Multidimensional Thresholding for Individual-Level Preference Elicitation.

OBJECTIVES: Multiple methods are available for collecting health preference information. However, information on the design and analysis of novel methods is limited. This article aims to provide the first introduction into the design and analysis of multidimensional thresholding (MDT). METHODS: We introduce MDT as a 2-step approach: First, participants rank the largest possible improvements in all considered attributes by their importance. Second, participants complete a series of systematically combined trade-off questions. Hit-and-Run sampling is used for obtaining preference weights. We also use a computational experiment to compare different MDT designs. RESULTS: The outlined MDT can generate preference information suitable for specifying a multiattribute utility function at the individual level. The computational experiment demonstrates the method's ability to recover preference weights at a high level of precision. While all designs in the computation experiment perform comparably well on average, the design outlined in the paper stands out with a high level of precision even if differences in relative attribute importance are large. CONCLUSION: MDT is suitable for preference elicitation, in particular if sample sizes are small. Future research should help improve the methods (e.g., remove the need for an initial ranking) to increase the potential reach of MDT.

From Qualitative Research to Quantitative Preference Elicitation: An Example in Invasive Meningococcal Disease.

BACKGROUND: Qualitative research is fundamental for designing discrete choice experiments (DCEs) but is often underreported in the preference literature. We developed a DCE to elicit preferences for vaccination against invasive meningococcal disease (IMD) among adolescents and young people (AYP) and parents and legal guardians (PLG) in the United States. This article reports the targeted literature review and qualitative interviews that informed the DCE design and demonstrates how to apply the recent reporting guidelines for qualitative developmental work in preference studies. METHODS: This study included two parts: a targeted literature review and qualitative interviews. The Medline and Embase databases were searched for quantitative and qualitative studies on IMD and immunization. The results of the targeted literature review informed a qualitative interview guide. Sixty-minute, online, semi-structured interviews with AYP and PLG were used to identify themes related to willingness to be vaccinated against IMD. Participants were recruited through a third-party recruiter's database and commercial online panels. Interviews included vignettes about IMD and vaccinations and three thresholding exercises examining the effect of incidence rate, disability rate, and fatality rate on vaccination preferences. Participant responses related to the themes were counted. RESULTS: The targeted literature review identified 31 concepts that were synthesized into six topics for the qualitative interviews. Twenty AYP aged 16-23 years and 20 PLG of adolescents aged 11-17 years were interviewed. Four themes related to willingness to be vaccinated emerged: attitudes towards vaccination, knowledge and information, perception of IMD, and vaccine attributes. Most participants were concerned about IMD (AYP 60%; PLG 85%) and had positive views of vaccination (AYP 80%; PLG 60%). Ninety percent of AYP and 75% of PLG always chose vaccination over no vaccination, independent of IMD incidence rate, disability rate, or fatality rate. CONCLUSION: Willingness to be vaccinated against IMD was affected by vaccine attributes but largely insensitive to IMD incidence and severity. This article provides an example of how to apply the recent reporting guidelines for qualitative developmental work in preference studies, with 21 out of 22 items in the guidelines being considered.

What benefit-risk trade-offs are acceptable to rheumatoid arthritis patients during treatment selection? Evidence from a multicountry choice experiment.

OBJECTIVE: Understanding 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. METHODS: Patients 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. RESULTS: Of 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). CONCLUSION: Treatment preferences of patients with RA were individual-specific, but driven by benefits and risks, with no single attribute dominating the decision-making.

Two Methods, One Story? Comparing Results of a Choice Experiment and Multidimensional Thresholding From a Clinician Preference Study in Aneurysmal Subarachnoid Hemorrhage.

