Patient Preferences for Attributes of Androgen Deprivation Therapies in Prostate Cancer: A Discrete Choice Experiment with Latent Class Analysis.

INTRODUCTION: Medical androgen deprivation therapy (ADT) options have expanded for patients with advanced prostate cancer (PC). Historically, ADT was primarily available in long-acting injectable formulations. In 2020, the first oral formulation was US Food and Drug Administration-approved for adults with advanced PC. This study's aim was to assess patient preferences for attributes of medical ADT, including mode of administration, side effects, impact on sexual interest, and out-of-pocket (OOP) costs, and to segment respondents into distinct groups based on their treatment choice patterns. METHODS: A cross-sectional survey was conducted among US residents aged > 40 years with PC, employing a discrete choice experiment to assess preferences for ADT attributes. For each choice task, respondents were asked to select the hypothetical treatment profile that they preferred out of two presented. Latent class analysis (LCA) was conducted to estimate attribute-level preference weights and calculate attribute relative importance for groups of respondents with similar treatment preferences. RESULTS: A total of 304 respondents completed the survey (mean age 64.4 years). LCA identified four preference groups, named according to the attribute each group considered most important: Sexual interest, Cost-sensitive, Favors daily pill, and Favors injection. Most respondents in the Sexual interest group were < 65 years, while the Cost-sensitive group was mostly ≥ 65 years. Favors daily pill had the highest proportion of ADT-naïve individuals. On average, respondents in these groups preferred an oral medication. Favors injection, which had the highest proportion of ADT-experienced individuals, preferred infrequent intramuscular injections, lower chance of post-ADT testosterone recovery, and lower OOP cost. CONCLUSION: Respondents differed in their preferences regarding ADT attributes, highlighting the need for patient involvement in their treatment decisions. Effective communication between healthcare providers and patients about the benefits and risks of available therapies should be encouraged to ensure that patients receive the PC treatment that best meets their needs.

Prostate cancers often depend on the male sex hormone, testosterone, to grow. Androgen deprivation therapy (ADT) is used to lower testosterone levels in patients with advanced prostate cancer. ADT options available to patients have different characteristics, including how they are taken (injection or pill), side effects, impact on sexual interest, and costs. Researchers wanted to understand which ADT characteristics were most important to groups of patients with similar preferences. To do this, they gave 304 patients a series of two hypothetical (meaning not real) examples of ADT options with different characteristics and asked them to choose the option that they preferred most. Researchers found that patients could be separated into four different groups based on their preferences for ADT characteristics. One group preferred an ADT that had the least impact on their interest in sex. These patients were mainly younger than 65 years old. A second group preferred a lower cost ADT. These patients were mainly 65 years or older. A third group preferred a pill that could be taken once a day by mouth. Most of these patients did not take ADT in the past. A fourth group preferred an ADT that was given in a physician's office as an injection every 6 months. These patients mainly had taken ADT in the past. This study shows that patients have different preferences for ADT treatment characteristics. It is important for doctors to discuss the different ADT options with patients to find the treatment that best meets their needs.

Identifying drivers of first-line HR+/HER2- metastatic breast cancer treatment choices.

Aim: Assess factors associated with first-line (1L) treatment for HR+/HER2- metastatic breast cancer. Materials & methods: A cross-sectional survey of 250 US oncologists was conducted. Correlations were calculated between treatment class and demographics, treatment perceptions and other clinical/nonclinical characteristics. Results: Efficacy and safety/tolerability were critical in oncologists' 1L decision-making. CDK4/6i use positively correlated with proportion of Medicare and postmenopausal patients (r = 0.54-0.67). Chemotherapy use demonstrated positive correlations with perimenopausal and premenopausal patients and symptom burden (r = 0.31-0.42). Aromatase inhibitor (AI) monotherapy correlated positively with anticipated treatment compliance (r = 0.42). Conclusion: Efficacy and safety/tolerability were most important to 1L decision-making. Clinical characteristics corresponded with CDK4/6i and chemotherapy use. Anticipated compliance was associated with AI monotherapy use.

