Administration options for pegfilgrastim prophylaxis: patient and physician preferences from a cross-sectional survey.

OBJECTIVE: Although clinical guidelines recommend administration of pegfilgrastim 1-4 days after a myelosuppressive chemotherapy cycle to decrease the incidence of febrile neutropenia (FN), some physicians administer pegfilgrastim on the same day as chemotherapy administration. A novel on-body injector (OBI) that automatically delivers pegfilgrastim the day after chemotherapy is also available. Our objective was to estimate patient and physician preferences among the pegfilgrastim administration options. METHODS: We conducted a cross-sectional survey of patients receiving pegfilgrastim and physicians prescribing pegfilgrastim. Respondents' preferences for pegfilgrastim administration options were elicited using direct elicitation; the relative importance of features associated with the options was estimated in a point-allocation exercise. Physicians considered two hypothetical patient profiles when completing the exercises. RESULTS: The samples included 200 patients and 200 physicians. Patients generally preferred the administration option with which they had experience. Among patients, 48.5% with previous in-clinic injections 24 hours after chemotherapy preferred this option; 56.8% with previous OBI administration preferred this option. For a clinically compromised patient, 37.5% of physicians preferred an in-clinic injection option; 49.5% preferred the OBI. For a less compromised patient, 55.5% preferred an in-clinic injection option; 28.0% preferred the OBI. Avoiding the need to return to the clinic was chosen most often as the most important treatment feature for patients and physicians. CONCLUSIONS: Patients and physicians identified that returning clinic visits for pegfilgrastim administration may be burdensome. A potential solution to mitigate this burden is the OBI, which allows adherence to the labeled use of pegfilgrastim without return visits to the clinic.

Relative importance of benefits and risks associated with antithrombotic therapies for acute coronary syndrome: patient and physician perspectives.

BACKGROUND: In acute coronary syndrome (ACS), antithrombotic therapies prevent thrombotic events, but also increase bleeding risk. Knowledge is limited about how patients and physicians balance these benefits and risks. OBJECTIVE: To quantify US patient and physician preferences for outcomes associated with antithrombotic therapies in ACS. METHODS: Two independent web-based surveys were conducted using best-worst scaling in board-certified cardiologists and adult patients hospitalized within the last 5 years due to heart attack and who used aspirin or prescription antithrombotic therapies. Participants selected best and worst of three possible outcomes across a series of questions. Outcomes included death, various levels of stroke, myocardial infarction (MI), and bleeding. Data were analyzed using a maximum difference model employing random-parameters logit. Relative importance of each outcome was estimated relative to death. FINDINGS: Patients (n = 206) and physicians (n = 273) who met face validity requirements, viewed death and nonfatal major disabling stroke as nearly equivalent and most important outcomes to avoid. Relative to death and disabling stroke, physicians considered nondisabling stroke, all nonfatal bleeding, and mild MI all as least important to avoid, while patients considered all bleeds, except major bleeding requiring transfusion, as least important to avoid. Physicians considered severe MI equivalent to 0.92 (0.02 SE) deaths. Patients (∼0.35 [0.04] deaths) and physicians (∼0.64 [0.05] deaths) had different views for nonfatal moderate stroke. Patients viewed nonfatal major bleeding requiring transfusion ∼0.13 (0.02) deaths, and nonfatal heart attack ∼0.09 (0.02) deaths. CONCLUSION: US patients and physicians agree on the relative importance of avoiding death, disabling stroke and bleeding without transfusions. Differing perspectives on bleeding requiring transfusions, MI, and moderately disabling stroke suggest that patients and physicians may have different benefit-risk preferences. Transparent discussion between physicians and patients in ACS treatment shared decision-making seems warranted, although limitations of survey methodology and cultural differences compared with US participants should be considered.

Quantifying benefit-risk preferences for medical interventions: an overview of a growing empirical literature.