OBJECTIVES: An increasing number of methods are used to elicit health preference information. It is unclear whether different elicitation methods produce similar results and policy advice. Here, we compared the results from a discrete choice experiment (DCE) and multidimensional thresholding (MDT) that were conducted in the same sample. METHODS: Clinicians (N = 350) completed a DCE and MDT to elicit their preferences for 4 attributes related to the medical management of subarachnoid hemorrhage after aneurysm repair. Preference weights were compared between the DCE and MDT using a complete combinatorial convolution test. Additionally, data from the DCE and MDT were used to compute preference-based net treatment values for 16 hypothetical treatment profiles versus 1000 simulated comparators. The implied treatment recommendations were compared between the DCE and MDT. RESULTS: Preference weight distributions and median weights did not differ significantly between the DCE and MDT for any attribute: likelihood of delayed cerebral ischemia (medians 0.48 vs 0.40; P = .41), risk of lung complications (medians 0.27 vs 0.30; P = .52), risk of hypotension (medians 0.10 vs 0.11; P = .55), and risk of anemia (medians 0.07 vs 0.07; P = .50). The DCE and MDT produced similar treatment net value distributions (P > .05) and implied the same treatment recommendations in 82.3% of cases. CONCLUSIONS: The DCE and MDT elicited similar preference distributions and produced the same treatment recommendations for most tested cases. However, the share of people supporting the average treatment recommendation differed. More research is needed to determine how these findings would compare with those in other populations (in particular, patients) and applications.

Preferences of Patients with Amyotrophic Lateral Sclerosis for Intrathecal Drug Delivery: Choosing between an Implanted Drug-Delivery Device and Therapeutic Lumbar Puncture.

BACKGROUND: Novel intrathecal treatments for amyotrophic lateral sclerosis (ALS) may require delivery using lumbar puncture (LP). Implanted drug-delivery devices (IDDDs) could be an alternative but little is known about patients' preferences for intrathecal drug-delivery methods. OBJECTIVE: We aimed to elicit preferences of patients with ALS for routine LP and IDDD use. METHODS: A discrete choice experiment (DCE) and a threshold technique (TT) exercise were conducted online among patients with ALS in the US and Europe. In the DCE, patients made trade-offs between administration attributes. Attributes were identified from qualitative interviews. The TT elicited maximum acceptable risks (MARs) of complications from device implantation surgery. DCE data were analyzed using mixed logit to quantify relative attribute importance (RAI) as the maximum contribution of each attribute to a preference, and to estimate MARs of device failure. TT data were analyzed using interval regression. Four scenarios of LP and IDDD were compared. RESULTS: Participants (N = 295) had a mean age of 57.7 years; most (74.2%) were diagnosed < 3 years ago. Preferences were affected by device failure risk (RAI 28.6%), administration frequency (26.4%), administration risk (19.7%), overall duration (17.8%), and appointment location (7.5%). Patients accepted a 5.6% device failure risk to reduce overall duration from 2 h to 30 min and a 3.6% risk for administration in a local clinic instead of a hospital. The average MAR of complications from implantation surgery was 29%. Patients preferred IDDD over LP in three of four scenarios. CONCLUSION: Patients considered an IDDD as a valuable alternative to LP in multiple clinical settings.

Patient Preferences for Treatment of Bacillus Calmette-Guérin-unresponsive Non-muscle-invasive Bladder Cancer: A Cross-country Choice Experiment.

Background: Patients with non-muscle-invasive bladder cancer (NMIBC) that is unresponsive to bacillus Calmette-Guérin (BCG) immunotherapy face a difficult choice. Immediate radical cystectomy (RC) is effective but might represent overtreatment. Continuing bladder preservation with medical therapy is an alternative, but it risks progression to muscle-invasive bladder cancer (MIBC) and a reduction in survival. Objective: To understand the trade-offs patients are willing to make in selecting treatments for BCG-unresponsive NMIBC. Design setting and participants: Adults with NMIBC from the UK, France, Germany, and Canada who reported current receipt of BCG, disease unresponsive to BCG, or receipt of RC in the previous 12 mo after failure of BCG were recruited to participate in an online choice experiment. Patients were asked to make repeated choices between two hypothetical medical treatments and the option to undergo immediate RC. The medical treatments required trade-offs between the time to RC, the mode and frequency of administration, the risk of experiencing serious side effects, and the risk of disease progression. Outcome measurements and statistical analysis: Error component logit models were used to calculate relative attribute importance (RAI) scores as the maximum percentage contribution to a preference and acceptable benefit-risk trade-offs. Results and limitations: Most of the 107 participants (average age 63 yr) never selected RC (89%) as their preferred option in the choice experiment. Preferences were most affected by time to RC (RAI 55%), followed by risk of progressing to MIBC (RAI 25%), medication administration (RAI 12%), and the risk of serious side effects (RAI 8%). To increase the time to RC from 1 yr to 6 yr, patients accepted a 43.8% increase in the risk of progression and a 66.1% increase in the risk of serious side effects. Conclusions: Patients with BCG-treated NMIBC valued bladder-sparing treatments and were willing to make substantial benefit-risk trade-offs to delay RC. Patient summary: Adults with bladder cancer not invading the bladder muscle completed an online experiment in which they chose between hypothetical medications and bladder removal. The results show that patients would be willing to accept different risks associated with medications to delay bladder removal. Patients considered disease progression the most important risk of medicinal treatment.

