Drug Preferences Among Patients And Physicians For Diagnosed Dyslipidemia
Overview
The study focused on evaluating patient and physician drug preferences regarding lipid-lowering drugs, particularly proprotein convertase subtilisin/kexin type 9 inhibitors, as an alternative to statins for dyslipidemia treatment. Employing an online discrete choice experiment (DCE), the research aimed to ascertain the relative importance of six key attributes related to these drugs among dyslipidemia patients and cardiovascular physicians in China. These attributes included frequency and mode of administration, reduction of low-density lipoprotein cholesterol (LDL-C) level, risk of myopathy, risk of liver damage, and monthly out-of-pocket cost.
Findings from the study, which involved 708 patients and 507 physicians, revealed divergent preferences between the two groups. Patients prioritized attributes such as the risk of liver damage, mode of administration, and frequency of administration, while physicians placed greater importance on LDL-C level reduction, followed by concerns about liver damage and myopathy risk.
Notably, patients valued attributes related to administration frequency and mode more than physicians, whereas physicians emphasized LDL-C level reduction and myopathy risk to a greater extent. Moreover, physicians exhibited higher willingness to pay for most attributes compared to patients, except for administration frequency and mode.
Through latent class analysis (LCA), the study identified distinct patient and physician classes based on their preferences. Patients were grouped into categories focusing on oral administration, hepatic safety and frequency, and hepatic safety and cost, while physicians were categorized into frequency-insensitive, efficacy-focused, and safety-focused classes.
The study underscored the importance of considering patient preferences in treatment decision-making, emphasizing the need for personalized treatment plans based on patient-specific preferences. Patient involvement in the study, from questionnaire design to DCE survey participation, contributed to promoting shared decision-making and optimizing treatment regimens. Overall, the findings highlighted the significance of understanding and integrating patient preferences into lipid-lowering treatment strategies to enhance treatment outcomes and patient satisfaction.
Introduction
This study addresses a critical aspect of healthcare: the effective management of dyslipidemia, a significant risk factor for atherosclerotic cardiovascular disease (ASCVD), including heart attacks and strokes. Despite the well-documented benefits of lipid-lowering medications, such as statins, patient adherence to treatment remains a global challenge. This issue is particularly pronounced in China, where dyslipidemia prevalence is increasing rapidly, yet only a small fraction of patients receive adequate lipid-lowering therapy.
While statins are the cornerstone of cholesterol-lowering treatment, some patients fail to achieve target cholesterol levels even with high doses, and others experience adverse effects like liver damage or muscle pain. As an alternative, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors have emerged as effective lipid-lowering agents. However, their non-oral administration and high cost have limited their widespread use, posing financial barriers to patients. Recent policy changes in China, however, aim to alleviate this burden by including PCSK9 inhibitors in drug reimbursement lists.
In contrast to the United States, where shared decision-making between physicians and patients is advocated in cholesterol management guidelines, such practices are not prevalent in China. Patient preferences often take a backseat, leading to potential mismatches between treatment plans and patient needs. Given the variability in preferences observed across different medical contexts, understanding and comparing patient and physician preferences regarding lipid-lowering medications are crucial steps towards improving patient-centered care.
To achieve this, the study employs a discrete choice experiment (DCE), a quantitative method that allows for the systematic evaluation of individual preferences by presenting respondents with hypothetical treatment scenarios and assessing their preferences through trade-offs among different attributes. By quantifying and comparing patient and physician preferences, the study aims to identify key attributes deemed important for lipid-lowering drug therapy and explore potential differences in preferences between the two groups. Ultimately, the insights gained from this research endeavor can inform clinical decision-making, foster patient-centered care, and enhance treatment adherence in the management of dyslipidemia.
Method
This study meticulously followed the recommended research practices outlined by the International Society for Pharmacoeconomics and Outcomes Research, adhering to a well-defined framework comprising key steps. The initial phase involved formulating a precise research question and identifying pertinent attributes and levels essential for the discrete choice experiment (DCE). Attributes were discerned through a comprehensive literature review and validated via a focus group discussion involving patients and physicians familiar with lipid-lowering drugs.
Based on feedback, six attributes were selected for inclusion in the DCE: frequency and mode of administration, reduction of LDL-C level, risk of myopathy, risk of liver damage, and out-of-pocket monthly cost. These attributes were meticulously defined and stratified into appropriate levels based on literature review, expert opinion, and patient preference validation.
The experiment design utilized a Bayesian D-optimal approach to generate choice sets, ensuring optimal efficiency in data collection. A fractional factorial design was employed, with each choice set containing two scenarios and an ‘opt-out’ option to mitigate bias. Choice sets were presented in a randomized order to prevent order effects, and graphical representations aided participants’ decision-making process.
Both patient and physician questionnaires comprised three sections, collecting sociodemographic data, introducing attributes and levels, and administering choice sets. To ensure comprehension, a comprehension-checking question was included, and only respondents providing accurate responses proceeded further. An online survey was conducted to administer the questionnaire, with a robust sample size of 500 respondents achieved through collaboration with national dyslipidemia patient organizations and cardiovascular physician associations.
Recruitment quotas and inclusion criteria were enforced to ensure a diverse and representative sample. Monetary incentives were provided to respondents upon survey completion, and stringent criteria were applied to exclude respondents exhibiting inconsistent selections or completing the survey too quickly.
