Preferences of people living with HIV for injectable and oral antiretroviral treatment in the Netherlands: a discrete choice experiment.

ABSTRACTInjectable antiretroviral treatment (ART) represents a new effective and potentially more convenient alternative to oral ART for people living with HIV (PLWH). This study assessed preferences of PLWH for long-acting injectable compared with oral ART in the Netherlands. A labelled discrete choice experiment presented 12 choice sets of long-acting injectable and oral ART. PLWH were asked to select their preferred ART, described by six attributes: location of administration, dosing frequency, risk of short-term side effects, drug-drug interaction, forgivability, and food and mealtime restrictions. Random parameters logit and latent class models were used to estimate preferences of PLWH. 98.6% of 76 respondents were experienced oral ART users that had taken ART for a median of 12 years (Q1-Q3: 7.0-20.0). 30 (39.5%) respondents chose long-acting injectable ART in all choice tasks and 22 (28.9%) always chose oral ART. The random parameter model showed that, on average, respondents significantly favoured long-acting injectable ART over oral ART, preferred administration of the long-acting injectable ART at home, and a less frequent regimen. The latent class model confirmed one class strongly preferring long-acting injectable ART and one class slightly preferring oral ART. This study highlights the value for both long-acting injectable and oral ART.

Exploring the Cost Effectiveness of Shared Decision Making for Choosing between Disease-Modifying Drugs for Relapsing-Remitting Multiple Sclerosis in the Netherlands: A State Transition Model.

BACKGROUND: Up to 31% of patients with relapsing-remitting multiple sclerosis (RRMS) discontinue treatment with disease-modifying drug (DMD) within the first year, and of the patients who do continue, about 40% are nonadherent. Shared decision making may decrease nonadherence and discontinuation rates, but evidence in the context of RRMS is limited. Shared decision making may, however, come at additional costs. This study aimed to explore the potential cost-effectiveness of shared decision making for RRMS in comparison with usual care, from a (limited) societal perspective over a lifetime. METHODS: An exploratory economic evaluation was conducted by adapting a previously developed state transition model that evaluates the cost-effectiveness of a range of DMDs for RRMS in comparison with the best supportive care. Three potential effects of shared decision making were explored: 1) a change in the initial DMD chosen, 2) a decrease in the patient's discontinuation in using the DMD, and 3) an increase in adherence to the DMD. One-way and probabilistic sensitivity analyses of a scenario that combined the 3 effects were conducted. RESULTS: Each effect separately and the 3 effects combined resulted in higher quality-adjusted life years (QALYs) and costs due to the increased utilization of DMD. A decrease in discontinuation of DMDs influenced the incremental cost-effectiveness ratio (ICER) most. The combined scenario resulted in an ICER of €17,875 per QALY gained. The ICER was sensitive to changes in several parameters. CONCLUSION: This study suggests that shared decision making for DMDs could potentially be cost-effective, especially if shared decision making would help to decrease treatment discontinuation. Our results, however, may depend on the assumed effects on treatment choice, persistence, and adherence, which are actually largely unknown.

Comparison of statistical analysis methods for object case best-worst scaling.

Aims: Different methods have been used to analyze "object case" best-worst scaling (BWS). This study aims to compare the most common statistical analysis methods for object case BWS (i.e. the count analysis, multinomial logit, mixed logit, latent class analysis, and hierarchical Bayes estimation) and to analyze their potential advantages and limitations based on an applied example. Methods: Data were analyzed using the five analysis methods. Ranking results were compared among the methods, and methods that take respondent heterogeneity into account were presented specifically. A BWS object case survey with 22 factors was used as a case study, tested among 136 policy-makers and HTA experts from the Netherlands, Germany, France, and the UK to assess the most important barriers to HTA usage. Results: Overall, the five statistical methods yielded similar rankings, particularly in the extreme ends. Latent class analysis identified five clusters and the mixed logit model revealed significant preference heterogeneity for all, with the exception of three factors. Limitations: The variety of software used to analyze BWS data may affect the results. Moreover, this study focuses solely on the comparison of different analysis methods for the BWS object case. Conclusions: The most common statistical methods provide similar rankings of the factors. Therefore, for main preference elicitation, count analysis may be considered as a valid and simple first-choice approach. However, the latent class and mixed logit models reveal additional information: identifying latent segments and/or recognizing respondent heterogeneity.

Comparison of preferences of healthcare professionals and MS patients for attributes of disease-modifying drugs: A best-worst scaling.

BACKGROUND: The choice between disease-modifying drugs (DMDs) for the treatment of multiple sclerosis (MS) becomes more often a shared decision between the patient and the neurologist and MS nurse. This study aimed to assess which DMD attributes are most important for the healthcare professionals in selecting a DMD for a patient. Subsequently, within this perspective, the neurologists' and nurses' perspectives were compared. Lastly, the healthcare professionals' perspective was compared with the patients' perspective to detect any differences that may need attention in the communication about DMDs. DESIGN: A best-worst scaling (BWS) was conducted among 27 neurologists and 33 MS nurses treating patients with MS to determine the importance of 27 DMD attributes. These attributes were identified through three focus groups with MS patients in a previous study (N=19). Relative importance scores (RISs) were estimated for each attribute. Multivariable linear regression analyses were used to compare the different perspectives. RESULTS: According to the neurologists and nurses, safety of the DMD was the most important DMD attribute in the treatment decision, closely followed by effect on disability progression, quality of life and relapse rate. Patients with MS agreed with the importance of the last three attributes, but valued safety significantly lower (b=-2.59, P<.001). CONCLUSIONS: This study suggests that, overall, neurologists and nurses regard the same DMD attributes as important as MS patients with the notable exception of safety. This study provides valuable information for the development of interventions to support shared decision making and highlights which attributes of DMDs may need additional attention.

