Brett Hauber

Conjoint Analysis Applications in Health - How are Studies being Designed and Reported?: An Update on Current Practice in the Published Literature between 2005 and 2008.

Despite the increased popularity of conjoint analysis in health outcomes research, little is known about what specific methods are being used for the design and reporting of these studies. This variation in method type and reporting quality sometimes makes it difficult to assess substantive findings. This review identifies and describes recent applications of conjoint analysis based on a systematic review of conjoint analysis in the health literature. We focus on significant unanswered questions for which there is neither compelling empirical evidence nor agreement among researchers.We searched multiple electronic databases to identify English-language articles of conjoint analysis applications in human health studies published since 2005 through to July 2008. Two independent reviewers completed the detailed data extraction, including descriptive information, methodological details on survey type, experimental design, survey format, attributes and levels, sample size, number of conjoint scenarios per respondent, and analysis methods. Review articles and methods studies were excluded. The detailed extraction form was piloted to identify key elements to be included in the database using a standardized taxonomy.We identified 79 conjoint analysis articles that met the inclusion criteria. The number of applied studies increased substantially over time in a broad range of clinical applications, cancer being the most frequent. Most used a discrete-choice survey format (71%), with the number of attributes ranging from 3 to 16. Most surveys included 6 attributes, and 73% presented 7–15 scenarios to each respondent. Sample size varied substantially (minimum = 13, maximum = 1258), with most studies (38%) including between 100 and 300 respondents. Cost was included as an attribute to estimate willingness to pay in approximately 40% of the articles across all years.Conjoint analysis in health has expanded to include a broad range of applications and methodological approaches. Although we found substantial variation in methods, terminology, and presentation of findings, our observations on sample size, the number of attributes, and number of scenarios presented to respondents should be helpful in guiding researchers when planning a new conjoint analysis study in health.

Stated Preferences of Patients with Cancer for Health-related Quality-of-life (HRQOL) Domains During Treatment

Objectives: It is postulated that patients with different cancer diagnoses, stages of disease and treatments will exhibit different individual preferences for health-related quality-of-life (HRQOL) functional domains and symptoms. Methods: A stated-preference (SP) instrument incorporating all functional domains and symptoms of the EORTC Quality of Life Questionnaire (QLQ-C30) was administered to 400 patients with either breast (n=150); colorectal (n=150) or non-small cell lung cancer (n=100) who had previously experienced chemotherapy. The SP survey asked patients to make choices between a series of hypothetical functional/symptom pairs defined by combinations of HRQOL attributes, and depicted by levels of functioning and symptomatology. Results: In the 400 patients, considered as one group, role, cognitive, and social functioning, fatigue, nausea/vomiting, pain, appetite loss, diarrhea and financial difficulties were most important, whereas physical and emotional functioning, dyspnea, constipation and insomnia were less important. The four effects that patients with breast cancer most wished to avoid were nausea and vomiting, pain, and decreases in emotional and role functioning. Patients with colorectal cancer listed diarrhea as the second most important effect to avoid (after nausea/vomiting, but before pain and role functioning), whereas those with non-small cell lung cancer listed dyspnea as the fourth most important effect to avoid. Conclusion: These results provide more precise information regarding patient treatment concerns than that provided by the usual measurement of HRQOL. This information can be used by clinical trial investigators to design more precise interventions to improve HRQOL in the domains of greatest importance to patients and by all health care professionals to improve counseling of patients.

Patient preferences for treatments to delay bone metastases.

Most patients with advanced prostate cancer (PCa) develop bone metastases (BM) and present with bone complications like fracture. Bone‐targeted agents that prevent metastasis‐induced bone complications can cause adverse events. Understanding how patients view treatment options may optimize care. This study aimed to quantify how PCa patients value a hypothetical treatment that delays BM but can cause osteonecrosis of the jaw (ONJ). The study also assessed the value patients place on avoiding metastasis‐induced bone complications versus increased survival.

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.