Patients in the USA with a certain type of metastatic breast cancer (mBC, i.e., HR+/HER2−) might get chemotherapy or hormone therapy alone instead of new and potentially better medicines called cyclin-dependent kinase 4/6 inhibitors (CDK4/6i) as their first treatment.Researchers wanted to understand how US cancer specialists decided the first treatment for this type of mBC. In a survey of 250 cancer specialists, researchers looked at different factors that might influence decision-making, including patient characteristics, doctors' opinions about the treatments and other medical and non-medical features. This study also examined the connections between these factors and the cancer specialists' choice of first treatment.Researchers found that cancer specialists care most about how well a treatment works and how safe it is when choosing the first treatment for HR+/HER2− mBC. They are more likely to use CDK4/6i if their patients have Medicare coverage or are older (i.e., women who have been through menopause). Chemotherapy is chosen if their patients are younger (i.e., women who are near and before menopause) or have more symptoms. Cancer specialists tend to choose first treatment with hormone therapy alone if they think their patients have a hard time following their treatment plan. The results showed that patient characteristics, doctors' opinions of treatments and other medical and non-medical factors play a role in choosing treatment for HR+/HER2− mBC. By understanding these factors, researchers can work toward improving treatment choices for patients with this type of mBC.

Patient experience and unmet needs in high-risk nonmuscle-invasive bladder cancer: Insights from qualitative interviews and a cross-sectional survey.

OBJECTIVES: To evaluate patient experience, unmet needs, and burden among patients with high-risk nonmuscle-invasive bladder cancer (HR-NMIBC) treated with Bacillus Calmette-Guérin (BCG). METHODS: This cross-sectional study included HR-NMIBC patients who received BCG treatment in the past 3 years. The study, preceded by a focused literature review, was conducted in 2 phases: 1) qualitative interviews with 32 patients in the United States (US), France, Germany, and United Kingdom (UK) and 2) quantitative survey of 150 patients in the US. Both phases of the study assessed patient characteristics, treatment history, experience, and perceptions, as well as side effects, pain, discomfort, and time burden associated with BCG treatment. The quantitative survey included additional items related to BCG treatment satisfaction, health-related quality of life (HRQoL), productivity, and healthcare resource utilization. Descriptive statistics and bivariate subgroup comparisons were reported. RESULTS: All patients in both study phases received transurethral resection of the bladder tumor (TURBT). Nearly all patients reported keeping their bladder/avoiding radical cystectomy (RC) was important (99%). Results from the quantitative survey reported a substantial impact to cancer-specific HRQoL of patients, with lower mean scores on physical (64.7), social (62.8), and role functioning (56.7) as measured by the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-30). Most patients (69%) were satisfied overall with BCG treatment, although satisfaction declined with increased number of side effects, higher numbers of BCG administrations, and greater discomfort (all P < 0.05). CONCLUSIONS: Most HR-NMIBC patients were satisfied overall with BCG treatment. Approximately half of the patients had stopped BCG treatment, notably, most during the induction phase, suggesting nonadherence to guidelines which recommend maintenance treatment after induction. Future treatments should focus on delaying or avoiding recurrence and cystectomy while reducing patient discomfort and discontinuation prior to completing the recommended course of treatment.

Psychosocial Wellbeing among Patients with Breast Cancer during COVID-19.