Clinicians' preferences for managing aneurysmal subarachnoid hemorrhage using endothelin receptor antagonists.

Background: The endothelin receptor antagonist (ERA) clazosentan is being investigated for the medical prevention of cerebral vasospasm and associated complications, such as delayed cerebral ischemia (DCI), after aneurysmal subarachnoid hemorrhage (aSAH). This study quantified how clinicians weigh the benefits and risks of ERAs for DCI prevention to better understand their treatment needs and expectations. Methods: An online choice experiment was conducted to elicit preferences of neurologists, intensivists, and neurosurgeons treating aSAH in the US and UK for the use of ERAs. The design of the choice experiment was informed by a feasibility assessment (N = 100), one-on-one interviews with clinicians (N = 10), a qualitative pilot (N = 13), and a quantitative pilot (N = 50). Selected treatment attributes included in the choice experiment were: one benefit (likelihood of DCI); and three risks (lung complications, hypotension, and anemia). In the choice experiment, clinicians repeatedly chose best and worst treatment options based on a scenario of a patient being treated in the ICU after aneurism repair. A correlated mixed logit model determined the relative attribute importance (RAI) and associated highest density interval (HDI) as well as acceptable benefit-risk trade-offs. Results: The final choice experiment was completed by 350 clinicians (116 neurologists, 129 intensivists/intensive care clinicians, and 105 neurosurgeons; mean age, 47.4 years). Reducing the likelihood of DCI (RAI = 56.5% [HDI, 53.6-59.5%]) had the largest impact on clinicians' treatment choices, followed by avoiding the risks of lung complications (RAI = 29.6% [HDI, 27.1-32.3%]), hypotension (RAI = 9.2% [HDI, 7.5-10.8%]), and anemia (RAI = 4.7% [HDI, 3.7-5.8%]). Clinicians expected the likelihood of DCI to decrease by ≥8.1% for a 20% increase in the risk of lung complications, ≥2.4% for a 20% increase in the risk of hypotension, and ≥1.2% for a 20% increase in the risk of anemia. Conclusions: Clinicians were willing to accept certain increased risks of adverse events for a reduced risk of DCI after aSAH. The likelihood of DCI occurring after aSAH can therefore be considered a clinically relevant endpoint in aSAH treatment development. Thus, evaluations of ERAs might focus on whether improvements (i.e., reductions) in the likelihood of DCI justify the risks of adverse events.

A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force.

Many qualitative and quantitative methods are readily available to study patient preferences in health. These methods are now being used to inform a wide variety of decisions, and there is a growing body of evidence showing studies of patient preferences can be used for decision making in a wide variety of contexts. This ISPOR Task Force report synthesizes current good practices for increasing the usefulness and impact of patient-preference studies in decision making. We provide the ISPOR Roadmap for Patient Preferences in Decision Making that invites patient-preference researchers to work with decision makers, patients and patient groups, and other stakeholders to ensure that studies are useful and impactful. The ISPOR Roadmap consists of 5 key elements: (1) context, (2) purpose, (3) population, (4) method, and (5) impact. In this report, we define these 5 elements and provide good practices on how patient-preference researchers and others can actively contribute to increasing the usefulness and impact of patient-preference studies in decision making. We also present a set of key questions that can support researchers and other stakeholders (eg, funders, reviewers, readers) to assess efforts that promote the ongoing impact (both intended and unintended) of a particular preference study and additional studies in the future.

