Patient-Focused Drug Development Methods for Benefit-Risk Assessments: A Case Study Using a Discrete Choice Experiment for Antiepileptic Drugs.
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SUBMITTER: Holmes EAF
PROVIDER: S-EPMC6491963 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:To conduct the benefit-risk assessment of 3-hydroxy-3-methyl-glutaryl (HMG) coenzyme A reductase inhibitors (statins) using a discrete choice experiment, based on 3 major stakeholders' perspectives including patients, experts and policymakers in Thailand.A discrete choice experiment questionnaire survey in three stakeholders' perspectives.Public hospitals in Thailand.A total of 353 policymakers, experts and patients.Stakeholders' preferences for assessment criteria (stroke reduction, myocardial infarction reduction, myalgia and hepatotoxicity). Statins' ranking and maximum acceptable risk in all perspectives were also calculated.For any perspective, the most and least important criteria were the risk of hepatotoxicity and the benefit of myocardial infarction reduction, respectively. Patients and experts agreed on the order of importance for myalgia and stroke reduction, but policymakers had different order of importance in these criteria. Overall, results showed that the highest and lowest chances of being chosen were atorvastatin and rosuvastatin, respectively. Only patients' ranking order was different from others. Maximum acceptable risk of hepatotoxicity was lower than that of myalgia, reflecting the greater concern of all perspectives to statin consequence on liver.The results of benefit-risk assessment from every perspective were somewhat consistent. This study demonstrated the feasibility of applying a discrete choice experiment in the benefit-risk assessment of drugs and encouraged the engagement of multiple stakeholders in the decision-making process.
| S-EPMC4769438 | biostudies-other
Project description:Multiple sclerosis is a chronic, progressive, and common disease affecting the central nervous system in young adults. Interferon-beta is one of the most widely used medicines to reduce the disease progression. Given the variety of drugs in this category, we aimed to identify the preferences of patients for IFN-β that play an important role in policymaking in this area. Discrete choice experiment method was used in the present study to identify and prioritize those attributes that are of interest to MS patients and increases the utility of the use of IFN-β in their treatment. Questionnaires were given to 358 patients in Isfahan-Iran, who were asked to choose between the two treatment choices in each scenario. The results of the logit model showed that the changes in the efficacy lead to the most changes in the patient utility. Changes in side effects and ease of injection have been placed in the next rankings. Considering the drug attributes considered more desirable by patients can lead to greater medication adherence and possibly better treatment outcomes. Also, pharmaceutical companies, the health ministry, the Food and Drug Administration, insurance organizations, and neurologists can benefit from this information in production and importation, policymaking, and prescription.
| S-EPMC5873931 | biostudies-literature
Project description:ObjectivesTo elicit and value patient preferences for the processes and outcomes of surgical management of stress urinary incontinence in women.DesignA discrete choice experiment survey to elicit preferences for type of anaesthesia, postoperative recovery time, treatment success, adverse events, impact on daily activities and cost. An experimental design generated 40 choice tasks, and each respondent completed 1 block of 10 and 2 validity tests. Analysis was by multinomial logistical regression.SettingN=21 UK hospitals.ParticipantsN=325 adult women who were a subsample of those randomised to the single-incision mini-slings clinical trial.OutcomesPatient preferences; valuation obtained using willingness to pay.ResultsN=227 of 325 (70%) returned a questionnaire, and 94% of those completed all choice tasks. Respondents preferred general anaesthesia, shorter recovery times, improved stress urinary incontinence symptoms and avoidance of adverse events. Women were willing to pay (mean (95% CI)) £76 (£33 to £119) per day of reduction in recovery time following surgery. They valued increases in Patient Global Impression of Improvement, ranging from £8173 (£5459 to £10 887) for 'improved' to £11 706 (£8267 to £15 144) for 'very much improved' symptoms, compared with no symptom improvement. This was offset by negative values attached to the avoidance of complications ranging between £-8022 (£-10 661 to £-5383) and £-10 632 (£-14 077 to £-7187) compared to no complications. Women valued treatments that reduced the need to avoid daily activities, with willingness to pay ranging from £-967 (£-2199 to £266) for rarely avoiding activities to £-5338 (£-7258 to £-3417) for frequently avoiding daily activities compared with no avoidance.ConclusionThis discrete choice experiment demonstrates that patients place considerable value on improvement in stress urinary incontinence symptoms and avoidance of treatment complications. Trade-offs between symptom improvement and adverse event risk should be considered within shared decision-making. The willingness to pay values from this study can be used in future cost-benefit analyses.Trial registration numberISRCTN: 93264234; Post-results.