The impact of coronavirus disease 2019 (COVID-19) on the wellbeing of breast cancer (BC) patients is not well understood. This study described psychosocial problems among these patients in the United States (US) during the COVID-19 pandemic. Data were collected from BC patients via an online self-report survey between 30 March-6 July 2021 to assess the prevalence of COVID-19 diagnosis history and potential depression, health-related quality of life, COVID-related stress, and financial toxicity. Patients with early-stage (eBC) and metastatic (mBC) disease were compared. Of 669 patients included in the analysis, the prevalence of COVID-19 diagnosis history (10.9% versus 7.7%) and potential depression (33.7% versus 28.3%) were higher in mBC than eBC patients. Patients with eBC (versus mBC) had higher scores on nearly all Functional Assessment of Cancer Therapy-Breast scales (all, p < 0.001). For the Psychological Impact of Cancer subscales measuring negative coping strategies, the emotional distress score was the highest (9.1 ± 1.8) in the overall sample. Comprehensive Score for Financial Toxicity scores were higher in eBC than in mBC patients (24.2 ± 11.3 vs. 21.3 ± 10.2, p < 0.001). Overall, the COVID-19-related stress score was highest for danger/contamination fears (8.2 ± 5.6). In conclusion, impairments to psychosocial wellbeing among patients during the pandemic were observed, particularly financial toxicity and poor mental health and emotional functioning, with greater problems among mBC patients.

Financial Toxicity among Patients with Breast Cancer during the COVID-19 Pandemic in the United States.

This study reported the prevalence of financial distress (financial toxicity (FT)) and COVID-19-related economic stress in patients with breast cancer (BC). Patients with BC were recruited from the Ciitizen platform, Breastcancer.org, and patient advocacy groups between 30 March and 6 July 2021. FT was assessed with the COmprehensive Score for financial Toxicity (COST) instrument. COVID-19-related economic stress was assessed with the COVID-19 Stress Scale. Among the 669 patients, the mean age was 51.6 years; 9.4% reported a COVID-19 diagnosis. The prevalence rates of mild and moderate/severe FT were 36.8% and 22.4%, respectively. FT was more prevalent in patients with metastatic versus early BC (p < 0.001). The factors associated with FT included income ≤ USD 49,999 (adjusted odds ratio (adj OR) 6.271, p < 0.0001) and USD 50,000-USD 149,999 (adj OR 2.722, p < 0.0001); aged <50 years (adj OR 3.061, p = 0.0012) and 50-64 years (adj OR 3.444, p = 0.0002); living alone (adj OR 1.603, p = 0.0476); and greater depression severity (adj OR 1.155, p < 0.0001). Black patients (adj OR 2.165, p = 0.0133), patients with income ≤ USD 49,999 (adj OR 1.921, p = 0.0432), or greater depression severity (adj OR 1.090, p < 0.0001) were more likely to experience COVID-19-related economic stress. FT was common in patients with BC, particularly metastatic disease, during COVID-19. Multiple factors, especially lower income and greater depression severity were associated with financial difficulties during COVID-19.

Real-world usage of bevacizumab-bvzr biosimilar in US oncology practice.

OBJECTIVES: Bevacizumab is commonly used to treat solid tumors. However, little is known about the manner and the extent to which bevacizumab biosimilars are utilized in real-world oncology practice in the United States. The objective of this study was to describe patient and provider characteristics and treatment patterns associated with the recently introduced bevacizumab-bvzr biosimilar. STUDY DESIGN: Retrospective cohort study. METHODS: A retrospective analysis of medical and pharmacy claims between January 24, 2019, and July 31, 2020, was performed. Adult patients with at least 1 claim indicating usage of bevacizumab-bvzr were included. Patients who could not be assigned to an applicable diagnosis group were excluded. Index treatment date was defined as the date of the first claim for bevacizumab-bvzr. Descriptive analysis was conducted for all study variables. RESULTS: A total of 206 patients were included; patients most often were 65 years or older (49.5%), were female (62.6%), and resided in the West (45.1%). The most common indications observed for bevacizumab-bvzr were metastatic colorectal cancer (mCRC; 51.0%), cancer of the female genital organs (CFGO; 27.2%), glioblastoma (11.2%), and non-small cell lung cancer (8.7%). Overall, 72.4% and 48.2% of patients with mCRC and CFGO, respectively, had switched to bevacizumab-bvzr from the reference drug or another bevacizumab biosimilar. Bevacizumab-bvzr was used in chemotherapy combination regimens for patients with mCRC and CFGO. CONCLUSIONS: Utilization was observed in extrapolated indications. Findings suggest that both switching between reference product and bevacizumab biosimilars and using bevacizumab-bvzr as part of chemotherapy combination regimens have been adopted in US oncology practice.

