The Best of Both Worlds: An Example Mixed Methods Approach to Understand Men’s Preferences for the Treatment of Lower Urinary Tract Symptoms

Ikenwilo, Divine; Heidenreich, Sebastian; Ryan, Mandy; Mankowski, Colette; Nazir, Jameel; Watson, Verity

doi:10.1007/s40271-017-0263-7

Cited by 20 publications

(29 citation statements)

References 55 publications

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“…Considering the observation by Boeri et al ( 2020 ) that the LC model has an advantage over the RPL approach, as it is a model that can simultaneously estimate marginal preference weights associated with different attribute levels characterizing alternative medical treatments for different segments in the sample (referred to as classes) and can assign each class a probability that can be dependent on the respondent's characteristics, the LC model approach was selected for the main analysis of this paper. To determine the optimal number of classes for the model we considered three information criteria based on maximizing the log‐likelihood of the model and minimizing the number of parameters to be estimated: the Bayesian information criteria (BIC, Hurvich & Tsai, 1989 ), the Akaike information criteria (AIC, McLachlan & Peel, 2000 ) and the AIC3, a variation of the AIC that penalizes more for the number of parameters used in estimation (Wedel & Kamakura, 2000 ). Nevertheless, it is worth noting that these criteria also fail some of the regularity conditions for a valid test under the null (Leroux, 1992 ).…”

Section: Methodsmentioning

confidence: 99%

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Exploring patient preference heterogeneity for pharmacological treatments for chronic pain: A latent class analysis

Walsh

Boeri

Abraham

et al. 2022

European Journal of Pain

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Background: Several pharmaceutical treatments for chronic pain caused by osteoarthritis (OA) and chronic low back pain (CLBP) are available or currently under development, each associated with different adverse events (AEs) and efficacy profiles. It is therefore important to understand what trade-offs patients are willing to make when choosing between treatments. Methods:A discrete-choice experiment (DCE) was conducted with 437 adults with chronic pain caused by OA and/or CLBP. Respondents were presented with a series of scenarios and asked to choose between pairs of hypothetical treatments, each defined by six attributes: level of symptom control; risks of heart attack, rapidly progressive osteoarthritis and dependency; frequency and mode of administration and cost. Attributes were based on known profiles of oral nonsteroidal anti-inflammatory drugs, opioids and injected nerve growth factor inhibitors, the last of which were under clinical development at the time of the study. Data were analysed using a latent class (LC) model to explore preference heterogeneity.Results: Overall, respondents considered improving symptom control and reducing risk of physical dependency to be the most important attributes. The LC analysis identified four participant classes: an 'efficacy-focused' class (33.7%), a 'cost-averse' class (29.4%), a 'physical-dependence-averse' class (19.6%) and a 'needle-averse' class (17.3%). Subgroup membership was incompletely predicted by participant age and their responses to comprehension questions.Conclusions: Preference heterogeneity across respondents indicates a need for a personalized approach to offering treatment options. Symptom improvement, cost, physical dependence and route of administration might be important to different patients.

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Section: Methodsmentioning

confidence: 99%

“…The benefits of quantifying the monetary benefits of health interventions in this way, even in 'free at the point of use' health care systems such as the UK's NHS, have been explored previously (e.g. Boeri et al, 2019;Ikenwilo et al, 2018;Porteous et al, 2016;Scalone et al, 2011).…”

Section: Survey Developmentmentioning

confidence: 99%

Exploring patient preference heterogeneity for pharmacological treatments for chronic pain: A latent class analysis

Walsh

Boeri

Abraham

et al. 2022

European Journal of Pain

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“…The use of qualitative research to inform instrument development has gained considerable attention in the literature and has been identified as a major contributor to study quality [32][33][34][35]. In addition, qualitative findings may be useful to contextualise elicited quantitative preferences within a mixed-methods context [118]. However, the extent to which such research is conducted in practice varies and remains underreported [32,33].…”