| S-EPMC10465896 | biostudies-literature
Project description:BackgroundDiscrete choice experiments (DCEs) are increasingly used to elicit preferences for benefit-risk tradeoffs. The primary aim of this study was to explore how eye-tracking methods can be used to understand DCE respondents' decision-making strategies. A secondary aim was to explore if the presentation and communication of risk affected respondents' choices.MethodTwo versions of a DCE were designed to understand the preferences of female members of the public for breast screening that varied in how risk attributes were presented. Risk was communicated as either 1) percentages or 2) icon arrays and percentages. Eye-tracking equipment recorded eye movements 1000 times a second. A debriefing survey collected sociodemographics and self-reported attribute nonattendance (ANA) data. A heteroskedastic conditional logit model analyzed DCE data. Eye-tracking data on pupil size, direction of motion, and total visual attention (dwell time) to predefined areas of interest were analyzed using ordinary least squares regressions.ResultsForty women completed the DCE with eye-tracking. There was no statistically significant difference in attention (fixations) to attributes between the risk communication formats. Respondents completing either version of the DCE with the alternatives presented in columns made more horizontal (left-right) saccades than vertical (up-down). Eye-tracking data confirmed self-reported ANA to the risk attributes with a 40% reduction in mean dwell time to the "probability of detecting a cancer" ( P = 0.001) and a 25% reduction to the "risk of unnecessary follow-up" ( P = 0.008).ConclusionThis study is one of the first to show how eye-tracking can be used to understand responses to a health care DCE and highlighted the potential impact of risk communication on respondents' decision-making strategies. The results suggested self-reported ANA to cost attributes may not be reliable.
| S-EPMC6088456 | biostudies-literature
Project description:BACKGROUND:Economic evidence relating to tobacco control is generally derived from the cost effectiveness of smoking-cessation programs or the economic impact of tobacco-induced disease, based on revealed-preference data. However, empirical estimates from stated-preference data on tobacco users' preferences, smoking behaviour and smoking cessation aids using analytical techniques such as discrete-choice experiments (DCEs) could be important for policy decision making in tobacco control. OBJECTIVES:Our objective was to review the practice and utility of DCE methodology across nicotine- and tobacco-related issues, particularly smoking and smoking-cessation behaviour, anti-smoking policies and preferences for smoking-cessation aids. METHODS:We searched the PubMed, MEDLINE and ECONLIT databases for full-text original research articles on tobacco-related issues published between January 2000 and April 2016 that used a DCE method. We summarised the evidence and methodological characteristics of DCEs according to Lancsar and Louviere, 2008. RESULTS:Our review of the 12 eligible studies showed that DCE methodology was used to elicit smoker preferences and to evaluate tobacco-control policies. The majority of the studies were published in the last 5 years. The areas of application were smoking cessation, smoking behaviour, electronic cigarette use, water-pipe smoking and tobacco packaging. Monetary attributes were the most influential attributes in all studies. The design of the DCEs varied. CONCLUSION:DCE studies of tobacco-related issues were methodologically consistent with guidelines proposed for conducting health-related DCEs.