Hemodialysis patients’ preferences for the management of secondary hyperparathyroidism

BackgroundPatient engagement and patient-centered care are critical in optimally managing patients with end-stage renal disease (ESRD). Understanding patient preferences is a key element of patient-centered care and shared decision making. The objective of this study was to elicit patients’ preferences for the treatment of secondary hyperparathyroidism (SHPT) associated with ESRD using a discrete-choice experiment survey.MethodsClinical literature, nephrologist input, patient-education resources, and a patient focus group informed development of the survey instrument, which was qualitatively pretested before its administration to a broader sample of patients. The National Kidney Foundation invited individuals in the United States with ESRD who were undergoing hemodialysis to participate in the survey. Respondents chose among three hypothetical SHPT treatment alternatives (two medical alternatives and surgery) in each of a series of questions, which were defined by attributes of efficacy (effect on laboratory values and symptoms), safety, tolerability, mode of administration, and cost. The survey instrument included a best-worst scaling exercise to quantify the relative bother of the individual attributes of surgery. Random-parameters logit models were used to evaluate the conditional relative importance of the attributes.ResultsA total of 200 patients with ESRD completed the survey. The treatment attributes that were most important to the respondents were whether a treatment was a medication or surgery and out-of-pocket cost. Patients had statistically significant preferences for efficacy attributes related to symptom management and laboratory values, but placed less importance on the attributes related to mode of administration and side effects. The most bothersome attribute of surgery was the risk of surgical mortality.ConclusionsPatients with ESRD and SHPT who are undergoing hemodialysis understand SHPT and have clear and measurable treatment preferences. These results may help inform clinicians about patients’ preferences regarding treatment options for a common complication of ESRD.

Hemodialysis patients’ preferences for the management of anemia

BackgroundPatient engagement in end-stage renal disease (ESRD) is expected to result in a more patient-centered approach to care that aligns with patients’ values, preferences, and goals for treatment. Nevertheless, no previous studies of which we are aware have evaluated patients’ benefit-risk preferences for the management of anemia associated with ESRD. The primary objective of this study was to quantify the tradeoffs patients are willing to make between cardiovascular risks associated with some anemia medicines and red blood cell (RBC) transfusions. A secondary objective was to quantify the importance of avoiding transfusion-related risks.MethodsA survey instrument was developed from the clinical literature, clinician input, patient-education resources, and a patient focus group. The survey instrument was qualitatively pretested before its administration to a broader sample of patients. The National Kidney Foundation invited individuals in the United States to participate in the survey. In a discrete-choice experiment (DCE), respondents chose between two hypothetical anemia medications in a series of questions. Each medication was defined by symptom relief, frequency of transfusions, cardiovascular risk, mode of administration, and out-of-pocket cost. The survey also included a best-worst scaling (BWS) exercise to quantify the importance of avoiding attributes of blood transfusions. Results from the DCE were used to estimate relative importance and marginal willingness to pay. Results from the BWS were converted to relative importance weights.ResultsA total of 200 individuals completed the survey. Patients were willing to accept a 6% medication-related risk of heart attack to avoid having two RBC transfusions per month. Symptom relief and mode of administration were of moderate importance. The most important transfusion-related risk to avoid was transfusion-related lung injury.ConclusionsPatients with ESRD and anemia have measurable treatment preferences and are willing to accept risks associated with anemia medications to avoid transfusions.

Comparison of US patient, rheumatologist, and dermatologist perceptions of psoriatic disease symptoms: results from the DISCONNECT study

