Did a bot eat your homework? An assessment of the potential impact of bad actors in online administration of preference surveys.

BACKGROUND: Online administration of surveys has a number of advantages but can also lead to increased exposure to bad actors (human and non-human bots) who can try to influence the study results or to benefit financially from the survey. We analyze data collected through an online discrete-choice experiment (DCE) survey to evaluate the likelihood that bad actors can affect the quality of the data collected. METHODS: We developed and fielded a survey instrument that included two sets of DCE questions asking respondents to select their preferred treatments for multiple myeloma therapies. The survey also included questions to assess respondents' attention while completing the survey and their understanding of the DCE questions. We used a latent-class model to identify a class associated with perverse preferences or high model variance, and the degree to which the quality checks included in the survey were correlated with class membership. Class-membership probabilities for the problematic class were used as weights in a random-parameters logit to recover population-level estimates that minimizes exposure to potential bad actors. RESULTS: Results show a significant proportion of respondents provided answers with a high degree of variability consistent with responses from bad actors. We also found that a wide-ranging selection of conditions in the survey screener is more consistent with choice patterns expected from bad actors looking to qualify for the study. The relationship between the number of incorrect answers to comprehension questions and problematic choice patterns peaked around 5 out of 10 questions. CONCLUSIONS: Our results highlight the need for a robust discussion around the appropriate way to handle bad actors in online preference surveys. While exclusion of survey respondents must be avoided under most circumstances, the impact of "bots" on preference estimates can be significant.

Discrete-Choice Experiment to Understand the Preferences of Patients with Hormone-Sensitive Prostate Cancer in the USA, Canada, and the UK.

BACKGROUND: Treatment options for patients with metastatic hormone-sensitive prostate cancer (mHSPC) have broadened, and treatment decisions can have a long-lasting impact on patients' quality of life. Data on patient preferences can improve therapeutic decision-making by helping physicians suggest treatments that align with patients' values and needs. OBJECTIVE: This study aims to quantify patient preferences for attributes of chemohormonal therapies among patients with mHSPC in the USA, Canada, and the UK. METHODS: A discrete-choice experiment survey instrument was developed and administered to patients with high- and very-high-risk localized prostate cancer and mHSPC. Patients chose between baseline androgen-deprivation therapy (ADT) alone and experimentally designed, hypothetical treatment alternatives representing chemohormonal therapies. Choices were analyzed using logit models to derive the relative importance of attributes for each country and to evaluate differences and similarities among patients across countries. RESULTS: A total of 550 respondents completed the survey (USA, 200; Canada, 200; UK, 150); the mean age of respondents was 64.3 years. Treatment choices revealed that patients were most concerned with treatment efficacy. However, treatment-related convenience factors, such as route of drug administration and frequency of monitoring visits, were as important as some treatment-related side effects, such as skin rash, nausea, and fatigue. Patient preferences across countries were similar, although patients in Canada appeared to be more affected by concomitant steroid use. CONCLUSION: Patients with mHSPC believe the use of ADT alone is insufficient when more effective treatments are available. Efficacy is the most significant driver of patient choices. Treatment-related convenience factors can be as important as safety concerns for patients.

Use of Patient Preferences Data Regarding Multiple Risks to Inform Regulatory Decisions.

Background and Objectives. Risk-tolerance measures from patient-preference studies typically focus on individual adverse events. We recently introduced an approach that extends maximum acceptable risk (MAR) calculations to simultaneous maximum acceptable risk thresholds (SMART) for multiple treatment-related risks. We extend these methods to include the computation and display of confidence intervals and apply the approach to 3 published discrete-choice experiments to evaluate its utility to inform regulatory decisions. Methods. We generate MAR estimates and SMART curves and compare them with trial-based benefit-risk profiles of select treatments for depression, psoriasis, and thyroid cancer. Results. In the depression study, SMART curves with 70% to 95% confidence intervals portray which combinations of 2 adverse events would be considered acceptable. In the psoriasis example, the asymmetric confidence intervals for the SMART curve indicate that relying on independent MARs versus SMART curves when there are nonlinear preferences can lead to decisions that could expose patients to greater risks than they would accept. The thyroid cancer application shows an example in which the clinical incidence of each of 3 adverse events is lower than the single-event MARs for the expected treatment benefit, yet the collective risk profile surpasses acceptable levels when considered jointly. Limitations. Nonrandom sample of studies. Conclusions. When evaluating conventional MARs in which the observed incidences are near the estimated MARs or where preferences demonstrate diminishing marginal disutility of risk, conventional MAR estimates will overstate risk acceptance, which could lead to misinformed decisions, potentially placing patients at greater risk of adverse events than they would accept. Implications. The SMART method, herein extended to include confidence intervals, provides a reproducible, transparent evidence-based approach to enable decision makers to use data from discrete-choice experiments to account for multiple adverse events. Highlights: Estimates of maximum acceptable risk (MAR) for a defined treatment benefit can be useful to inform regulatory decisions; however, the conventional metric considers one adverse event at a time.This article applies a new approach known as SMART (simultaneous maximum acceptable risk thresholds) that accounts for multiple adverse events to 3 published discrete-choice experiments.Findings reveal that conventional MARs could lead decision makers to accept a treatment based on individual risks that would not be acceptable if multiple risks are considered simultaneously.

