Comparing preferences for outcomes of psoriasis treatments among patients and dermatologists in the U.K.: results from a discrete-choice experiment.

BACKGROUND: Plaque psoriasis can have a significant negative effect on patients' quality of life, and treatments can result in serious toxicities. Although there have been several studies of patients' and physicians' relative preferences for the benefits and risks of psoriasis treatments, it is unclear how and whether patients' and physicians' preferences for the outcomes of psoriasis treatments differ. OBJECTIVES: To quantify patient and dermatologist preferences for improvements in psoriasis symptoms and for increases in the risk of treatment-related serious adverse events. METHODS: Members of the U.K. Psoriasis Association and U.K. dermatologists with experience prescribing biologics completed a web-enabled discrete-choice experiment survey in which they evaluated efficacy and safety features of biological treatments for psoriasis. Choices between hypothetical treatment options were used to estimate preference weights indicating respondents' relative trade-off preferences among treatment outcomes. These outcomes included improvements in the severity and coverage of psoriatic plaques and treatment-related risks of tuberculosis, serious infections and lymphoma. Preference estimates were used to derive the maximum level of side-effect risks that respondents would accept for improvements in psoriasis symptoms. RESULTS: Respondents' tolerance for side-effect risks varied with side-effect severity and location of plaques, and risk tolerance for serious side-effects was greater for patients than for dermatologists. CONCLUSIONS: Estimates of patients' risk tolerance for serious side-effects indicate that patients valued psoriasis symptom control highly and suggest that psoriasis symptoms have a significant effect on patients' quality of life. In light of research showing increased treatment satisfaction and improved treatment adherence among patients who receive therapies that are consistent with their preferences, our findings suggest that greater communication between dermatologists and patients about risk tolerance could help improve patient care.

Psychiatrists' judgments about antipsychotic benefit and risk outcomes and formulation in schizophrenia treatment.

OBJECTIVE: The objectives were to quantify psychiatrists' judgments of the benefits and risks of antipsychotic treatments of patients with schizophrenia and to evaluate how patient adherence history affects these judgments. METHODS: Weights assigned by respondents to risks, benefits, and alternative drug formulations in the treatment of schizophrenia were assessed via a Web-based survey by using a discrete-choice experiment. Respondents in the United States and the United Kingdom chose among alternative scenarios characterized by various levels of improvement in positive symptoms, negative symptoms, social functioning, weight gain, extrapyramidal symptoms (EPS), hyperprolactinemia, and hyperglycemia and by formulation. The effect of patient adherence history on respondents' judgments was also assessed. Random-parameters logit and bivariate probit models were estimated. RESULTS: The sample included 394 psychiatrists. Improvement in positive symptoms from "no improvement" to "very much improved" was the most preferred outcome over the range of improvements included and was assigned a relative importance score of 10. Other outcomes, in decreasing order of importance, were improvement in negative symptoms from "no improvement" to "very much improved" (5.2; 95% confidence interval [CI]=4.2-6.2), social functioning from "severe problems" to "mild problems" (4.6, CI=3.8-5.4), no hyperglycemia (1.9, CI=1.5-2.4), <15% weight gain (1.5, CI=.9-2.0), no hyperprolactinemia (1.3, CI=.8-1.6), and no EPS (1.1, CI=.7-1.5). As adherence decreased, formulation became more important than modest efficacy changes and injections were preferred to daily pills (p<.05). CONCLUSIONS: Psychiatrists favored treatments that primarily improve positive symptoms. Choice of formulation became more important as likely adherence declined.

Avoidance of weight gain is important for oral type 2 diabetes treatments in Sweden and Germany: patient preferences.

AIMS: The aim of the study was to quantify patient preferences for outcomes associated with oral antidiabetic medications (OAMs) in Sweden and Germany through a discrete-choice experiment. METHODS: Adults taking OAMs who had a self-reported physician's diagnosis of type 2 diabetes mellitus (T2DM) made a series of nine choices between pairs of hypothetical profiles. Each profile had a predefined range of attributes: blood glucose control, frequency of mild-to-moderate hypoglycaemia, annual severe hypoglycaemic events, annual weight gain, pill burden and frequency of administration, and cost. Choice questions were based on an experimental design with known statistical properties. Bivariate probit analysis estimated the probabilities of choice of medication administration from patient characteristics and, conditional on that choice, preferences for treatment outcomes. RESULTS: The final sample consisted of 188 Swedish and 195 German patients. For both countries, weight gain was the most important attribute, followed by blood glucose control. Avoiding a 5-kg weight gain was 1.5 times more important in Sweden and 2.3 times more important in Germany than achieving moderate blood glucose control, thereby, suggesting that blood glucose control is relatively more important to Swedish than to German patients. Least important outcomes were the number of daily pills (Sweden) and frequency of mild-to-moderate hypoglycaemia (Germany). CONCLUSION: Patients in both Sweden and Germany preferred OAMs not associated with weight gain.