| S-EPMC5820233 | biostudies-literature
Project description:OBJECTIVE:To quantify patient preferences for maintenance therapy of Crohn's disease and understand the impact on treatment selection. METHODS:We conducted a discrete-choice experiment in patients with Crohn's disease (n = 155) to measure the importance of attributes relevant to choosing between different medical therapies for maintenance of Crohn's disease. The attributes included efficacy and withdrawals due to adverse events, as well as dosing and other rare risks of treatment. From the discrete-choice experiment we estimated the part-worth (importance) of each attribute level, and explored preference heterogeneity through latent class analysis. We then used the part-worths to apply weights across each outcome from a prior network meta-analysis to estimate patients' preferred treatment in pairwise comparisons and for the overall group of treatments. RESULTS:The discrete-choice experiment revealed that maintaining remission was the most important attribute. Patients would accept a rare risk of infection or cancer for a 14% absolute increased chance of remission. Latent class analysis demonstrated that 45% of the cohort was risk averse, either to adverse events or requiring a course of prednisone. When these preferences were used in modelling studies to compare pairs of treatments, there was a ? 78% probability that all biologic treatments were preferred to azathioprine and methotrexate, based on the balance of benefits and harms. When comparing all treatments, adalimumab was preferred by 53% of patients, who were motivated by efficacy, and vedolizumab was preferred by 30% who were driven by the preference to avoid risks. However, amongst biologic treatment options, there was considerable uncertainty regarding the preferred treatment at the individual patient level. CONCLUSION:Patients with Crohn's disease from our population were, on average, focused on the benefits of treatment, supporting intensive treatment approaches aimed at maintaining remission. Important preference heterogeneity was identified, however, highlighting the importance of shared decision making when selecting treatments.
| S-EPMC6964885 | biostudies-literature
Project description:IntroductionTreatment options for Waldenström's Macroglobulinemia (WM) have expanded rapidly in the last decades. However, there is no consensus on a preferred treatment. Therefore, patient preferences become increasingly important in making individualized treatment plans. Still, WM patients' priorities and perspectives regarding their treatment options are unknown. We evaluated treatment preferences of WM patients using a discrete choice experiment (DCE).MethodsA mixed-method approach was utilized for identification and selection of attributes/levels. The DCE questionnaire included five attributes: type of agent (targeted versus chemotherapy); frequency and route of administration; 5-year progression-free survival (PFS); adverse events; and risk of secondary malignancies. An orthogonal design and a mixed logit panel data model were used to construct choice tasks and assess patient preferences, respectively.ResultsThree hundred thirty WM patients participated in the project. In total, 214 (65%) complete questionnaires were included for data analysis. The 5-year PFS, followed by risk of secondary malignancies were the most important attributes for making treatment choices. Regarding side effects, patients chose to avoid neuropathy the most compared to nausea/vomiting and extreme fatigue. Patients preferred a fixed-duration treatment with IV/SC administration at the hospital over a continuous daily oral regimen at home.ConclusionThese are the first systematic data obtained on WM patient preferences for treatment. The results may help discussions with individual patients about their treatment choices. Also, these data can help design clinical trials in WM and inform health-care decision-making regarding outcomes that are most relevant to patients.
| S-EPMC9939214 | biostudies-literature
Project description:OBJECTIVE:Limited evidence for the optimal venous thromboembolism (VTE) prophylaxis regimen in orthopaedic trauma leads to variability in regimens. We sought to delineate patient preferences towards cost, complication profile, and administration route (oral tablet vs. subcutaneous injection). DESIGN:Discrete choice experiment (DCE). SETTING:Level 1 trauma center in Baltimore, USA. PARTICIPANTS:232 adult trauma patients (mean age 47.9 years) with pelvic or acetabular fractures or operative extremity fractures. PRIMARY AND SECONDARY OUTCOME MEASURES:Relative preferences and trade-off estimates for a 1% reduction in complications were estimated using multinomial logit modelling. Interaction terms were added to the model to assess heterogeneity in preferences. RESULTS:Patients preferred oral tablets over subcutaneous injections (marginal utility, 0.16; 95% CI: 0.11 - 0.21, P<0.0001). Preferences changed in favor of subcutaneous injections with an absolute risk reduction of 6.98% in bleeding, 4.53% in wound complications requiring reoperation, 1.27% in VTE, and 0.07% in death from pulmonary embolism (PE). Patient characteristics (sex, race, type of injury, time since injury) affected patient preferences (P<0.01). CONCLUSIONS:Patients preferred oral prophylaxis and were most concerned about risk of death from PE. Furthermore, the findings estimated the trade-offs acceptable to patients and heterogeneity in preferences for VTE prophylaxis.