Quantifying Value of Hope.

BACKGROUND: 'Hope' is a construct in patient-centered value frameworks, but few studies have attempted to measure the value of hope separately from treatment-related gains in quality of life and survival to support its application in economic evaluation. OBJECTIVE: To generate quantitative information on the "value of hope". METHODS: We designed a discrete-choice experiment in which treatment alternatives varied the probability of achieving 10-year survival, expected survival as the weighted sum of short-term and long-term survival, health status, and out-of-pocket cost. Two-hundred patients with cancer or history of cancer recruited by Cancer Support Community each completed 10 choice questions. We used mixed-logit and latent-class models to analyze the choice data. RESULTS: Relative to fixed survival periods of two, three or five years with 0% chance of 10-year survival, participants positively valued treatments with 5% and 10% chances of 10-year survival. However, participants negatively valued a 20% chance of 10-year survival that required an offsetting 80% chance of shorter survival. This finding was particularly strong when expected survival was two years. Compared to a 0% chance, dollar-equivalent values of 5% and 10% chances of long-term survival were $5,975 and $12,421, respectively, independent of health status or expected survival. The corresponding value for 20% versus 0% chance of long-term survival was negative. Latent-class analysis revealed 4 groups with distinct preference patterns. CONCLUSIONS: Our findings affirm positive value for hope independent of expected survival and health status. However, this finding does not universally hold in all situations nor across all groups.

The preferences of women with ovarian cancer for oral versus intravenous recurrence regimens.

OBJECTIVE: To assess preferences of women with ovarian cancer regarding features of available anti-cancer regimens for platinum-resistant, biomarker-positive disease, with an emphasis on oral PARP inhibitor and standard intravenous (IV) chemotherapy regimens. METHODS: A discrete-choice-experiment preferences survey was designed, tested, and administered to women with ovarian cancer, with 11 pairs of treatment profiles defined using seven attributes (levels/ranges): regimen (oral daily, IV weekly, IV monthly); probability of progression-free (PFS) at 6 months (40%-60%); probability of PFS at 2 years (10%-20%); nausea (none, moderate); peripheral neuropathy (none, mild, moderate); memory problems (none, mild); and total out-of-pocket cost ($0 to $10,000). RESULTS: Of 123 participants, 38% had experienced recurrence, 25% were currently receiving chemotherapy, and 18% were currently taking a PARP inhibitor. Given attributes and levels, the relative importance weights (sum 100) were: 2-year PFS, 28; cost, 27; 6-month PFS, 19; neuropathy,14; memory problems, nausea, and regimen, all ≤5. To accept moderate neuropathy, participants required a 49% (versus 40%) chance of PFS at 6 months or 14% (versus 10%) chance at 2 years. Given a 3-way choice where PFS and cost were equal, 49% preferred a monthly IV regimen causing mild memory problems, 47% preferred an oral regimen causing moderate nausea, and 4% preferred a weekly IV regimen causing mild memory and mild neuropathy. CONCLUSIONS: These findings challenge the assumption that oral anti-cancer therapies are universally preferred by patients and demonstrate that there is no "one size fits all" regimen that is preferable to women with ovarian cancer when considering recurrence treatment regimens.

The Impact of the Risk Functional Form Assumptions on Maximum Acceptable Risk Measures.

