professionals to remain in areas defined as ‘difficult’. Understanding health professional’s preferences is crucial for this policy development. Methods Working with the Senegalese Government, a choice experiment (CE) was developed to elicit the job preferences of physicians and non-physicians. Attributes were defined using a novel mixed-methods approach, combining interviews and best-worst scaling (Case 1). Six attributes were categorised as ‘individual (extrinsic) incentive’ attributes (‘type of contract’, ‘provision of training opportunities’, ‘provision of an allowance’ and ‘provision of accommodation’) or ‘functioning health system’ attributes (‘availability of basic equipment in health facilities’ and ‘provision of supportive supervision by health administrators’). Using face-to-face interviews, the CE was administered to 55 physicians (3909 observations) and 246 non-physicians (17 961 observations) randomly selected from those working in eight ‘difficult’ regions in Senegal. Conditional logit was used to analyse responses. This is the first CE to both explore the impact of contract type on rural retention and to estimate value of attributes in terms of willingness to stay (WTS) in current rural post. Results For both physicians and non-physicians, a permanent contract is the most important determinant of rural job retention, followed by availability of equipment and provision of training opportunities. Retention probabilities suggest that policy reform affecting only a single attribute is unlikely to encourage health professionals to remain in ‘difficult’ regions. The relative importance of an allowance is low; however, the level of such financial incentives requires further investigation. Conclusion Contract type is a key factor impacting on retention. This has led the Senegalese Health Ministry to introduce a new rural assignment policy that recruits permanent staff from the pool of annually contracted healthcare professionals on the condition that they take up rural posts. While this is a useful policy development, further efforts to retain rural health workers, considering both personal incentives and the functioning of health systems, are necessary to ensure health worker numbers are adequate to meet the needs of rural communities.

Despite being effective in slowing the spread of the disease, the policies have detrimental effects on the economy, elective health care provision, mental health, and societal freedom [10, 11]. Furthermore, with the COVID-19 cases falling in several countries and many vaccines as well as treatments being tested in parallel, clinical trials may not be able to demonstrate efficacy with the same statistical power usually required for regulatory approval [12, 21]. [...]societies may face a trade-off between vaccine efficacy and the duration of implemented interventions such as social distancing. [...]the preference elicitation exercise deliberately simplified the vaccine choice context to average efficacy and waiting time, excluding other potentially relevant attributes such as risk of side effects.

Newer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients' preferences is essential. This study assessed patients' preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects. Patients' preferences were evaluated using a discrete choice experiment (DCE). Patients with RRMM who reported failing two lines of anti-myeloma treatment (immunomodulatory agent and a proteasome inhibitor [PI]) or ≥ 3 lines (including ≥1 PI, immunomodulatory agent, or anti-CD38 monoclonal antibody), were recruited across the US, UK, Italy, Germany, France, and Spain. DCE attributes and levels were identified using a targeted literature review, a review of clinical data for relevant RRMM treatments, qualitative patient interviews, and input from clinical and myeloma patient experts. The DCE was administered within an online survey from February-June 2022. Preference data were analyzed using an error-component logit model and willingness to make trade-offs for potential benefits, and relative attribute importance scores were calculated. Overall, 296 patients from the US ( = 100), UK ( = 49), Italy ( = 45), Germany ( = 43), France ( = 39), and Spain ( = 20) participated in the DCE. Mean (standard deviation) age was 63.8 (8.0) years, 84% had a caregiver, and patients had a median of 3 (range: 2-8) prior lines of therapy. Efficacy attributes most influenced patients' preferences, with increasing overall response rate (25-85%) and overall survival (6 months to 2 years) contributing to ~50% of treatment decision-making. Administration procedures were also considered important to patients. Avoiding individual side effects was considered relatively less important, with patients willing to tolerate increases in side effects for gains in efficacy. Patient characteristics such as rate of disease progression, sociodemographics, or clinical characteristics also influenced treatment preferences. Patients with RRMM were willing to tolerate increased risk of side effects for higher efficacy. Preferences and risk tolerance varied between patients, with preference patterns differing by certain patient characteristics. This highlights the importance of shared decision-making for optimal treatment selection and patient outcomes.