Patients' Preferences for Connected Insulin Pens: A Discrete Choice Experiment Among Patients with Type 1 and Type 2 Diabetes.

BACKGROUND: This study quantified how people with diabetes value the unique features of connected insulin pens and related mobile apps, and the underlying reasons for preferring connected versus non-connected insulin pens. METHODS: A discrete choice experiment (DCE) was conducted in the USA and UK to elicit preferences of adults (≥ 18 years) with type 1 or 2 diabetes for attributes of insulin pens. Attributes included device type, dosing support, glucose monitoring, additional app features, and data sharing. Relative attribute importance (RAI) scores were calculated to capture the relative importance of an attribute. Predicted choice probabilities were obtained to compare different profiles for connected and non-connected insulin pens. RESULTS: The DCE was completed by 540 participants (58.9% male; 90.7% Caucasian; mean age, 58.3 years; 69.4% type 2 diabetes). Participants most valued the possibility of using a connected insulin pen with dosing support and automated dose logging (RAI = 39.9%), followed by automatic transfer of glucose levels (RAI = 29.0%), additional features of tracking diet and physical activity (RAI = 14.6%), data sharing (RAI = 13.6%), and device type (RAI = 2.9%). All profiles of connected insulin pens were preferred over a non-connected pen (p < 0.001), and pen profiles with advanced features were preferred over those without (p < 0.001). Preferences differed by age but not diabetes type, country of residence, or insulin regimen. CONCLUSION: People with diabetes in the USA and UK prefer connected over non-connected insulin pens due largely to the availability of automated logging of dose and glucose levels.

People With Diabetes and Caregivers Prefer Rescue Glucagon Treatment With a Wider Storage Temperature Range and a Nasal Administration, When Efficacy is Similar: A Discrete Choice Experiment in Spain.

BACKGROUND: Conventional injectable glucagon (IG) and nasal glucagon (NG), both having similar efficacy, are two options for the emergency treatment of severe hypoglycemia in Spain. This study elicited the effect of changes in key attributes on preferences for NG and IG medication profiles of people with diabetes and caregivers in Spain. METHODS: The relative attribute importance (RAI) that participants placed on glucagon preparation, preparation and administration time, delivery method, recovery time, device size, storage temperature, and headache risk was estimated from an online discrete choice experiment. In addition, patients and caregivers were presented with NG and IG profiles that included rates of successful administration; the proportion of participants choosing each profile was summarized. RESULTS: The analysis included 276 adults with diabetes (65% type 1) and 270 caregivers (49% type 1). Overall mean age was 40 years; 51% were female. The most important attributes were storage temperature (RAI [95% confidence interval] = 27.3% [22.9-32.2]) and delivery method (17.4% [13.1-21.9]). Headache risk (16.2% [11.8-20.7]), time to prepare and administer (14.5% [10.1-18.8]), glucagon preparation (11.4% [6.8-15.8]), recovery time (8.9% [4.3-13.3]), and device size (4.3% [0.3-8.8]) were also relevant. When comparing medication profiles, significantly more participants (78%) preferred NG over IG profiles (P < .001). CONCLUSION: Adults with diabetes and caregivers prefer a glucagon treatment with a higher rate of successful administration, wider storage temperature, and nasal delivery method, when efficacy is similar. Participants favored NG over conventional IG as a rescue medication for severe hypoglycemia. This information may help decision-making by payers and treatment discussions between health care professionals and patients.

Patients with Inflammatory Bowel Disease Have Heterogeneous Treatment Preferences That Are Largely Determined by the Avoidance of Abdominal Pain and Side Effects [P-POWER IBD Study].