BACKGROUND: Discrete-choice experiments (DCEs) are increasingly conducted to quantify risk tolerance by computing maximum acceptable risk (MAR) for improvements in efficacy or other benefits gained from new medical treatments. To compute MARs from DCE data, respondents are asked to make choices under uncertainty between treatments. Specific treatment-related harms are included in the choice questions as probabilistic adverse events (AEs), and choice variation with the probability of these outcomes is assumed to indicate their effect on the expected utility of treatments. With a limited number of comparisons between profiles, calculation of MARs requires understanding how outcome probabilities that are not explicitly considered in the DCE can change the value of medical technologies. This study aims to examine how various assumptions on the expected disutility of these excluded probabilities can result in different MAR measures. METHODS: We summarize commonly used empirical specifications for the expected disutility of AEs and derive the resulting MAR functions. We then discuss an empirical application on treatments to delay bone metastases in oncology patients with solid tumors. RESULTS: A total of 187 respondents completed the DCE. Results show the impact of making various assumptions about the expected disutility of AEs, and the resulting MAR values for specific health benefits. As expected, different assumptions resulted in variations in MAR values for specific health benefits. Even with small differences in MAR measures, our results suggest that the assumptions evaluated here can lead to different conclusions about the acceptability of a medical technology. CONCLUSION: Results show possible systematic variations in MARs caused by the assumed form of the effect of changes in the probability of AEs. Furthermore, we find that different assumptions can lead to different conclusions about the acceptability of a medical technology, even when MAR distributions overlap. This result suggests that researchers should evaluate the assumptions they are making for these effects and use sensitivity analysis to evaluate the robustness of risk-tolerance measures from stated-preference data.

Patient Preferences for Outcomes Following DCIS Management Strategies: A Discrete Choice Experiment.

PURPOSE: Ductal carcinoma in situ (DCIS), a nonobligate precursor of breast cancer, is often aggressively managed with multimodal therapy. However, there is limited research on patients' preferences for trade-offs among treatment-related outcomes such as breast appearance, side effects, and future cancer risk. We sought to investigate whether women consider treatment features aside from cancer risk when making treatment choices for ductal carcinoma in situ and if so, to what degree other features influence these decisions. METHODS: A discrete choice experiment was administered to participants in a comprehensive cancer screening mammography clinic. The experimental design was used to generate constructed health profiles resulting from different management strategies. Health profiles were defined by breast appearance, severity of infection within the first year, chronic pain, hot flashes, and risk of developing or dying from breast cancer within 10 years. RESULTS: One hundred ninety-four women without a personal history of breast cancer completed the choice task. Across 10 choice questions, 29% always selected the health profile with a lower risk of invasive breast cancer (ie, dominated on cancer risk), regardless of the effects of treatment. For nonrisk dominators, breast cancer risk remained the most important factor but was closely followed by chronic pain (24% [95% CI, 20 to 28]) and infection (22% [95% CI, 18 to 25]). Depending on treatment outcomes, the tolerable increase in breast cancer risk was as high as 3.4%. CONCLUSION: Most women were willing to make some trade-offs between invasive cancer risk and treatment-related outcomes. Our findings highlight the importance of shared decision-making weighing risks and benefits between patient and provider management of low-risk disease.

Surgical treatment for recurrent shoulder instability: factors influencing surgeon decision making.

BACKGROUND: The optimal surgical approach for recurrent anterior shoulder instability remains controversial, particularly in the face of glenoid and/or humeral bone loss. The purpose of this study was to use a contingent-behavior questionnaire (CBQ) to determine which factors drive surgeons to perform bony procedures over soft tissue procedures to address recurrent anterior shoulder instability. METHODS: A CBQ survey presented each respondent with 32 clinical vignettes of recurrent shoulder instability that contained 8 patient factors. The factors included (1) age, (2) sex, (3) hand dominance, (4) number of previous dislocations, (5) activity level, (6) generalized laxity, (7) glenoid bone loss, and (8) glenoid track. The survey was distributed to fellowship-trained surgeons in shoulder/elbow or sports medicine. Respondents were asked to recommend either a soft tissue or bone-based procedure, then specifically recommend a type of procedure. Responses were analyzed using a multinomial-logit regression model that quantified the relative importance of the patient characteristics in choosing bony procedures. RESULTS: Seventy orthopedic surgeons completed the survey, 33 were shoulder/elbow fellowship trained and 37 were sports medicine fellowship trained; 52% were in clinical practice ≥10 years and 48% <10 years; and 95% reported that the shoulder surgery made up at least 25% of their practice. There were 53% from private practice, 33% from academic medicine, and 14% in government settings. Amount of glenoid bone loss was the single most important factor driving surgeons to perform bony procedures over soft tissue procedures, followed by the patient age (19-25 years) and the patient activity level. The number of prior dislocations and glenoid track status did not have a strong influence on respondents' decision making. Twenty-one percent glenoid bone loss was the threshold of bone loss that influenced decision toward a bony procedure. If surgeons performed 10 or more open procedures per year, they were more likely to perform a bony procedure. CONCLUSION: The factors that drove surgeons to choose bony procedures were the amount of glenoid bone loss with the threshold at 21%, patient age, and their activity demands. Surprisingly, glenoid track status and the number of previous dislocations did not strongly influence surgical treatment decisions. Ten open shoulder procedures a year seems to provide a level of comfort to recommend bony treatment for shoulder instability.