Overall, data collection was conducted anonymously and meticulously from July to November 2022, adhering to rigorous methodological standards to yield reliable and insightful results for analyzing preferences regarding lipid-lowering drugs.
Statistical Analysis
The study employed a comprehensive analytical approach to investigate respondent preferences in healthcare choices. Descriptive statistics were first utilized to characterize the respondents. Then, a mixed logit model and latent class analysis were applied to estimate preferences. The ‘out-of-pocket monthly cost’ attribute was continuously coded to determine marginal willingness to pay (mWTP), while dummy coding was used for other attributes. Alternative specific constants were also considered in the models to enhance accuracy.
Random parameters were estimated using 500 standard Halton sequences, ensuring robustness in the analysis. mWTP was calculated based on coefficients, reported in US dollars using the 2022 exchange rate. Relative importance (RI) of each attribute was determined to assess its significance in decision-making. The study compared preferences between patients and physicians using independent t-tests.
Latent class analysis (LCA) was employed to identify distinct respondent segments based on preferences. Model fit indices such as Akaike information criterion (AIC), Bayesian information criteria (BIC), and log-likelihood were used to determine the optimal number of latent classes. Lower values of AIC, BIC, and log-likelihood indicated better model fit.
The chi-square test was utilized to examine variations in individual characteristics across different respondent classes. Sensitivity analyses were conducted, treating the cost variable as categorical to capture its impact accurately. Statistical significance was set at p < .05, and 95% confidence intervals (CIs) were calculated using the bootstrap method. All analyses were performed using Stata software, ensuring rigorous and reliable statistical analysis.
Result
The study encompassed 708 patients and 507 physicians, analyzing their preferences regarding various attributes related to hyperlipidemia management. Patients, predominantly male (48.16%), aged 40 to 50 years (35.45%), and educated (76.55%), exhibited preferences influenced by factors such as LDL-C levels, duration of hyperlipidemia, and mode of treatment. Physicians, comprising 47.53% males, with 58.58% aged between 30 and 40 years and 72.39% holding a master’s degree or above, demonstrated preferences shaped by similar attributes but with differing emphases.
Utilizing a mixed logit model, the study discerned significant coefficients for all attributes among patients and physicians, except for the frequency of administration between ‘once a week’ and ‘once a day’. Patients and physicians exhibited nuanced preferences, with negative inclinations towards subcutaneous injections, myopathy and liver damage risks, and higher costs, while favoring less frequent administration and lower LDL-C levels.
Moreover, patients prioritized attributes differently from physicians. Patients valued ‘risk of liver damage’, ‘mode of administration’, and ‘frequency of administration’ as the most significant factors, while physicians emphasized ‘reduction of LDL-C level’ followed by ‘risk of liver damage’ and ‘risk of myopathy’. Preference heterogeneity was evident among patients and physicians across three distinct classes, each exhibiting unique preferences and priorities.
Furthermore, the sensitivity analysis reaffirmed the main findings, highlighting patients’ continued concern regarding liver damage risk and physicians’ focus on LDL-C level reduction. Overall, the study’s comprehensive analysis sheds light on the intricate preferences of patients and physicians in hyperlipidemia management, providing valuable insights for healthcare decision-making and treatment optimization.
Conclusion
This study represents a groundbreaking endeavor, offering empirical insights into the contrasting preferences of patients and physicians concerning lipid-lowering drug therapy, a crucial aspect of managing dyslipidemia. Notably, patients prioritized safeguarding against liver damage, while physicians emphasized the imperative of reducing LDL-C levels. Additionally, patients placed significant value on attributes like oral administration and infrequent dosing, which were somewhat underestimated by physicians. The study identified three distinct preference classes within both patient and physician cohorts, indicating the nuanced nature of preferences within these groups.
Rigorous measures were implemented to ensure the integrity of the questionnaire data, including pilot testing, comprehension assessments, and internal validity checks. Methodologically, the study employed the mixed logit model to account for unobserved preference heterogeneity, a robust approach compared to traditional models. Notably, the bootstrap method was utilized for estimating confidence intervals, enhancing the reliability of the findings.
Comparisons with similar dyslipidemia prevalence studies in China revealed demographic similarities, albeit with differences in age and education levels among participants. These disparities were attributed to challenges in recruiting older adults for online surveys. Importantly, patient preference alignment across age groups affirmed the robustness of the study’s findings.
Findings underscored the divergence in priorities between patients and physicians, echoing similar observations in other medical domains. While physicians emphasized LDL-C reduction, patients favored attributes related to administration convenience and safety. Notably, patients exhibited heightened concern regarding the risk of liver damage, reflecting their broader health considerations beyond lipid control.
Insights gleaned from patient preferences shed light on potential gaps in patient education and awareness regarding the cardiovascular risks associated with elevated LDL-C levels. Moreover, the study revealed distinct preference patterns within patient and physician cohorts, emphasizing the need for tailored, patient-centered approaches to treatment decision-making.
The study’s implications extend to clinical practice, emphasizing the importance of shared decision-making between patients and physicians in lipid-lowering drug therapy. Recognizing and accommodating diverse patient preferences can enhance treatment adherence and ultimately improve health outcomes. Moreover, the study underscores the significance of ongoing patient and public education initiatives to enhance awareness of cardiovascular risk factors and treatment options.
While the study provides valuable insights, certain limitations exist, including sample representativeness and the number of attributes considered. Nonetheless, the findings offer a valuable foundation for future research and clinical practice in optimizing lipid-lowering therapy strategies.
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