BACKGROUND AND AIMS: Patient-centric management of inflammatory bowel disease [IBD] is important, with consensus considering patient-reported outcomes alongside clinical and endoscopic assessment by healthcare providers. However, evidence regarding patients' treatment priorities is still limited. This study aimed to elicit benefit-risk trade-offs that patients with IBD are willing to make, to help inform discussions about patient-centric treatment targets. METHODS: This was a cross-sectional online survey of adults with self-confirmed Crohn's disease [CD] or ulcerative colitis [UC] receiving IBD treatment. The impact of efficacy, administration and safety on treatment preferences was elicited using a discrete choice experiment. Relative attribute importance [RAI] and maximum acceptable risk of mild-to-moderate side effects [SEs] were estimated from a mixed logit model. RESULTS: In total, 400 patients [CD: 54%; UC: 46%; female: 38.0%; age range: 18-78 years] were recruited. Efficacy, administration and safety affected treatment preferences to varying degrees, with abdominal pain being most important [RAI 33%] followed by risks of mild-to-moderate SEs [RAI 27%] and serious infections [RAI 16%]. To reduce abdominal pain from severe to moderate/mild, patients accepted an additional 18.8% or 30.6% risk of mild-to-moderate SEs, respectively. While average preferences between patients with CD and UC were similar, patients with CD placed greater importance on abdominal pain [p < 0.05], and patients with UC on bowel urgency [p < 0.05]. However, preferences varied notably. CONCLUSIONS: While avoiding abdominal pain, SEs and serious infections had on average the highest treatment priority, preferences varied between patients. Treatment strategies should consider the trade-offs individuals are willing to make.

Preferences of patients for benefits and risks of insomnia medications using data elicited during two phase III clinical trials.

STUDY OBJECTIVES: To elicit the trade-offs patients are willing to make between benefits and risks of medications for chronic insomnia, with the purpose of allowing a patient-centric interpretation of clinical trial data. METHODS: A discrete choice experiment (DCE) was included in the two placebo-controlled phase III trials that evaluated the efficacy and safety of daridorexant. The DCE design was informed by a two-phase qualitative study, followed by qualitative and quantitative pilot testing before fielding. Relative attribute importance (RAI) and acceptable trade-offs between benefits and risks were obtained using a mixed logit model. RESULTS: Preferences were elicited from 602 trial participants (68.1% female, aged 58.6 ± 14.5 years). Preferences were most affected by daytime functioning (RAI = 33.7%) as a treatment benefit and withdrawal symptoms (RAI = 27.5%) as a risk. Patients also valued shorter sleep onset (RAI = 6.4%), longer sleep maintenance (RAI = 5.4%), reduced likelihood of abnormal thoughts and behavioral changes (RAI = 11.3%), reduced likelihood of dizziness/grogginess (RAI = 9.2%), and reduced likelihood of falls at night (RAI = 6.5%). Patients were willing to make trade-offs between these attributes. For example, they would accept an additional 18.8% risk of abnormal thoughts and behavioral changes to improve their daytime functioning from difficult to restricted and an additional 8.1% risk of abnormal thoughts and behavioral changes to avoid moderate withdrawal effects. CONCLUSIONS: Patients with insomnia were willing to make trade-offs between multiple benefits and risks of pharmacological treatments. Because patients valued daytime functioning more than sleep latency and duration, we recommend that functional outcomes and sleep quality be considered in treatment development and evaluation.

Biophysical aspects underlying the swarm to biofilm transition.

Bacteria organize in a variety of collective states, from swarming-rapid surface exploration, to biofilms-highly dense immobile communities attributed to stress resistance. It has been suggested that biofilm and swarming are oppositely controlled, making this transition particularly interesting for understanding the ability of bacterial colonies to adapt to challenging environments. Here, the swarm to biofilm transition is studied in Bacillus subtilis by analyzing the bacterial dynamics both on the individual and collective scales. We show that both biological and physical processes facilitate the transition. A few individual cells that initiate the biofilm program cause nucleation of large, approximately scale-free, stationary aggregates of trapped swarm cells. Around aggregates, cells continue swarming almost unobstructed, while inside, trapped cells are added to the biofilm. While our experimental findings rule out previously suggested purely physical effects as a trigger for biofilm formation, they show how physical processes, such as clustering and jamming, accelerate biofilm formation.

Accounting for Preference Heterogeneity in Discrete-Choice Experiments: An ISPOR Special Interest Group Report.