Patient Preferences for Surgical Treatment of Knee Osteoarthritis: A Discrete-Choice Experiment Evaluating Total and Unicompartmental Knee Arthroplasty.

BACKGROUND: Total knee arthroplasty (TKA) is a common treatment for end-stage knee osteoarthritis but is associated with increased complication rates compared with unicompartmental knee arthroplasty (UKA). UKA offers better functional outcomes but is associated with a higher risk of revision. The purpose of this study was to apply good-practice, stated-preference methods to quantify patient preferences for benefit-risk tradeoffs associated with arthroplasty treatments for end-stage knee osteoarthritis. METHODS: A discrete-choice experiment was developed with the following attributes: chance of complications, functional ability, awareness of the knee implant, and chance of needing another operation within 10 years. Patients included those aged 40 to 80 years with knee osteoarthritis. A pivot design filtered respondents into 1 of 2 surveys on the basis of self-reported functional ability (good compared with fair or poor) as measured by the Oxford Knee Score. Treatment-preference data were collected, and relative attribute-importance weights were estimated. RESULTS: Two hundred and fifty-eight completed survey instruments from 92 males and 164 females were analyzed, with 72 respondents in the good-function cohort and 186 in the fair/poor-function cohort. Patients placed the greatest value or relative importance on serious complications and rates of revision in both cohorts. Preference weights did not vary between cohorts for any attribute. In the good-function cohort, 42% of respondents chose TKA and 58% chose UKA. In the fair/poor-function cohort, 54% chose TKA and 46% chose UKA. CONCLUSIONS: Patient preferences for various treatment attributes varied among patients in a knee osteoarthritis population. Complication and revision rates were the most important factors to patients, suggesting that physicians should focus on these areas when discussing treatments. The proportion of patients who chose UKA suggests that the current trend of increased UKA utilization is aligned with patient preferences. CLINICAL RELEVANCE: Systematic elicitation of patient preferences for knee arthroplasty procedures, which lays out evidence-based risks and benefits of different treatments, indicates a larger subset of the knee osteoarthritis population may prefer UKA than would be suggested by the current rates of utilization of the procedure. Arthroplasty treatment should align with patient preferences and eligibility criteria to better deliver patient-centered care.

Do Patient Preferences Align With Value Frameworks? A Discrete-Choice Experiment of Patients With Breast Cancer.

Purpose. Assess patient preferences for aspects of breast cancer treatments to evaluate and inform the usual assumptions in scoring rubrics for value frameworks. Methods. A discrete-choice experiment (DCE) was designed and implemented to collect quantitative evidence on preferences from 100 adult female patients with a self-reported physician diagnosis of stage 3 or stage 4 breast cancer. Respondents were asked to evaluate some of the treatment aspects currently considered in value frameworks. Respondents' choices were analyzed using logit-based regression models that produced preference weights for each treatment aspect considered. Aggregate- and individual-level preferences were used to assess the relative importance of treatment aspects and their variability across respondents. Results. As expected, better clinical outcomes were associated with higher preference weights. While life extensions with treatment were considered to be most important, respondents assigned great value to out-of-pocket cost of treatment, treatment route of administration, and the availability of reliable tests to help gauge treatment efficacy. Two respondent classes were identified in the sample. Differences in class-specific preferences were primarily associated with route of administration, out-of-pocket treatment cost, and the availability of a test to gauge treatment efficacy. Only patient cancer stage was found to be correlated with class assignment (P = 0.035). Given the distribution of individual-level preference estimates, preference for survival benefits are unlikely to be adequately described with two sets of preference weights. Conclusions. Although value frameworks are an important step in the systematic evaluation of medications in the context of a complex treatment landscape, the frameworks are still largely driven by expert judgment. Our results illustrate issues with this approach as patient preferences can be heterogeneous and different from the scoring weights currently provided by the frameworks.