BackgroundThe perceived bother of skin and joint-related manifestations of psoriatic disease may differ among patients, rheumatologists, and dermatologists. This study identified and compared the patient and dermatologist/rheumatologist-perceived bother of psoriatic disease manifestations.MethodsOnline surveys were administered to patients with both psoriasis and psoriatic arthritis and to dermatologists and rheumatologists. Object-case best–worst scaling was used to identify the most and least bothersome items from a set of five items in a series of questions. Each item set was drawn from 20 items describing psoriatic disease skin and joint symptoms and impacts on daily activities. Survey responses were analyzed using random-parameters logit models for each surveyed group, yielding a relative-bother weight (RBW) for each item compared with joint pain, soreness, or tenderness.ResultsSurveys were completed by 200 patients, 150 dermatologists, and 150 rheumatologists. Patients and physicians agreed that joint pain, soreness, and tenderness are among the most bothersome manifestations of psoriatic disease (RBW 1.00). For patients, painful, inflamed, or broken skin (RBW 1.03) was more bothersome, while both rheumatologists and dermatologists considered painful skin much less bothersome (RBW 0.17 and 0.22, respectively) than joint pain. Relative to joint pain, rheumatologists were more likely to perceive other joint symptoms as bothersome, while dermatologists were more likely to perceive other skin symptoms as bothersome.ConclusionsThis study has identified important areas of discordance both between patients and physicians and between rheumatologists and dermatologists about the relative bother of a comprehensive set of psoriatic disease symptoms and functional impacts. Both physician specialists should ask patients which manifestations of psoriatic disease are most bothersome to them, as these discussions may have important implications for drug and other patient management options.

608PPATIENT AND PHYSICIAN PREFERENCES FOR METASTATIC COLORECTAL CANCER TREATMENTS: A DISCRETE CHOICE EXPERIMENT

ABSTRACT Aim: To quantify the relative importance of benefits and risks of metastatic colorectal cancer (mCRC) treatments among patients and physicians. Methods: Patients with mCRC and physicians treating patients with mCRC in the United States completed an online discrete-choice experiment survey including questions with pairs of hypothetical mCRC treatments. Respondents were asked to choose their preferred treatment in each question. Treatments were described using 5 outcomes identified through a survey of 5 clinical experts (attributes) and the degree to which treatments induce those outcomes (levels). The attributes in the survey were: progression-free survival (PFS), chance of a severe skin rash, chance of serious bleeding, chance of cardiac arrest, and chance of gastrointestinal (GI) perforation. Chance of serious infusion reactions (SIR) was not included as a study attribute because it comprises multiple possible outcomes. Cardiac arrest was the most severe type of infusion reaction identified by clinical experts and was thus included instead of SIR. Using results from a main-effects random-parameters logit model, attribute importance was calculated as the expected change in treatment choice induced by varying outcomes between clinically relevant levels. Results: 127 patients and 150 physicians completed the survey. Given the clinically-relevant levels for each attribute (PFS: 6-10 months; chance of a severe skin rash: 0-20%; chance of serious bleeding: 0-5%; chance of cardiac arrest: 0-2%; chance of GI perforations: 0-2%) patients considered PFS to be most important, followed by chance of a severe skin rash, serious bleeding, cardiac arrest, and GI perforation. For physicians the most important attribute was PFS, followed by chance of cardiac arrest, severe skin rash, serious bleeding and GI perforation. Conclusions: Patients and physicians agree that PFS is the most important attribute of treatment. Physicians and patients had different rankings for risks suggesting the potential need for additional communication between patients and physicians regarding treatment-related risks. Disclosure: J.M. Gonzalez: Genentech Inc provided funding for this study to RTI-HS. Juan Marcos Gonzalez is an employee of RTI HS. The publication of study results was not contingent on Genentechs approval. RTI HS maintained independent scientific control of the study; S. Ogale: is a full time employee and stock owner of Roche/Genentech; R. Morlock: was paid by Genentech at the time of this work and is not a stock owner of Roche/Genentech; J. Posner: Genentech Inc provided funding for this study to RTI-HS. Joshua Posner is an employee of RTI HS. The publication of study results was not contingent on Genentechs approval. RTI HS maintained independent scientific control of the study; B. Hauber: Genentech Inc provided funding for this study to RTI-HS. B. Hauber is an employee of RTI HS- The publication of study results was not contingent on Genentechs approval. RTI HS maintained independent scientific control of the study.