OBJECTIVES: Discrete choice experiments (DCEs) are increasingly used to elicit preferences for health and healthcare. Although many applications assume preferences are homogenous, there is a growing portfolio of methods to understand both explained (because of observed factors) and unexplained (latent) heterogeneity. Nevertheless, the selection of analytical methods can be challenging and little guidance is available. This study aimed to determine the state of practice in accounting for preference heterogeneity in the analysis of health-related DCEs, including the views and experiences of health preference researchers and an overview of the tools that are commonly used to elicit preferences. METHODS: An online survey was developed and distributed among health preference researchers and nonhealth method experts, and a systematic review of the DCE literature in health was undertaken to explore the analytical methods used and summarize trends. RESULTS: Most respondents (n = 59 of 70, 84%) agreed that accounting for preference heterogeneity provides a richer understanding of the data. Nevertheless, there was disagreement on how to account for heterogeneity; most (n = 60, 85%) stated that more guidance was needed. Notably, the majority (n = 41, 58%) raised concern about the increasing complexity of analytical methods. Of the 342 studies included in the review, half (n = 175, 51%) used a mixed logit with continuous distributions for the parameters, and a third (n = 110, 32%) used a latent class model. CONCLUSIONS: Although there is agreement about the importance of accounting for preference heterogeneity, there are noticeable disagreements and concerns about best practices, resulting in a clear need for further analytical guidance.

Patient and Physician Preferences for Regimen Attributes for the Treatment of HIV in the United States and Canada.

A long-acting injectable (LAI) antiretroviral therapy (ART) regimen is now available as a treatment option for virologically suppressed adults with HIV-1. This study assessed preference for a LAI regimen using an online survey of virally suppressed people living with HIV (PLWH) and physicians treating HIV in the US and Canada. Preference was elicited in a discrete choice experiment (DCE) with three choice options (switch to a LAI regimen, switch to another daily oral ART regimen, or stay on their current daily oral ART regimen) and four treatment attributes. A total of 553 PLWH and 450 physicians completed the survey. From the DCE results, 59% of PLWH were predicted to prefer a LAI over an alternative oral ART or staying on their current oral treatment, and 55-66% of physicians were predicted to recommend LAI for PLWH, depending on the treatment challenge scenario presented. PLWH indicated LAI would remove daily reminders of HIV (75%) and reduce feelings of being stigmatized (68%). A majority of PLWH and physicians preferred a LAI over oral ART to overcome treatment challenges such as daily pill burden and adherence. These benefits of LAI ART along with preferences of PLWH and physicians can help to inform ART choice.

Colorectal cancer screening preferences among physicians and individuals at average risk: A discrete choice experiment.

BACKGROUND: Guidelines include several options for average-risk colorectal cancer (CRC) screening that vary in aspects such as invasiveness, recommended frequency, and precision. Thus, patient and provider preferences can help identify an appropriate screening strategy. This study elicited CRC screening preferences of physicians and individuals at average risk for CRC (IAR). METHODS: IAR aged 45-75 years and licensed physicians (primary care or gastroenterology) completed an online discrete choice experiment (DCE). Participants were recruited from representative access panels in the US. Within the DCE, participants traded off preferences between screening type, screening frequency, true-positive, true-negative, and adenoma true positive (physicians only). A mixed logit model was used to obtain predicted choice probabilities for colonoscopy, multi-target stool DNA (mt-sDNA), fecal immunochemical test (FIT), and methylated septin 9 (mSEPT9) blood test. RESULTS: Preferences of IAR and physicians were affected by screening precision and screening type. IAR also valued more regular screening. Physicians preferred colonoscopy (96.8%) over mt-sDNA (2.8%; p < 0.001), FIT (0.3%; p < 0.001) and mSEPT9 blood test (0.1%; p < 0.01). IAR preferred mt-sDNA (38.8%) over colonoscopy (32.5%; p < 0.001), FIT (19.2%; p < 0.001), and mSEPT9 blood test (9.4%; p < 0.001). IAR naïve to screening preferred non-invasive screening (p < 0.001), while the opposite was found for those who previously underwent colonoscopy or sigmoidoscopy. CONCLUSIONS: While physicians overwhelmingly preferred colonoscopy, preferences of IAR were heterogenous, with mt-sDNA being most frequently preferred on average. Offering choices in addition to colonoscopy could improve CRC screening uptake among IAR. This study used a discrete choice experiment in the US to elicit preferences of physicians and individuals at average risk for colorectal cancer screening modalities and their characteristics.