Patient Preferences Around Extent of Surgery in Low-Risk Thyroid Cancer: A Discrete Choice Experiment.

Background: Patient preferences pertaining to surgical options for thyroid cancer management are not well studied. Our aim was to conduct a discrete choice experiment (DCE) to characterize participants' views on the relative importance of various risks and benefits associated with lobectomy versus total thyroidectomy for low-risk thyroid cancer. Methods: Adult participants with low-risk thyroid cancer or a thyroid nodule requiring surgery were asked to choose between experimentally designed surgical options with varying levels of risk of nerve damage (1%, 9%, 14%), hypocalcemia (0%, 3%, 8%), risk of needing a second surgery (0%, 40%), cancer recurrence (1%, 3%, 5%), and need for daily thyroid hormone supplementation (yes, no). Their choices were analyzed using random-parameters logit regression. Results: One hundred fifty participants completed an online DCE survey. Median age was 58 years; 82% were female. Twenty-four participants (16%) had a diagnosis of thyroid cancer at the time of completing the survey, and 126 (84%) had a thyroid nodule necessitating surgery. On average, 35% of participants' choices were explained by differences in the risk of cancer recurrence; 28% by the chance of needing a second surgery; 19% by the risk of nerve damage; and 9% by differences in risks of hypocalcemia and the need for thyroid hormone supplementation. When accounting for differences in postoperative risks, the average patient favored lobectomy over total thyroidectomy as long as the chance of needing a second (i.e., completion) surgery after initial lobectomy remained below 30%. Participants would accept a 4.1% risk of cancer recurrence if the risk of a second surgery could be reduced from 40% to 10%. Conclusions: While patients with thyroid cancer may have clear preferences for extent of surgery, common themes moderating preferences for surgical interventions were identified in the DCE. Adequate preoperative evaluation to decrease the chance of a second surgery and providing patients with a good understanding of risks and benefits associated with extent of surgery can lead to better treatment decision-making.

Patient preferences for maintenance PARP inhibitor therapy in ovarian cancer treatment.

OBJECTIVE: To measure preferences of women with ovarian cancer regarding risks, side effects, costs and benefits afforded by maintenance therapy (MT) with a poly ADP ribose polymerase (PARP) inhibitor. METHODS: A discrete-choice experiment elicited preferences of women with ovarian cancer regarding 6 attributes (levels in parentheses) relevant to decisions for MT versus treatment break: (1) overall survival (OS; 36, 38, 42 months); (2) progression-free survival (PFS; 15, 17, 21 months); (3) nausea (none, mild, moderate); (4) fatigue (none, mild, moderate); (5) probability of death from myelodysplastic syndrome/acute myelogenous leukemia (MDS/AML; 0% to 10%); (6) monthly out-of-pocket cost ($0 to $1000). Participants chose between 2 variable MT scenarios and a static scenario representing treatment break, with multiple iterations. Random-parameters logit regression was applied to model choices as a function of attribute levels. RESULTS: 95 eligible participants completed the survey; mean age was 62, 48% had recurrence, and 17% were ever-PARP inhibitor users. Participants valued OS (average importance weight 24.5 out of 100) and monthly costs (24.6) most highly, followed by risk of death from MDS/AML (17.9), nausea (14.7), PFS (10.5) and fatigue (7.8). Participants would accept 5% risk of MDS/AML if treatment provided 2.2 months additional OS or 4.8 months PFS. Participants would require gains of 2.6 months PFS to accept mild treatment-related fatigue and 4.4 months to accept mild nausea. CONCLUSIONS: When considering MT, women with ovarian cancer are most motivated by gains in OS. Women expect at least 3-4 months of PFS benefit to bear mild side effects of treatment.

Parental Preferences for Vesicoureteral Reflux Treatment: A Crowd-sourced, Best-worst Scaling Study.