A discrete-choice experiment of United Kingdom patients' willingness to risk adverse events for improved function and pain control in osteoarthritis.

OBJECTIVE: To assess patient preferences for treatment-related benefits and risks associated with the use of nonsteroidal anti-inflammatory drugs (NSAIDs) in the management of osteoarthritis (OA). DESIGN: Using a chronic-illness panel in the United Kingdom, patients 45 years or older with a self-reported diagnosis of OA were eligible to participate in the study. Patient preferences were assessed using a discrete-choice experiment that compared hypothetical treatment profiles of benefits and risks consistent with NSAID use. Benefit outcomes (ambulatory pain, resting pain, stiffness, and difficulty doing daily activities) were presented on a 0-to-100 mm scale. Risk outcomes (bleeding ulcer, stroke, and myocardial infarction [MI]) were expressed as probabilities over a fixed time period. Each patient answered 10 choice tasks comparing different treatment profiles. Preference weights were estimated using a random-parameters logit model. RESULTS: Final sample included 294 patients. Patients ranked reductions in ambulatory pain and difficulty doing daily activities (both: 6.32; 95% confidence interval [CI]: 5.0-7.6) as the most important benefit outcomes, followed by reductions in resting pain (2.80; 95% CI: 1.8-3.8) and stiffness (2.65; 95% CI: 0.9-4.4). Incremental changes (3%) in the risk of MI or stroke were assessed as the most important risk outcomes (10.00; 95% CI: 8.2-11.8; and 8.90; 95% CI: 7.3-10.5, respectively). CONCLUSION: Patients ranked ambulatory pain as a more important benefit than resting pain; likely due to its impact on ability to do daily activities. For a 25-mm reduction, patients were willing to accept four times the risk of MI in ambulatory pain vs resting pain.

Treatment preferences and medication adherence of people with Type 2 diabetes using oral glucose-lowering agents.

AIMS: Medication non-adherence is particularly common in patients with Type 2 diabetes. We constructed a discrete-choice experiment to examine the relative importance of oral glucose-lowering medication features and to estimate the likely effect of effectiveness and side effects on medication adherence in patients with Type 2 diabetes in the UK and the USA. METHODS: Preferences were elicited using a cross-sectional, web-enabled survey. Patients with a self-reported physician-made diagnosis of Type 2 diabetes, who were currently taking oral glucose-lowering medications were recruited through an existing online chronic-disease panel. In each discrete-choice question, patients were asked to choose between two hypothetical medication alternatives, each defined by improvement in glycated haemoglobin, frequency of mild-to-moderate hypoglycaemia, water retention, weight gain, mild stomach upset and medication-related cardiovascular risk. Patients were also asked to indicate how likely they would be to miss or skip doses of each hypothetical medication. RESULTS: Two hundred and four patients in the UK and 203 patients in the USA completed the survey. Preferences did not differ between the two countries. Overall, glucose control was the most important medication feature, followed by medication-related cardiovascular risk and weight gain, respectively. Water retention was not important to patients. Weight gain and cardiovascular risk had significant negative effects on likely medication adherence. CONCLUSIONS: While patients with Type 2 diabetes believe glucose control is important, medication side effects and risks influence patients' treatment choices. Medication-related weight gain and cardiovascular risk are significant predictors of likely medication non-adherence.

Modeling choice behavior for new pharmaceutical products.

This paper presents a dynamic generalization of a model often used to aid marketing decisions relating to conventional products. The model uses stated-preference data in a random-utility framework to predict adoption rates for new pharmaceutical products. In addition, this paper employs a Markov model of patient learning in drug selection. While the simple learning rule presented here is only a rough approximation to reality, this model nevertheless systematically incorporates important features including learning and the influence of shifting preferences on market share. Despite its simplifications, the integrated framework of random-utility and product attribute updating presented here is capable of accommodating a variety of pharmaceutical marketing and development problems. This research demonstrates both the strengths of stated-preference market research and some of its shortcomings for pharmaceutical applications.

Willingness to pay for improved respiratory and cardiovascular health: a multiple-format, stated-preference approach.

This study uses stated-preference (SP) analysis to measure willingness to pay (WTP) to reduce acute episodes of respiratory and cardiovascular ill health. The SP survey employs a modified version of the health state descriptions used in the Quality of Well Being (QWB) Index. The four health state attributes are symptom, episode duration, activity restrictions and cost. Preferences are elicited using two different SP formats: graded-pair and discrete-choice. The different formats cause subjects to focus on different evaluation strategies. Combining two elicitation formats yields more valid and robust estimates than using only one approach. Estimates of indirect utility function parameters are obtained using advanced panel econometrics for each format separately and jointly. Socio-economic differences in health preferences are modelled by allowing the marginal utility of money relative to health attributes to vary across respondents. Because the joint model captures the combined preference information provided by both elicitation formats, these model estimates are used to calculate WTP. The results demonstrate the feasibility of estimating meaningful WTP values for policy-relevant respiratory and cardiac symptoms, even from subjects who never have personally experienced these conditions. Furthermore, because WTP estimates are for individual components of health improvements, estimates can be aggregated in various ways depending upon policy needs. Thus, using generic health attributes facilitates transferring WTP estimates for benefit-cost analysis of a variety of potential health interventions.

