Making Use of Technology to Improve Stated Preference Studies.

The interest in quantifying stated preferences for health and healthcare continues to grow, as does the technology available to support and improve health preference studies. Technological advancements in the last two decades have implications and opportunities for preference researchers designing, administering, analysing, interpreting and applying the results of stated preference surveys. In this paper, we summarise selected technologies and how these can benefit a preference study. We discuss empirical evaluations of the technology in preference research, with examples from health where possible. The technologies reviewed include serious games, virtual reality, eye tracking, innovative formats and decision aids with values clarification components. We conclude with a critical reflection on the benefits and limitations of implementing (often costly) technology alongside stated preference studies.

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.

Discrete Choice Experiment to Understand Japanese Patients' and Physicians' Preferences for Preventive Treatments for Migraine.

INTRODUCTION: Self-injectable calcitonin gene-related peptide (CGRP) monoclonal antibody (mAb) auto-injectors and non-CGRP oral medications are currently available for migraine prevention in Japan. This study elicited the preferences for self-injectable CGRP mAbs and non-CGRP oral medications and determined differences in the relative importance of auto-injector attributes for patients and physicians in Japan. METHODS: Japanese adults with episodic (EM) or chronic (CM) migraine and physicians who treat migraine completed an online discrete choice experiment (DCE), asking participants to choose a hypothetical treatment they preferred between two self-injectable CGRP mAb auto-injectors and a non-CGRP oral medication. The treatments were described by seven treatment attributes, with attribute levels varying between questions. DCE data were analyzed using a random-constant logit model to estimate relative attribution importance (RAI) scores and predicted choice probabilities (PCP) of CGRP mAb profiles. RESULTS: A total of 601 patients (79.2% with EM, 60.1% female, mean age: 40.3 years) and 219 physicians (mean length of practice: 18.3 years) completed the DCE. About half (50.5%) of patients favored CGRP mAb auto-injectors, while others were skeptical of (20.2%) or averse (29.3%) to them. Patients most valued needle removal (RAI = 33.8%), shorter injection duration (RAI = 32.1%), and auto-injector base shape and need for skin pinching (RAI = 23.2%). Most physicians (87.8%) favored auto-injectors over non-CGRP oral medications. Physicians most valued less-frequent dosing RAI = 32.7%), shorter injection duration (30.4%), and longer storage outside the fridge (RAI = 20.3%). A profile comparable to galcanezumab showed a higher likelihood of being chosen by patients (PCP = 42.8%) than profiles comparable to erenumab (PCP = 28.4%) and fremanezumab (PCP = 28.8%). The PCPs of the three profiles were similar among physicians. CONCLUSION: Many patients and physicians preferred CGRP mAb auto-injectors over non-CGRP oral medications and preferred a treatment profile similar to galcanezumab. Our results may encourage physicians in Japan to consider patient preferences when recommending migraine preventive treatments.

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.

Patient Perspectives and Experiences of Preventive Treatments and Self-Injectable Devices for Migraine: A Focus Group Study.

BACKGROUND: Although several self-injectable preventive treatments for migraine have become available, they are not yet widely used. Thus, understanding patients' perceptions towards them is limited. OBJECTIVE: This study aimed to inform the design of a preference-elicitation instrument, which is being developed to quantify preventive treatment preferences of people with migraine. METHODS: We conducted a qualitative study involving nine in-person focus groups (three per country) in the United States, the United Kingdom, and Germany. Participants were adults (n = 47) with episodic or chronic migraine who were currently using or had used a prescription preventive treatment for migraine within the previous 5 years. During the focus groups, participants described their experiences of migraine and preventive treatments; handled and simulated self-injection using five different unbranded, fired demonstration auto-injectors and prefilled syringes; and ranked different aspects of preventive treatments by importance. Focus groups were analyzed with a focus on themes that would be feasible or meaningful to include in a subsequent preference-elicitation instrument. RESULTS: Reducing the frequency and severity of migraine attacks was consistently ranked as the most important aspect of preventive treatment. Participants expressed dissatisfaction with available daily oral preventive treatments for migraine they had previously used because they were ineffective or caused intolerable adverse events. Many participants were willing to self-inject a treatment that was effective and tolerable. When presented with devices for self-injecting a preventive treatment for migraine, participants generally preferred autoinjectors over prefilled syringes. Participants especially valued safety features such as the unlocking step and automated needle insertion, and audible and visual dose confirmation increased confidence in autoinjector use. Autoinjector needle protection mechanisms were also appreciated, especially by participants averse to needles, as the needles are not visible. CONCLUSIONS: This study highlights the fact that many people with migraine still lack access to a preventive treatment that is effective and tolerable. In addition to efficacy and safety considerations, treatment decisions may be guided by the mode of administration. In the case of self-injectable preventive treatments, key device characteristics affecting these decisions may be ease of use, comfort, and confidence in self-injection. Insights gained from this study were used to help develop a preliminary set of attributes and levels for a preference-elicitation instrument.