OBJECTIVE: To quantitatively evaluate parental preferences for the various treatments for vesicoureteral reflux using crowd-sourced best-worst scaling, a novel technique in urologic preference estimation. METHODS: Preference data were collected from a community sample of parents via 2 best-worst scaling survey instruments published to Amazon's Mechanical Turk online community. Attributes and attribute levels were selected following extensive review of the reflux literature. Respondents completed an object case best-worst scaling exercise to prioritize general aspects of reflux treatments and multiprofile case best-worst scaling to elicit their preferences for the specific differences in reflux treatments. Data were analyzed using multinomial logistic regression. Results from the object-case provided probability scaled values (PSV) that reflected the order of importance of attributes. RESULTS: We analyzed data for 248 and 228 respondents for object and multiprofile case BWS, respectively. When prioritizing general aspects of reflux treatment, effectiveness (PSV = 20.37), risk of future urinary tract infection (PSV = 14.85), and complication rate (PSV = 14.55) were most important to parents. Societal cost (PSV = 1.41), length of hospitalization (PSV = 1.09), and cosmesis (PSV = 0.91) were least important. Parents perceived no difference in preference for the cosmetic outcome of open vs minimally invasive surgery (P = .791). Bundling attribute preference weights, parents in our study would choose open surgery 74.9% of the time. CONCLUSION: High treatment effectiveness was the most important and preferred attribute to parents. Alternatively, cost and cosmesis were among the least important. Our findings serve to inform shared parent-physician decision-making for vesicoureteral reflux.

Testing a behavioral intervention to improve adherence to adjuvant endocrine therapy (AET).

Adjuvant endocrine therapy (AET) is used to prevent recurrence and reduce mortality for women with hormone receptor positive breast cancer. Poor adherence to AET is a significant problem and contributes to increased medical costs and mortality. A variety of problematic symptoms associated with AET are related to non-adherence and early discontinuation of treatment. The goal of this study is to test a novel, telephone-based coping skills training that teaches patients adherence skills and techniques for coping with problematic symptoms (CST-AET). Adherence to AET will be assessed in real-time for 18 months using wireless smart pill bottles. Symptom interference (i.e., pain, vasomotor symptoms, sleep problems, vaginal dryness) and cost-effectiveness of the intervention protocol will be examined as secondary outcomes. Participants (N = 400) will be recruited from a tertiary care medical center or community clinics in medically underserved or rural areas. Participants will be randomized to receive CST-AET or a general health education intervention (comparison condition). CST-AET includes ten nurse-delivered calls delivered over 6 months. CST-AET provides systematic training in coping skills for managing symptoms that interfere with adherence. Interactive voice messaging provides reinforcement for skills use and adherence that is tailored based on real-time adherence data from the wireless smart pill bottles. Given the high rates of non-adherence and recent recommendations that women remain on AET for 10 years, we describe a timely trial. If effective, the CST-AET protocol may not only reduce the burden of AET use but also lead to cost-effective changes in clinical care and improve breast cancer outcomes. Trials registration: ClinicalTrials.gov, NCT02707471, registered 3/3/2016.

Comparing the Relative Importance of Attributes of Metastatic Renal Cell Carcinoma Treatments to Patients and Physicians in the United States: A Discrete-Choice Experiment.

OBJECTIVES: Value assessments of new treatments for metastatic renal cell carcinoma (RCC) should include outcomes that are most important to patients. This study aimed to quantify and compare the conditional relative importance of the attributes of RCC treatments to patients and physicians in the United States. METHODS: Patients with RCC and physicians who treat RCC completed an online discrete-choice experiment survey with a fractional factorial D-optimal experimental design. In a series of 12 questions, respondents chose between two hypothetical treatments defined in terms of six attributes: progression-free survival (PFS), probability of living ≥ 3 years (PL3Y), skin reactions, severity of fatigue, mode of administration, and monthly co-payment. Treatment choices were analyzed using a random-parameters logit model to estimate relative preference weights for the attribute levels and conditional relative attribute importance (i.e. the importance of an attribute relative to all other attributes conditional on the range of levels of that attribute). RESULTS: Overall, 201 patients and 142 physicians completed the survey. For both patients and physicians, PL3Y was the attribute with the greatest and statistically significant conditional relative importance. Estimates of the conditional relative importance of PFS, skin reactions, and mode of administration for patients, and for PFS and mode of administration for physicians, were not statistically significant. The preferences for improvements in PFS were independent of the level of PL3Y for both patients and physicians. Conditional relative attribute importance varied by patient disease stage. CONCLUSIONS: Patients and physicians indicated that PL3Y was the most important treatment attribute and was significantly more important than PFS. Importance rankings differed between physicians and patients and between all patients and those with advanced/metastatic disease.