Section: Study Development and Testingmentioning

confidence: 99%

A Systematic Review of Discrete Choice Experiments in Oncology Treatments

Collacott

Soekhai

Thomas

et al. 2021

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Background As the number and type of cancer treatments available rises and patients live with the consequences of their disease and treatments for longer, understanding preferences for cancer care can help inform decisions about optimal treatment development, access, and care provision. Discrete choice experiments (DCEs) are commonly used as a tool to elicit stakeholder preferences; however, their implementation in oncology may be challenging if burdensome trade-offs (e.g. length of life versus quality of life) are involved and/or target populations are small. Objectives The aim of this review was to characterise DCEs relating to cancer treatments that were conducted between 1990 and March 2020. Data Sources EMBASE, MEDLINE, and the Cochrane Database of Systematic Reviews were searched for relevant studies. Study Eligibility Criteria Studies were included if they implemented a DCE and reported outcomes of interest (i.e. quantitative outputs on participants' preferences for cancer treatments), but were excluded if they were not focused on pharmacological, radiological or surgical treatments (e.g. cancer screening or counselling services), were non-English, or were a secondary analysis of an included study. Analysis Methods Analysis followed a narrative synthesis, and quantitative data were summarised using descriptive statistics, including rankings of attribute importance. Result Seventy-nine studies were included in the review. The number of published DCEs relating to oncology grew over the review period. Studies were conducted in a range of indications (n = 19), most commonly breast (n =10, 13%) and prostate (n = 9, 11%) cancer, and most studies elicited preferences of patients (n = 59, 75%). Across reviewed studies, survival attributes were commonly ranked as most important, with overall survival (OS) and progression-free survival (PFS) ranked most important in 58% and 28% of models, respectively. Preferences varied between stakeholder groups, with patients and clinicians placing greater importance on survival outcomes, and general population samples valuing health-related quality of life (HRQoL). Despite the emphasis of guidelines on the importance of using qualitative research to inform attribute selection and DCE designs, reporting on instrument development was mixed. Limitations No formal assessment of bias was conducted, with the scope of the paper instead providing a descriptive characterisation. The review only included DCEs relating to cancer treatments, and no insight is provided into other health technologies such as cancer screening. Only DCEs were included. Conclusions and Implications Although there was variation in attribute importance between responder types, survival attributes were consistently ranked as important by both patients and clinicians. Observed challenges included the risk of attribute dominance for survival outcomes, limited sample sizes in some indications, and a lack of reporting about instrument development processes. Protocol Registration PROSPERO 2020 CRD42020184232.

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“…Additionally, it has been shown that men undergoing medical treatment for LUTS/BPH prefer treatment options with a low risk of adverse events; and that up to 93% prefer a treatment with no sexual side effects (erectile dysfunction, loss of libido, and ejaculatory dysfunction) [31]. Furthermore, a recent qualitative analysis showed that side effects affecting sexual function are of greater importance than non-sexual side effects for sexually active patients [32]. In that regard, HESr has been shown to improve symptoms and QoL in BPH patients, but with only limited side effects, which do not impact sexual function [1,7,10,11,33], and this could help to maintain adherence in patients worried about the effect of medical LUTS/BPH treatment on their sexual function.…”

Section: Discussionmentioning

confidence: 99%

Efficacy and Tolerability of 6-Month Treatment with Tamsulosin Plus the Hexanic Extract of Serenoa repens versus Tamsulosin Plus 5-Alpha-Reductase Inhibitors for Moderate-to-Severe LUTS-BPH Patients: Results of a Paired Matched Clinical Study

Alcaraz

Castro-Díaz

Gacci

et al. 2022

JCM

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The objective of this subset analysis was to evaluate and compare the efficacy and tolerability of two combination treatments for men with moderate-to-severe lower urinary tract symptoms associated with benign prostatic hyperplasia (LUTS/BPH). Data were from a real-world, open-label, prospective, and multicenter study performed in outpatient urology clinics. Men with moderate-to-severe LUTS/BPH received 6-month treatment with tamsulosin (TAM) in combination with either the hexanic extract of S. repens (HESr) or a 5-alpha-reductase inhibitor (5ARI). Changes in urinary symptoms and quality of life were measured using the IPSS and BII questionnaires, respectively. Treatment tolerability was assessed by recording adverse effects (AEs). Patients in the two study groups were matched using iterative and propensity score matching approaches. After iterative matching, data were available from 136 patients (n = 68 treated with TAM + 5ARI, n = 68 with TAM + HESr). After 6 months of treatment, mean (SD) IPSS total score improved by 7.7 (6.3) and 6.7 (5.0) points in the TAM + 5ARI and TAM + HESr groups, respectively (p = 0.272); mean BII total scores improved by 3.1 (2.9) and 2.9 (2.4) points (p = 0.751), respectively. AEs were reported by 26.5% and 10.3% of patients in the same groups, mostly affecting sexual function (p < 0.027). When used in a real-world setting to treat patients with moderate-severe LUTS/BPH, 6-month treatment with TAM + HESr was as effective as TAM + 5ARI, but with better tolerability.

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The Best of Both Worlds: An Example Mixed Methods Approach to Understand Men’s Preferences for the Treatment of Lower Urinary Tract Symptoms

Abstract: We argue that the insights gained into men's preferences for treatment and how LUTS affects men's QoL could not have been obtained by either the qualitative research or the DCE alone.

Cited by 20 publications

References 55 publications

Exploring patient preference heterogeneity for pharmacological treatments for chronic pain: A latent class analysis

Exploring patient preference heterogeneity for pharmacological treatments for chronic pain: A latent class analysis

A Systematic Review of Discrete Choice Experiments in Oncology Treatments

Efficacy and Tolerability of 6-Month Treatment with Tamsulosin Plus the Hexanic Extract of Serenoa repens versus Tamsulosin Plus 5-Alpha-Reductase Inhibitors for Moderate-to-Severe LUTS-BPH Patients: Results of a Paired Matched Clinical Study

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