Benefit-risk trade-offs in treatment choice in advanced HER2 negative breast cancer: patient and oncologist perspectives.

Aim: To evaluate which treatment attributes US patients and oncologists prioritize in HER2 negative advanced breast cancer (ABC). Methods: Preferences were assessed via a discrete choice experiment. Also, treatment goal statements were rated on an agreement scale. Results: Patients (n = 169) most valued improving overall survival (OS), followed by improving nausea and neuropathy. Oncologists (n = 117) most valued improving OS, followed by neuropathy and progression-free survival. Regarding treatment goals, oncologists (67%) perceived that patients are more focused on efficacy than quality of life; fewer patients (29%) agreed with this statement; 81% of oncologists and 51% of patients agreed that patients prefer oral treatment. Conclusion: Patients and oncologists were willing to accept increases in toxicities in exchange for efficacy improvements in HER2 negative ABC.

The goal of this study was to understand the preferences of patients and physicians for the benefits and risks associated with the treatment of advanced breast cancer that expresses normal amounts of the HER2 protein (termed as HER2 negative). Respondents completed an exercise where they compared two treatment options at a time that varied in their level of effectiveness and their potential for certain side effects and then selected the treatment option they most preferred. From this exercise, the treatment features that matter the most to patients and physicians were discovered. The most important treatment features for patients were lengthening life expectancy, decreasing the chance of experiencing nausea affecting appetite, and decreasing the chance of experiencing nerve damage involving numbness and/or pain, possibly severe, in hands and feet which may limit daily activities. The most important treatment features for physicians were lengthening life expectancy, decreasing the change of experiencing nerve damage involving numbness and/or pain, possibly severe, in hands and feet which may limit daily activities, and lengthening the time that cancer remains stable and does not worsen. Patients and physicians also rated how much they agreed with statements about their goals for treatment. While 67% of oncologists believed that their patients are more focused on killing the cancer than their quality of life, only 27% of patients were more focused on killing the cancer than their quality of life.

Patient and Caregiver Preferences for First-Line Treatments of Metastatic Non-Small Cell Lung Cancer: A Discrete Choice Experiment.

PURPOSE: The approval of immune checkpoint inhibitors for metastatic non-small-cell lung carcinomas (mNSCLC) treatment has presented more care options. Therefore, it is important to identify the benefit-risk trade-offs patients and caregivers are willing to make among potential treatment options. This study quantified the preferences of patients and caregivers for attributes of mNSCLC treatment. METHODS: Patients with mNSCLC and caregivers completed an online survey assessing preferences using a discrete choice experiment. Respondents chose between hypothetical treatment profiles, with varying levels for 7 attributes associated with first-line treatment, including overall survival (OS), progression-free survival, select adverse events (AEs), and regimen (caregivers). Hierarchical Bayesian modeling was used to estimate attribute-level preference weights. RESULTS: Patients (n = 308) and caregivers (n = 166) most valued increasing OS from 11 to 30 months, followed by decreasing the risk of a serious AE (grade 3/4) that may lead to hospitalization from 70% to 18%. These attributes were over twice as important to both sets of respondents as the other attributes measured. Patients and caregivers would accept increases in the risks of a serious AE (grade 3/4) from 18% to 70% and all grades nausea from 10% to 69% if OS increased by 16.8 and 4.0 months, respectively. The least valued attributes were all grades of pneumonitis (patients) and all grades of skin rash (caregivers). CONCLUSION: Patients and caregivers are willing to make trade-offs between efficacy and toxicity and may require up to 1.5 years of increased OS to accept a higher risk of AEs. These results can provide guidance to oncologists when engaging in shared-decision making discussions.