| S-EPMC5629686 | biostudies-other
Project description:Objectives: While several authors have suggested using a multi-criteria approach for orphan drug assessment and proposed lists of determinants of orphan drug value, studies on social preferences regarding these determinants remain limited. The current study aimed to identify preferences of the French general population regarding attributes characterizing the value of orphan drugs in a discrete choice experiment. Methods: The list of attributes was formed based on a literature search and was refined through expert interviews, a focus group, and a pilot study. The final list included nine attributes: disease-associated disability, disease-associated mortality, number of patients, availability of alternative treatments, treatment impact on disease disability, treatment impact on mortality, treatment safety, uncertainty around therapeutic effect, and annual treatment cost per patient. Members of the General Public were presented with 12 choice sets containing two drug profiles described according to the attributes and an option to fund neither of these treatments. The questionnaire was disseminated online. A conditional logit model with random effects was used to estimate the weight of each attribute. Results: A total of 958 persons participated in the study (48.7% male, mean age: 47.5 years). All attributes except for disease-associated disability had a statistically significant influence on the choices made by participants. The attribute with the highest weight was treatment impact on mortality (p < 0.0001), followed by uncertainty around therapeutic effect (p < 0.0001). The direction of results was generally consistent with intuition: patients preferred a drug with a larger impact on mortality, a larger impact on disability, with mild or no adverse events, with less uncertainty. Although patients appeared to prefer drugs with a lower budget impact, the relationship between patient preferences and costs was more complex. Conclusions: Preferences of the general public between orphan drugs are mostly driven by the impact on mortality and the degree of certainty regarding the available evidence.
| S-EPMC7379418 | biostudies-literature
Project description:ObjectiveVitamin D is often prescribed as an adjuvant therapy to aid fracture healing due to its biological role in bone health. However, the optimal frequency, dosage and duration of vitamin D supplementation for non-osteoporotic fracture healing has not been established. The objective of this study was to determine patient preferences for fracture healing relative to hypothetical vitamin D supplementation dosing options.DesignDiscrete choice experiment.SettingLevel 1 trauma centre in Baltimore, Maryland, USA.Participants199 adult (18-60 years) patients with a fracture.Primary outcome measuresParameter estimates of utility for fracture healing relative to dosing regimens were analysed using hierarchical Bayesian modelling.ResultsA reduced risk of reoperation (34.3%) and reduced healing time (24.4%) were the attributes of greatest relative importance. The highest mean utility estimates were for a one-time supplementation dose (ß=0.71, 95% CI 0.41 to 1.00) followed by a reduced risk of reoperation (ß=0.41 per absolute % reduction, 95% CI 0.0.36 to 0.46). Supplementation for 24 weeks in duration (ß=-0.83, 95% CI -1.00 to -0.67) and a daily supplement (ß=-0.29, 95% CI -0.47 to -0.11) had the lowest mean utilities. The 'no supplement' option had a large negative value suggesting supplementation was generally desirable in this sample population. Among other possible clinical scenarios, patients expected a 2% reduction in the absolute risk of reoperation or a 3.1-week reduction in healing time from the baseline to accept a treatment regimen requiring two separate doses of supplementation, two blood tests and a cost of $20 within 3?months of injury.ConclusionsPatients with orthopaedic trauma demonstrated strong willingness to take a vitamin D supplement that would decrease risk of reoperation and reduce healing time. Furthermore, these findings specify the required decrease in reoperation risk and reduction in healing time patients would expect to adhere to possible vitamin D dosing regimens.
| S-EPMC5898332 | biostudies-literature