Adherence to Web-Based Self-Assessments in Long-Term Direct-to-Patient Research: Two-Year Study of Multiple Sclerosis Patients.

BACKGROUND: Direct-to-patient research via Web-based questionnaires is increasingly being used. Missed data or delayed reporting of data may negatively affect the quality of study results. It is insufficiently known to what degree patients adhere to agreed self-assessment schedule over the long term and whether questionnaires are filled out in a timely manner. OBJECTIVE: The objective of this study was to investigate patients' adherence to a self-assessment schedule with low-frequency long questionnaires versus that with a high-frequency short questionnaire. METHODS: In this study, the 36-item MS Impact Profile (MSIP) questionnaire measured (perceived) disabilities and the 54-item MS Quality of Life-54 (MSQoL-54) questionnaire measured health-related quality of life at 6-month intervals. Additionally, the 2-item Medication and Adherence (MA) questionnaire documented medication and adherence to disease-modifying medication every month. An experienced MS nurse assessed the Expanded Disability Status Scale (EDSS) score via phone. For both the self-assessment schedules, we calculated the percentage of patients who had completed all the questionnaires in the first 2 years (completion adherence), the percentage of patients who completed all the questionnaires within set time frames (interval adherence), the relationship between adherence and the EDSS score, and the timing of EDSS assessment. RESULTS: Of the 331 patients who enrolled themselves, 301 patients completed at least one questionnaire. At month six (M6), M12, M18, and M24, the MSIP was completed by 83.4% (251/301), 71.8% (216/301), 68.1% (205/301), and 58.5% (176/301) of the patients, respectively; the MSQoL-54 by 82.1% (247/301), 71.8% (216/301), 66.8% (201/301), and 57.1% (172/301), respectively; and the MA questionnaire by 80.1% (241/301), 70.4% (212/301), 62.1% (187/301), and 53.5% (161/301), respectively. For the MSIP, 56.8% (171/301) of the patients were 2-year completion adherent; 55.5% (167/301) and 53.5% (161/301) of the patients were completion adherent for the MSQoL-54 and MA questionnaires, respectively. Whereas 85.5% (142/166) of the patients were interval adherent for the MSIP and MSQoL-54, 25.5% (41/161) were interval adherent for the MA questionnaire, with 73.9% (119/161) exceeding the maximum MA monthly interassessment interval. Completion adherence for the monthly short MA questionnaire was higher in patients with moderately high disability (EDSS 5.0-5.5) than for those with no or minimal disability (EDSS 0-2.5) (OR 5.47, 95% CI 1.08-27.69; P=.040). Completion adherence was also higher in patients with EDSS assessment within 6 months after baseline than in those with later assessment (OR 1.810, 95% CI 0.999-3.280; P=.050). CONCLUSIONS: The 2-year completion adherence to Web-based self-assessments did not differ between the low-frequency long questionnaires and a high-frequency short questionnaire, but the interval adherence was substantially higher for the low-frequency long questionnaires. Personal contact with a member of the research team regarding a clinically relevant professional-reported outcome early in the study might positively affect the long-term completion adherence in direct-to-patient studies.

Identification and Prioritization of Important Attributes of Disease-Modifying Drugs in Decision Making among Patients with Multiple Sclerosis: A Nominal Group Technique and Best-Worst Scaling.

OBJECTIVES: Understanding the preferences of patients with multiple sclerosis (MS) for disease-modifying drugs and involving these patients in clinical decision making can improve the concordance between medical decisions and patient values and may, subsequently, improve adherence to disease-modifying drugs. This study aims first to identify which characteristics-or attributes-of disease-modifying drugs influence patients´ decisions about these treatments and second to quantify the attributes' relative importance among patients. METHODS: First, three focus groups of relapsing-remitting MS patients were formed to compile a preliminary list of attributes using a nominal group technique. Based on this qualitative research, a survey with several choice tasks (best-worst scaling) was developed to prioritize attributes, asking a larger patient group to choose the most and least important attributes. The attributes' mean relative importance scores (RIS) were calculated. RESULTS: Nineteen patients reported 34 attributes during the focus groups and 185 patients evaluated the importance of the attributes in the survey. The effect on disease progression received the highest RIS (RIS = 9.64, 95% confidence interval: [9.48-9.81]), followed by quality of life (RIS = 9.21 [9.00-9.42]), relapse rate (RIS = 7.76 [7.39-8.13]), severity of side effects (RIS = 7.63 [7.33-7.94]) and relapse severity (RIS = 7.39 [7.06-7.73]). Subgroup analyses showed heterogeneity in preference of patients. For example, side effect-related attributes were statistically more important for patients who had no experience in using disease-modifying drugs compared to experienced patients (p < .001). CONCLUSIONS: This study shows that, on average, patients valued effectiveness and unwanted effects as most important. Clinicians should be aware of the average preferences but also that attributes of disease-modifying drugs are valued differently by different patients. Person-centred clinical decision making would be needed and requires eliciting individual preferences.