Patients' and oncologists' preferences for second-line maintenance PARP inhibitor therapy in epithelial ovarian cancer.

Aim: To understand the preferences of US patients and oncologists for PARP inhibitors as second-line maintenance (2LM) for epithelial ovarian cancer. Methods: A discrete choice experiment was conducted to assess the preferences of treatment attributes. Results: The most valued attributes were risk of grade 3/4 adverse events (AEs; patients, n = 204) and progression-free survival (PFS; oncologists, n = 151). To accept a 37% increased risk of grade 3/4 AEs, PFS would need to increase by 27.9 months (patients) and 6.3 months (oncologists). The least valued attributes were dosing form/frequency (patients) and grade 3/4 anemia risk (oncologists). Conclusion: Patients' and oncologists' willingness to make benefit-risk trade-offs in the 2LM setting suggests that the PFS gains observed in selected studies of poly (ADP-ribose) polymerase inhibitors in BRCA-mutated disease are worth the toxicity risk.

Plain language summary Maintenance therapy is a treatment option intended to keep ovarian cancer from coming back or getting worse for as long as possible after responding to chemotherapy. PARP inhibitors are a new type of maintenance therapy for ovarian cancer. This study aimed to understand the patients' and physicians' preferences for the benefits and risks associated with different PARP inhibitors used as maintenance therapy for ovarian cancer. Participants were asked to compare various treatment options based on their different safety profiles, effectiveness and form of medication (e.g., three capsules by mouth once a day versus two tablets by mouth twice a day), and then choose the treatment they most preferred. Through this exercise, the treatment features that mattered most to patients and physicians were identified. The most important treatment feature for patients was decreasing the chance of experiencing a serious side effect that requires medical intervention or hospitalization. In contrast, physicians valued lengthening the time that a cancer remains stable and does not worsen. To accept a 37% higher chance of experiencing a side effect that requires medical intervention or hospitalization, patients expect their cancer to remain stable and not worsen for an additional 28 months. This was a large difference from the 6 months that the physicians would consider as acceptable. The least important treatment features for patients are the amount of pills required per dose, the form of the given medication (e.g., tablet vs capsule) and the schedule of taking the treatment. On the other hand, physicians were least concerned about lowering the risk of experiencing low blood counts that, requiring medical intervention.

Evolving Perceptions, Utilization, and Real-World Implementation Experiences of Oncology Monoclonal Antibody Biosimilars in the USA: Perspectives from Both Payers and Physicians.

BACKGROUND: The adoption of oncology biosimilars has been slow in the USA, which may be attributed in part to stakeholder perceptions and lack of operational guidance that supports favorable access to biosimilars. OBJECTIVE: Our objective was to understand the real-world implementation experiences with oncology biosimilars of US payers and healthcare professionals (HCPs) as their experience with biosimilars has evolved. METHODS: In-depth qualitative interviews with payers (n = 20) and HCPs (n = 17 physicians, n = 3 practice managers) were conducted. Payers included managed care organizations (MCOs), integrated delivery networks, and pharmacy benefit managers (PBMs). Physicians were affiliated with a healthcare network or were community based, specialized in hematology/oncology, and had prescribed oncology biosimilars. Audio transcripts of the interviews were coded using MaxQDA software to enable descriptive analysis of the qualitative data. RESULTS: Over 80.0% of physicians perceived the efficacy and safety of biosimilars to be highly comparable to that of originators. Up to 87.5% of physicians reported using biosimilars in > 50% of their treatment-naïve patients and were comfortable using biosimilars in all approved indications. To encourage utilization, 75.0% of MCOs/PBMs preferred biosimilars over originators in treatment-naïve patients and implementation via step therapy. Physician involvement in choosing biosimilars was minimal, which was largely dependent on practice protocols or insurance preferences. The major factor influencing payers' coverage decisions and biosimilar adoption was potential cost savings. CONCLUSIONS: US payers and physicians who have experience with biosimilars have favorable views of oncology biosimilars, particularly for treatment-naïve patients. A framework for integrating biosimilars into oncology practice is developing, primarily driven by insurance coverage, contracting, and cost benefits.