Eliciting stated health preferences: an application to willingness to pay for longevity.

The economic analysis of many health policies requires evaluation of the benefits of programs that may prolong human lives. This article contributes to the development of credible values for longevity, demonstrating the feasibility of applying stated-preference market-research techniques to a new area of preference revelation and framing the problem as extending longevity under realistic health states associated with advanced age. Respondents to the authors' stated-preference survey clearly indicated that quality of life affects the value of quantity of life. The results demonstrate the sensitivity of life-extension values to specific health and activity-limitation conditions. The article also discusses problems that remain to be solved before valid and reliable longevity values can be obtained.

Valuing morbidity: an integration of the willingness-to-pay and health-status index literatures.

Placing dollar values on human health has long been a controversial aspect of policy analysis and remains difficult given the relatively small number of morbidity-valuation studies available. By combining both the economic and health literature, this paper offers an alternative approach to morbidity valuation and provides estimates for a wide range of short-term health conditions.

Erythrokeratoderma variabilis: case report and review of the literature.

A 5-month-old boy with erythrokeratoderma variabilis is presented. The parents noted that the asymptomatic erythematous plaques changed over the course of hours to days. Topical therapy with retinoic acid proved ineffective. A brief review of the literature is presented.

Radon risk communication research: practical lessons.

Those responsible for state and local radon programs often express frustration about the small share of homes that have been tested for radon, and the small share of those with high readings that have been mitigated. There are now a number of completed studies that have examined how well alternative ways of communicating about radon risk have accomplished the goals of motivating appropriate testing and mitigation. This paper summarizes the research results that are most crucial for planning and implementing effective radon risk communication programs. We identify six reasons why people do not respond to radon as a serious threat and provide some remedies suggested by radon studies.

Economic costs of misinforming about risk: the EDB scare and the media.

This study reports results of an analysis of consumer responses to news reports of grain-product contamination by the pesticide ethylene dibromide (EDB). The results demonstrate that it is possible to quantify market disruption related to the dissemination of risk information. Implications include the need for increased awareness among risk managers that public perceptions, regardless of their objective accuracy, can induce real economic costs. Such costs should be considered in designing regulatory and information policies.

Radon risk information and voluntary protection: evidence from a natural experiment.

This study examines the perceived risks and mitigating behavior of Maine households who received new information on their exposures to significant health risks from indoor radon. The observed responses of these households illustrate conceptual issues related to designing an effective risk information program. Despite the involvement of generally well-motivated homeowners and well-intentioned researchers and government officials, we conclude that the risk information approach used in Maine failed to induce appropriate, cost-effective voluntary protection. The results indicate that, after receiving radon test results, information on associated health risks, and suggestions on how to reduce exposures: perceived risks tended to understate objective risks by orders of magnitude, and there was no statistically significant relationship between mitigating behavior and objective risks. These results suggest that the formation of risk perceptions and subsequent behavioral adjustments involve complex interactions among information, contextual, socioeconomic, and psychological variables. Therefore, government programs that seek to reduce health and safety risks with information programs, instead of using more conventional enforced standards, must be crafted very carefully to accommodate this complex process.

Esophageal atresia with double tracheoesophageal fistula.

Ninety cases of esophageal atresia (EA) with double tracheoesophageal fistula (TEF) from the literature and 4 new cases are examined. The incidence may be more common than is generally recognized with incidences up to 5.3% reported. The pre-, intra-, and postoperative diagnosis may be difficult, and half have been missed initially with almost half of these being first recognized at autopsy. During repair of EA and TEF the proximal esophagus should be mobilized looking for a proximal fistula. Many proximal TEF are missed at the initial operation and discovered in the postoperative period. The symptoms, diagnosis and treatment of the unrecognized proximal TEF are similar to that for isolated TEF. Results should be good since this problem occurs in large babies with fewer and less complex associated conditions.

Acute hemorrhagic pancreatitis and pseudocyst due to mumps.

This is the first reported case of mumps hemorrhagic pancreatitis in a child, documented at operation, and by rising mumps titers and complicated by a pseudocyst. In severe cases when surgery is indicated, drainage of the lesser sac usually prevents pseudocyst formation. If a pseudocyst occurs, drainage is required to eliminate disabling pain and chronic pancreatitis. Live attenuated mumps vaccine may eliminate this source of pancreatitis from American children in the future.