Patient Preferences of Low-Dose Aspirin for Cardiovascular Disease and Colorectal Cancer Prevention in Italy: A Latent Class Analysis.

BACKGROUND: Patients taking low-dose aspirin to prevent cardiovascular disease (CVD) may also benefit from a reduced risk of colorectal cancer (CRC). OBJECTIVE: The aim was to examine the preferences of people eligible for preventive treatment with low-dose aspirin and the trade-offs they are willing to make between CVD prevention, CRC prevention, and treatment risks. METHODS: A cross-sectional study using a discrete choice experiment (DCE) survey was conducted in Italy in 2019 to elicit preferences for three benefit attributes (prevention of ischemic stroke, myocardial infarction, and CRC) and four risk attributes (intracranial and gastrointestinal bleeding, peptic ulcer, and severe allergic reaction) associated with use of low-dose aspirin. Latent class logit models were used to evaluate variation in treatment preferences. RESULTS: The DCE survey was completed by 1005 participants eligible for use of low-dose aspirin. A four-class model had the best fit for the primary CVD prevention group (n = 491), and a three-class model had the best fit for the secondary CVD prevention group (n = 514). For the primary CVD prevention group, where classes differed on age, education level, type 2 diabetes, exercise, and low-dose aspirin use, the most important attributes were intracranial bleeding (two classes), myocardial infarction (one class), and CRC (one class). For the secondary CVD prevention group, where classes differed on various comorbidities, self-reported health, exercise, and CVD medication use, the most important attributes were intracranial bleeding (two classes), myocardial infarction (one class), and gastrointestinal bleeding (one class). CONCLUSION: Patient preferences for the benefits and risks of low-dose aspirin differ significantly among people eligible for treatment as primary or secondary CVD prevention.

Benefit-Risk or Risk-Benefit Trade-Offs? Another Look at Attribute Ordering Effects in a Pilot Choice Experiment.

BACKGROUND: Studies recommend randomising the order of attributes in discrete choice experiments (DCEs) to avoid bias; however, in a benefit-risk setting, this may increase the cognitive burden of respondents who compare the benefits and risks of treatments, or may affect their decision-making process. Based on these concerns, this paper explored attribute ordering effects in a benefit-risk DCE. METHOD: Attribute ordering effects were explored in a large pilot DCE relating to the medical treatment of insomnia. Participants were randomised to one of three presentation orders: (1) benefits were presented before risks (BR); (2) risks were presented before benefits (RB); (3) all attributes were randomised (RN). For the RB and BR presentation orders, attributes were randomised within benefits and risks. Responses were assessed in three ways. First, variations in respondents' self-reported choice certainty were obtained. Second, variations in failure rates of stability and dominance tests were calculated. Third, a heteroscedastic error component model tested for differences in choice consistency across the three attribute orderings. RESULTS: The final analysis included 156 respondents (RN: 54; BR: 49; RB: 53). No differences were found between the presentation orders with respect to stated choice certainty, or the proportion of respondents failing either the dominance or stability test. However, deterministic attribute grouping was associated with higher choice consistency. CONCLUSION: To increase choice consistency, DCE attributes should be randomised within logical groups that may be further randomised to reduce the risk of ordering effects.

Age at Diagnosis and Patient Preferences for Treatment Outcomes in AML: A Discrete Choice Experiment to Explore Meaningful Benefits.