Evaluating Risk Tolerance from a Systematic Review of Preferences: The Case of Patients with Psoriasis.

BACKGROUND: Stated-preference methods have been widely used to evaluate patient-relative preferences for the benefits and potential harms of psoriasis treatments. However, risk tolerance measures for treatment-related harms, a corollary of preferences, are rare despite their critical role in shared decision making and regulatory benefit-risk evaluations. This article presents a method to enhance information on patient risk tolerance through previously published preference results. OBJECTIVE: The objective of this article was to conduct the first meta-analysis of preferences to characterize the distribution of patients' maximum acceptable risk of harms associated with psoriasis treatments. DATA SOURCES: Maximum acceptable risks for treatment-related adverse events were extracted or derived from preference results published between 2011 and 2017. SYNTHESIS METHODS: Four different analyses were conducted to evaluate maximum acceptable risk information across studies: (1) listing of maximum acceptable risk values, (2) naïve aggregation of maximum acceptable risks, (3) estimation of maximum acceptable risk mother distribution, and (4) random-effect regression analysis of maximum acceptable risks. RESULTS: Nine publications with maximum acceptable risk results, or with enough information to derive maximum acceptable risks, were identified from the search and screening of preference studies. The most commonly evaluated treatment benefits were duration of benefits, percentage and probability of improvement, and reductions in the coverage of lesions. The adverse-event risks most often included in the publications were those commonly associated with biologics, such as serious infections and malignancies. As expected, maximum acceptable risks changed with treatment benefits and treatment-related adverse events. CONCLUSIONS: The results confirm the feasibility of using previously published preference information to characterize patient risk tolerance. The estimated distributions of maximum acceptable risk provide a benchmark against which future results can be compared, and signal gaps in our understanding of risk tolerance for specific health outcomes.

Patient and physician preferences for anticancer drugs for the treatment of metastatic colorectal cancer: a discrete-choice experiment.

OBJECTIVE: Many publications describe preferences for colorectal cancer (CRC) screening; however, few studies elicited preferences for anticancer-drug treatment for metastatic CRC (mCRC). This study was designed to elicit preferences and risk tolerance among patients and oncologists in the USA for anticancer drugs to treat mCRC. MATERIALS AND METHODS: Patients aged 18 years or older with a self-reported diagnosis of mCRC and board-certified (or equivalent) oncologists who had treated patients with mCRC were recruited by two survey research companies from existing online patient panels in the USA. Additional oncologists were recruited from a list of US physicians. Patients and oncologists completed a discrete-choice experiment (DCE) survey. DCEs offer a systematic method of eliciting preferences and quantifying both the relative importance of treatment attributes and the tradeoffs respondents are willing to make among benefits and risks. Treatment attributes in the DCE were progression-free survival (PFS) and risks of severe papulopustular rash, serious hemorrhage, cardiopulmonary arrest, and gastrointestinal perforation. Patients' and physicians' maximum levels of acceptable treatment-related risks for two prespecified increases in efficacy were estimated. RESULTS: A total of 127 patients and 150 oncologists completed the survey. Relative preferences for the treatment attributes in the study were mostly consistent with the expectation that better clinical outcomes were preferred over worse clinical outcomes. Risk tolerance varied between patients and physicians. On average, physicians were willing to tolerate higher risks than patients, although these differences were mostly not statistically significant. Post hoc latent-class analyses revealed that some patients and physicians were unwilling to forgo any efficacy to avoid toxicities, while others were willing to make such tradeoffs. CONCLUSION: Differences in preferences between patients and physicians suggest that there is the potential for improvement in patients' well-being. Initiating or enhancing discussions about patient tolerance for toxicities, such as skin rash and gastrointestinal perforations, may help prescribe treatments that entail more appropriate benefit-risk tradeoffs.

Neoadjuvant Systemic Therapy for Breast Cancer: Factors Influencing Surgeons' Referrals.