Patient preferences for the treatment of systemic sclerosis-associated interstitial lung disease: a discrete choice experiment.

OBJECTIVES: Treatments for SSc-associated interstitial lung disease (SSc-ILD) differ in attributes, i.e. mode of administration, adverse events (AEs) and efficacy. As physicians and patients may perceive treatments differently, shared decision-making can be essential for optimal treatment provision. We therefore aimed to quantify patient preferences for different treatment attributes. METHODS: Seven SSc-ILD attributes were identified from mixed-methods research and clinician input: mode of administration, shortness of breath, skin tightness, cough, tiredness, risk of gastrointestinal AEs (GI-AEs) and risk of serious and non-serious infections. Patients with SSc-ILD completed an online discrete choice experiment (DCE) in which they were asked to repeatedly choose between two alternatives characterized by varying severity levels of the included attributes. The data were analysed using a multinomial logit model; relative attribute importance and maximum acceptable risk measures were calculated. RESULTS: Overall, 231 patients with SSc-ILD completed the DCE. Patients preferred twice-daily oral treatments and 6-12 monthly infusions. Patients' choices were mostly influenced by the risk of GI-AEs or infections. Improvement was more important in respiratory symptoms than in skin tightness. Concerning trade-offs, patients accepted different levels of increase in GI-AE risk: +21% if it reduced the infusions' frequency; +15% if changing to an oral treatment; up to +37% if it improved breathlessness; and up to +36% if it reduced the risk of infections. CONCLUSIONS: This is the first study to quantitatively elicit patients' preferences for treatment attributes in SSc-ILD. Patients showed willingness to make trade-offs, providing a firm basis for shared decision-making in clinical practice.

Ising-like Critical Behavior of Vortex Lattices in an Active Fluid.

Turbulent vortex structures emerging in bacterial active fluids can be organized into regular vortex lattices by weak geometrical constraints such as obstacles. Here we show, using a continuum-theoretical approach, that the formation and destruction of these patterns exhibit features of a continuous second-order equilibrium phase transition, including long-range correlations, divergent susceptibility, and critical slowing down. The emerging vorticity field can be mapped onto a two-dimensional (2D) Ising model with antiferromagnetic nearest-neighbor interactions by coarse graining. The resulting effective temperature is found to be proportional to the strength of the nonlinear advection in the continuum model.

Continuing the sequence? Towards an economic evaluation of whole genome sequencing for the diagnosis of rare diseases in Scotland.

Novel developments in genomic medicine may reduce the length of the diagnostic odyssey for patients with rare diseases. Health providers must thus decide whether to offer genome sequencing for the diagnosis of rare conditions in a routine clinical setting. We estimated the costs of singleton standard genetic testing and trio-based whole genome sequencing (WGS), in the context of the Scottish Genomes Partnership (SGP) study. We also explored what users value about genomic sequencing. Insights from the costing and value assessments will inform a subsequent economic evaluation of genomic medicine in Scotland. An average cost of £1,841 per singleton was estimated for the standard genetic testing pathway, with significant variability between phenotypes. WGS cost £6625 per family trio, but this estimate reflects the use of WGS during the SGP project and large cost savings may be realised if sequencing was scaled up. Patients and families valued (i) the chance of receiving a diagnosis (and the peace of mind and closure that brings); (ii) the information provided by WGS (including implications for family planning and secondary findings); and (iii) contributions to future research. Our costings will be updated to address limitations of the current study for incorporation in budget impact modelling and cost-effectiveness analysis (cost per diagnostic yield). Our insights into the benefits of WGS will guide the development of a discrete choice experiment valuation study. This will inform a user-perspective cost-benefit analysis of genome-wide sequencing, accounting for the broader non-health outcomes. Taken together, our research will inform the long-term strategic development of NHS Scotland clinical genetics testing services, and will be of benefit to others seeking to undertake similar evaluations in different contexts.