ObjectiveUnderstanding preferences of patients with rheumatoid arthritis (RA) can facilitate tailored patient-centric care. This study elicited trade-offs that patients with RA were willing to make during treatment selection.MethodsPatients with RA completed an online discrete choice experiment, consisting of a series of choices between hypothetical treatments. Treatment attributes were selected based on literature review and qualitative patient interviews. Eligible patients were ≥18 years old, diagnosed with RA, receiving systemic disease-modifying antirheumatic drug therapy, and residents of Europe or USA. Male patients were oversampled for subgroup analyses. Data were analysed using a correlated mixed logit model.ResultsOf 2090 participants, 42% were female; mean age was 45.2 years (range 18–83). Estimated effects were significant for all attributes (p<0.001) but varied between patients. Average relative attribute importance scores revealed different priorities (p<0.001) between males and females. While reducing pain and negative effect on semen parameters was most important to males, females were most concerned by risk of blood clots and serious infections. No single attribute explained treatment preferences by more than 30%. Preferences were also affected by patients’ age: patients aged 18–44 years placed less importance on frequency and mode of treatment administration (p<0.05) than older age groups. Patients were willing to accept higher risk of serious infections and blood clots in exchange for improvements in pain, daily activities or administration convenience. However, acceptable trade-offs varied between patients (p<0.05).ConclusionTreatment preferences of patients with RA were individual-specific, but driven by benefits and risks, with no single attribute dominating the decision-making.

Background The increased use of patient preference data in healthcare decision making has raised concerns about the reliability and consistency of the estimates generated by patient preference studies. However, literature reviews to assess the consistency of preferences are confounded by heterogeneity in study designs. Methods This paper adopted a novel approach to evaluating preference consistency: comparing estimates of a single trade-off—the marginal rate of substitution (MRS) between survival improvements and risks of adverse events—across multiple patient groups and using meta-regression to assess whether MRS varied systematically between patients. A log-linear, random effects regression was run, weighted for the sample sizes of studies from which estimates were extracted. Results Using studies identified in published reviews of patient preference data, 42 estimates of MRS were generated from the 12 studies. On average, patients obtained the same utility from a 2.3% reduction in the risk of an adverse event as from a 1-month increase in survival, with a range of 0.002–13.5%. The regression model had an R 2 of over 90% and suggests that MRS depended on patients’ expected survival and the type of adverse event being traded. Conclusion These results suggest that although preferences vary between patients, they may do so in systematic and predictable ways. Further, they do so in ways consistent with societal preferences and decision maker priorities toward end-of-life settings. Further work is required to replicate this result in other patient groups and to explore the consistency of preferences for other treatment attributes.

Background The final outcome of any resource allocation decision in healthcare cannot be determined in advance. Thus, decision makers, in deciding which new program to implement (or not), need to accommodate the uncertainty of different potential outcomes (i.e., change in both health and costs) that can occur, the size and nature (i.e., ‘bad’ or ‘good’) of these outcomes, and how they are being valued. Using the decision-making plane, which explicitly incorporates opportunity costs and relaxes the assumptions of perfect divisibility and constant returns to scale of the cost-effectiveness plane, all the potential outcomes of each resource allocation decision can be described. Objective In this study, we describe the development and testing of an instrument, using a discrete choice experiment methodology, allowing the measurement of public preferences for potential outcomes falling in different quadrants of the decision-making plane. Method In a sample of 200 participants providing 4200 observations, we compared four versions of the preference-elicitation instrument using a range of indicators. Results We identified one version that was well accepted by the participants and with good measurement properties. Conclusion This validated instrument can now be used in a larger representative sample to study the preferences of the public for potential outcomes stemming from re-allocation of healthcare resources.