Real-world use and acceptance of biosimilar monoclonal antibodies of rituximab in oncology practice in the USA.

Aim: To describe treatment patterns and patient and provider characteristics associated with the recently introduced biosimilar rituximab-pvvr. Methods: This retrospective analysis included adult patients with one or more claims for rituximab-pvvr, with an index date of 23 January 2020 and a study period covering 1 January 2019-31 July 2020. Results: Of 249 patients included, the most common rituximab-pvvr indications were non-Hodgkin's lymphoma (77.5%) and chronic lymphocytic leukemia (11.2%). Some patients with non-Hodgkin's lymphoma (42.5%) and chronic lymphocytic leukemia (39.3%) switched to rituximab-pvvr from the reference product or another rituximab biosimilar. Most patients were aged ≥65 years (63.5%) and were male (54.6%). Most (59.0%) rituximab-pvvr prescribers practiced in the south of the USA. Conclusion: Utilization occurred in approved and extrapolated indications. These preliminary findings suggest switching between reference product and rituximab biosimilars; rituximab-pvvr combination regimens are being adopted in real-world oncology practice.

Lay abstract A biosimilar is a biological medication that is highly similar in structure and function to a biological medication already approved by the US FDA ­ the 'original biologic'. The first biosimilars approved to treat certain blood cancers have become available in the USA. This study examined how a recently introduced rituximab biosimilar was being utilized, looking at patient and physician characteristics from a medical and prescription insurance claims database. This study did not examine the safety or effectiveness of this medication. While initial data are limited, the biosimilar, rituximab-pvvr, appears to be utilized to treat the same types of cancer as the original biologic, rituximab. The biosimilar was most frequently prescribed for non-Hodgkin's lymphoma and chronic lymphocytic leukemia.

Patient, Oncologist, and Payer Preferences for Adjuvant Endocrine Therapy and CDK4/6 Inhibitor Regimens in Early-Stage Breast Cancer: A Discrete Choice Experiment.

PURPOSE: Several adjuvant phase III trials are evaluating cyclin-dependent kinase 4/6 inhibitors (CDK4/6is) in combination with endocrine therapy (ET) in hormonal receptor positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) early-stage breast cancer (eBC). This study examines preferences for this combination regimen and ET alone among patients, oncologists, and payers in the United States. METHODS: A web-based questionnaire, including a discrete choice experiment (DCE), was administered to patients, practicing oncologists, and payers. In the DCE, respondents selected between hypothetical treatment profiles with attributes associated with ET monotherapy and CDK4/6i + ET regimens. Each treatment alternative was defined by the following attributes: 5-year invasive disease-free survival (iDFS), nausea, diarrhea, neutropenia, alopecia, dosing schedule, and electrocardiogram (ECG) monitoring. Payers had the additional attribute of annual per-patient treatment cost. Hierarchical Bayesian models were used to estimate relative preference weights for each attribute-level and relative attribute importance. RESULTS: For patients (n=300) and oncologists (n=200), iDFS was most important (2 to 3 times more important than the next most important attribute), followed by neutropenia and diarrhea risks for patients and oncologists, respectively. Patients and oncologists required an improvement in iDFS of 8.0 and 5.6 percentage-points, respectively, to accept an increase in diarrhea risk from 11% to 81%. Payers (n=60) viewed annual per-patient cost as most important for treatment access decision-making, closely followed by iDFS. Payers required an improvement in iDFS of 21.8 percentage-points to accept an increase in cost from $5,100 to $149,400. Across all stakeholder groups, dosing schedule, alopecia risk, and ECG monitoring were perceived as least important. CONCLUSION: Patients, oncologists, and payers expect a large absolute risk reduction in efficacy to offset the potential risks and costs of adding a CDK4/6i to current standard of care. An open discussion between all stakeholders is necessary to ensure that decision-making, whether at patient- or system-level, is informed by preferences for novel treatments, like CDK4/6is.