BACKGROUND: The recent expansion of treatment options in acute myeloid leukemia (AML) has necessitated a greater understanding of patient preferences for treatment benefits, about which little is known. METHODS: We sought to quantify and assess heterogeneity of the preferences of AML patients for treatment outcomes. An AML-specific discrete choice experiment (DCE) was developed involving multiple stakeholders. Attributes included in the DCE were event-free survival (EFS), complete remission (CR), time in the hospital, short-term side effects, and long-term side effects. Continuously coded conditional, stratified, and latent-class logistic regressions were used to model preferences of 294 patients with AML. RESULTS: Most patients were white (89.4%) and in remission (95.0%). A 10% improvement in the chance of CR was the most meaningful offered benefit (P < 0.001). Patients were willing to trade up to 22 months of EFS or endure 8.7 months in the hospital or a two-step increase in long-term side effects to gain a 10% increase in chance of CR. Patients diagnosed at 60 years or older (21.6%) more strongly preferred to avoid short-term side effects (P = 0.03). Latent class analysis showed significant differences of preferences across gender and insurance status. CONCLUSIONS: In this national sample of mostly AML survivors, patients preferred treatments that maximized chance at remission; however, significant preference heterogeneity for outcomes was identified. Age and gender may affect patients' preferences. IMPACT: Survivor preferences for outcomes can inform patient-focused drug development and shared decision-making. Further studies are necessary to investigate the use of DCEs to guide treatment for individual patients.

Developing an instrument to assess patient preferences for benefits and risks of treating acute myeloid leukemia to promote patient-focused drug development.

Objective Acute myeloid leukemia (AML) is a progressive blood cancer with few effective treatment options. As part of a patient-focused drug development (PFDD) initiative led by the Leukemia and Lymphoma Society (LLS), this study sought to use a community-centered approach to develop and pilot an instrument to measure patient preferences for the benefits and risks of treating AML. Methods Instrument development was informed by a literature review, engagement with expert stakeholders (n = 12), engagement with community stakeholders, and pre-testing. A discrete-choice experiment (DCE), in which participants made choices between 16 pairs of hypothetical treatments, was developed with five attributes: event-free survival, complete remission, time in hospital, short-term side-effects, and long-term side-effects. A pilot test was conducted and analyzed using conditional logistic regression. Results are presented using relative attribute importance (RAI) scores. Results Patients with AML and caregivers were engaged in developing (n = 15), pre-testing (n = 13), and pilot testing (n = 26) the instrument. The pilot included patients with AML (n = 18) and caregivers of living or deceased patients with AML (n = 8). Participants had a mean age of 50 years (range =24-81), and were mostly college educated (n = 22), privately insured (n = 21), and employed (n = 13). Based on the DCE, complete remission was identified as the most important attribute (RAI =10), followed by event-free survival (3.7), time in hospital (2.8), long-term side-effects (2.3), and short-term side-effects (2.1). Conclusion The mixed-methods approach to PFDD was welcomed by all stakeholders and there was strong endorsement to implement this DCE as part of a national survey. Key points for decision makers The Leukemia and Lymphoma Society (LLS) initiated an independent effort to promote patient-focused drug development (PFDD). This study presents the development and piloting of a preference study as a first step in this initiative. Results of this pilot study were used to guide a PFDD meeting to discuss the lived experience of patients and caregivers affected by AML. Productive engagement by all patients, caregivers, and stakeholders throughout the process resulted in strong endorsement of the project's approach and recognition of the need to conduct a national study.

The unknown burden and cost of celiac disease in the U.S.

INTRODUCTION: Celiac disease is an autoimmune disease that results from exposure to gluten in genetically susceptible individuals and leads to a range of gastrointestinal and extraintestinal symptoms. Areas covered: In order to evaluate the literature with respect to burden associated with celiac disease in the U.S. and identify any knowledge gaps, we performed a literature review of journal articles published between 2000-2016. We note that celiac disease is a prevalent condition associated with a significant burden of disease through its impact on morbidity, quality of life, as well as through increased costs associated with its diagnosis and management. At the same time, knowledge gaps exist in our understanding of the precise epidemiologic burden in the U.S.; the trade-offs between burden and benefit of a gluten-free diet; and better estimation of the costs of diagnosis, treatment and management.Expert commentary: Additional research is necessary to better understand these gaps to be able to reduce burden of celiac disease, particularly the impact on health-related quality of life and the costs associated with inaccurate or delayed diagnoses and insufficient treatment of disease.