Decisions regarding the development, regulation, sale, and utilization of pharmaceutical and medical interventions require an evaluation of the balance between benefits and risks. Such evaluations are subject to two fundamental challenges-measuring the clinical effectiveness and harms associated with the treatment, and determining the relative importance of these different types of outcomes. In some ways, determining the willingness to accept treatment-related risks in exchange for treatment benefits is the greater challenge because it involves the individual subjective judgments of many decision makers, and these decision makers may draw different conclusions about the optimal balance between benefits and risks. In response to increasing demand for benefit-risk evaluations, researchers have applied a variety of existing welfare-theoretic preference methods for quantifying the tradeoffs decision makers are willing to accept among expected clinical benefits and risks. The methods used to elicit benefit-risk preferences have evolved from different theoretical backgrounds. To provide some structure to the literature that accommodates the range of approaches, we begin by describing a welfare-theoretic conceptual framework underlying the measurement of benefit-risk preferences in pharmaceutical and medical treatment decisions. We then review the major benefit-risk preference-elicitation methods in the empirical literature and provide a brief overview of the studies using each of these methods. The benefit-risk preference methods described in this overview fall into two broad categories: direct-elicitation methods and conjoint analysis. Rating scales (6 studies), threshold techniques (9 studies), and standard gamble (2 studies) are examples of direct elicitation methods. Conjoint analysis studies are categorized by the question format used in the study, including ranking (1 study), graded pairs (1 study), and discrete choice (21 studies). The number of studies reviewed here demonstrates that this body of research already is substantial, and it appears that the number of benefit-risk preference studies in the literature will continue to increase. In addition, benefit-risk preference-elicitation methods have been applied to a variety of healthcare decisions and medical interventions, including pharmaceuticals, medical devices, surgical and medical procedures, and diagnostics, as well as resource-allocation decisions such as facility placement. While preference-elicitation approaches may differ across studies, all of the studies described in this review can be used to provide quantitative measures of the tradeoffs patients and other decision makers are willing to make between benefits and risks of medical interventions. Eliciting and quantifying the preferences of decision makers allows for a formal, evidence-based consideration of decision-makers' values that currently is lacking in regulatory decision making. Future research in this area should focus on two primary issues-developing best-practice standards for preference-elicitation studies and developing methods for combining stated preferences and clinical data in a manner that is both understandable and useful to regulatory agencies.

Patient preferences and assessment of likely adherence to hepatitis C virus treatment.

To estimate patient preferences for attributes of hepatitis C virus (HCV) treatment and patients' assessment of the likely effect of treatment attributes on treatment adherence, HCV patients ≥18 years old completed an online survey that included nine 2-alternative choice questions. Each choice question was defined by the probability of sustained viral response (Efficacy), injection frequency (Frequency), duration of flu-like symptoms after every injection (Flu), injection device (Device), average number of days of work missed each week (Lost Work Days), probability of reversible hair thinning while on treatment (Alopecia) and probability of developing clinical depression while on treatment (Depression). We estimated a mean relative importance weight for each attribute. Patients also answered three rating questions to assess the extent to which treatment attributes might affect adherence. Hundred and fifty patients completed the survey. Efficacy was the most important attribute with a mean relative importance weight of 10 [95% CI: 7.9-12.1]. The remaining attributes were ranked in order of importance as follows: Depression (4.4 [95% CI: 3.6-5.1]), Flu Days(Frequency×Flu) (3.7 [95% CI: 2.2-5.3]), Lost Work Days (2.9 [95% CI: 2.3-3.5]), Alopecia (1.3 [95% CI: 0.7-1.9]) and Device (1.2 [95% CI: 0.4-2.0]). Patients with prior treatment experience were less likely to indicate that treatment attributes would affect adherence. Patients also indicated that increases in the number of flu days would increase the likelihood of nonadherence to treatment. Sustained viral response is the most important treatment attribute to patients but treatment side effects might affect treatment adherence.