Oncologist and Patient Preferences for Novel Agents in First-Line Treatment for Chronic Lymphocytic Leukemia: Commonalities and Disconnects.

PURPOSE: Treatment for chronic lymphocytic leukemia (CLL) has changed dramatically with the approval of novel agents. Information regarding how patients and oncologists make trade-offs across attributes of novel therapies is limited. The purpose of this study was to understand how variations in attributes impact treatment choice among patients and oncologists. PATIENTS AND METHODS: In this study, 371 participants (patients [n=220] and oncologists [n=151]) completed an online discrete choice experiment (DCE) to quantify preferences for first-line (1L) CLL treatment with novel agents; participants chose between hypothetical treatment profiles consisting of eight attributes with varying levels taken from published literature. Hierarchical Bayesian models were used to estimate attribute level preference weights. The weights were used to compute relative importance, a measure of how influential an attribute is to treatment choice. RESULTS: Increasing 2-year progression-free survival (PFS) from 75% to 95% had the greatest impact on preferences in 1L CLL treatment, accounting for 40% and 30% of the variation in preferences among patients and oncologists, respectively. Risk differences in atrial fibrillation (AF), infection, and discontinuation due to adverse events (AEs) were also important to patients and oncologists. Among both groups, risk differences in tumor lysis syndrome (TLS) and bleeding were least influential in treatment choice. Oncologists required 2-4 times higher increases in 2-year PFS than patients to accept increased risks of AF, discontinuation due to AEs, bleeding, TLS, and arthralgia/myalgia. CONCLUSION: Patient-oncologist communication may be improved by a more focused discussion on the risks of AEs, relative to treatment outcomes, with patient goals in mind.

Oncologist and Patient Preferences for Attributes of CDK4/6 Inhibitor Regimens for the Treatment of Advanced/Metastatic HR Positive/HER2 Negative Breast Cancer: Discrete Choice Experiment and Best-Worst Scaling.

PURPOSE: To understand and compare preferences for dosing- and toxicity-related attributes associated with selective cyclin-dependent 4/6 kinase inhibitors regimens among US oncologists and patients. MATERIALS AND METHODS: Oncologists and patients with mBC participated in an internet-based survey that included a discrete choice experiment (DCE) and a best-worst scaling (BWS) exercise. For the DCE, participants chose between two hypothetical treatment profiles, each with seven attributes: risk of dose reduction due to adverse events (AEs), risk of diarrhea, risk of abdominal pain, need for electrocardiogram (ECG) monitoring to assess heart function, risk of Grade 3/4 neutropenia, dosing regimen, and dosing schedule. The BWS exercise assessed the relative prioritization of a larger set of 16 attributes. Hierarchical Bayesian models were used to estimate preference weights for each attribute level. RESULTS: Oncologists (N=209) and patients (N=304) rated risks of diarrhea (25% each) and Grade 3/4 neutropenia (20% and 24%, respectively) as the most important attributes for treatment choice. The risks of diarrhea and Grade 3/4 neutropenia were 1.8 to 2.3 times (oncologists: 25% and 20%, respectively vs 11%) and 2.4 to 2.5 times (patients: 25% and 24%, respectively vs 10%) higher in relative importance than the risk of dose reduction due to AEs. Oncologists placed greater importance on the risk of dose reduction due to AEs and the need for ECG monitoring, whereas patients placed greater importance on the risk of Grade 3/4 neutropenia (all, p<0.05). The BWS exercise results were largely consistent with those from the DCE. CONCLUSION: The risks of diarrhea and Grade 3/4 neutropenia were key drivers of both oncologist and patient preferences. Overall, the palbociclib + aromatase inhibitor (AI) profile was most preferred, due to its association with a lower risk of diarrhea and no ECG monitoring, compared with abemaciclib + AI and ribociclib + AI profiles, respectively.