BACKGROUND: This study aimed to assess the influence of disease- and patient-related factors on surgeons' decisions to refer patients with early-stage breast cancer (EBC) for neoadjuvant systemic therapy (NST). METHODS: An online survey of United States surgeons evaluated the influence of selected disease- and patient-related factors on surgeons' decisions, rated their influence (individually and in combination), and provided a relative ranking of jointly considered factors using best-worst scaling. RESULTS: The participants in this study were 100 licensed surgeons. The surgeons referred approximately 25 % of EBC patients for NST to improve surgical management. Approximately 75 % of the surgeons agreed that NST is important for EBC, if only to improve surgical management. More than half were "very likely" to refer EBC patients for NST based on anatomicopathologic factors. Less than 50 % were "very likely" to do so when considering tumor phenotype factors. Tumor size and lymph node status were ranked highest in hypothetical patient scenarios. Regarding combinations of factors, the importance of any single factor varied according to the combinations presented. Less than half of the respondents were "very familiar," and half were "somewhat familiar" with NST guidelines for breast cancer. More than half of the respondents were unaware that findings have shown achievement of pathologic complete response (pCR) after NST to be associated with improved survival. CONCLUSIONS: Surgeons' decision to refer for NST is strongly driven by surgical management goals. Anatomicopathologic factors are more influential than tumor phenotype. However, no single disease or patient factor consistently drives the decision to refer for NST. Surgeons' awareness of the association between pCR achievement and longer survival could be improved.

Patient, Caregiver, and Nurse Preferences for Treatments for Bone Metastases from Solid Tumors.

BACKGROUND: Bone-targeted agents (BTAs) used for the prevention of skeletal-related events (SREs) associated with metastatic bone disease possess different attributes that factor into treatment decisions. OBJECTIVE: The aim of this study was to evaluate preferences of patients, caregivers, and nurses for features of BTAs used to prevent SREs in patients with a self-reported physician diagnosis of bone metastasis from solid tumors. METHODS: Patients (n = 187), primary caregivers (n = 197), or nurses (n = 196) completed a web-enabled discrete-choice experiment (10-question survey) in which they chose between pairs of hypothetical profiles of BTAs. Each profile was defined by six key treatment attributes, including efficacy and safety (two each) and route/frequency of administration and cost (one each). The relative importance of treatment attributes and levels was estimated. RESULTS: The most important treatment attribute for patients and nurses was out-of-pocket cost, and for caregivers, treatment-related risk of renal impairment. Risk of renal impairment was the second most important attribute for patients and nurses, while time until first SRE was the third most important attribute for all respondents. For nurses, risk of osteonecrosis of the jaw was least important, and for patients and caregivers, mode of administration was least important. LIMITATIONS: Respondents considered hypothetical medications; therefore, their decisions may not have the same consequences as actual decisions. CONCLUSIONS: The perspectives of patients, caregivers, and nurses are integral when making treatment decisions about BTAs to prevent SREs associated with solid tumors. Identifying the relative importance of attributes of BTAs will aid in the proper selection of therapy in this setting, which may improve patient outcomes.

Patient Benefit-Risk Tradeoffs for Radioactive Iodine-Refractory Differentiated Thyroid Cancer Treatments.

Background. The aims of this study were to assess patients' preferences to wait or start systemic treatment and understand how patients would make tradeoffs between certain severe adverse events (AEs) and additional months of progression-free survival (PFS). Materials and Methods. Adults in France, Germany, and Spain with a diagnosis of DTC and who have had at least one RAI treatment completed a direct-elicitation question and a discrete-choice experiment (DCE) online. The direct-elicitation question asked respondents whether they would opt out of treatment when their tumor is RAI-R. In the DCE, respondents chose between 12 pairs of hypothetical RAI-R DTC treatment profiles. Profiles were defined by magnitudes of efficacy (PFS) and safety (severe hand-foot skin reaction [HFSR], severe proteinuria, and severe hypertension). A main-effects random-parameters logit model was estimated. Results. 134 patients completed the survey. Most patients (86.6%) opted for treatment rather than "wait and see" decision. Patients placed a greater weight on the risk of severe hypertension than the risk of proteinuria and HFSR. Conclusions. DTC patients showed preference toward treatment for RAI-R DTC over watchful waiting. Patients' concerns about the risk of severe hypertension appeared to have had a greater effect on patients' choice than severe proteinuria or HFSR.