Caregiver priorities for endpoints to evaluate treatments for Prader-Willi syndrome: a best–worst scaling

Abstract

Objectives: Prader-Willi syndrome (PWS) is a rare genetic disorder associated with varying degrees of hyperphagia, obesity, intellectual disability, and anxiety across the affected individuals’ lifetimes. This study quantified caregiver priorities for potential treatment endpoints to identify unmet needs in PWS.

Methods: The authors partnered with the International Consortium to Advance Clinical Trials for PWS (PWS-CTC) and a diverse stakeholder advisory board to develop a best–worst scaling instrument. Seven relevant endpoints were assessed using a balanced incomplete block design. Caregivers were asked to determine the most and least important of a sub-set of four endpoints in each task. Caregivers were recruited nationally though patient registries, email lists, and social media. Best–worst score was calculated to determine caregiver priorities; ranging from 0 (least important) to 10 (most important). A novel kernel-smoothing approach was used to analyze caregiver endpoint priority variations with relation to age of the PWS individual.

Results: In total, 457 caregivers participated in the study. Respondents were mostly parents (97%), females (83%), and Caucasian (87%) who cared for a PWS individual ranging from 4–54 years. Caregivers value treatments addressing hyperphagia (score?=?7.08, SE?=?0.17) and anxiety (score?=?6.35, SE?=?0.16) as most important. Key variations in priorities were observed across age, including treatments targeting anxiety, temper outbursts, and intellectual functions.

Conclusions: This study demonstrates that caregivers prioritize hyperphagia and, using a novel method, demonstrates that this is independent of the age of the person with PWS. This is even the case for parents of young children who have yet to experience hyperphagia, indicating that these results are not subject to a hypothetical bias.     

AIA submission: CEO overconfidence and the incidence of financial restatement

The role of the Chief Executive Officer (hereafter, CEO) in financial reporting is almost universally assumed to be significant (Carcello, Neal, Palmrose & Scholz, 2011; Cohen, Krishnamoorthy, & Wright, 2002; Connelly, 2005; Paredes, 2004). While academics and regulators agree that the CEO can have a large impact on financial reporting decisions, there is very little research on how individual CEO characteristics actually influence the financial reporting process. This paper examines the impact of one such CEO characteristic – CEO overconfidence – on the incidence of financial restatement. We utilize a matched-pairs research design consisting of 75 restatement firms (obtained through the GAO restatement sample) and a set of 75 non-restatement control firms. Using an options-based measure of CEO overconfidence developed by Malmendier and Tate (2008), we document a statistically significant positive relation between CEO overconfidence and financial statement restatement.     

Profiling consumers in home ware stores according to their decision‐making process: an exploratory study

Despite the growing interest of South African consumers regarding home ware, insufficient research is available regarding their decision making of this product category. The objectives of this exploratory study were to evaluate consumers' decision‐making process regarding home ware, and to suggest consumer profiles of the home ware based on the decision‐making process. A qualitative research strategy was used to purposively recruit both male and female participants walking around a home ware store. Semi‐structured interviews were used to collect data from 33 participants at which point data saturation was reached. Each step of the decision‐making process was investigated during the interviews, using questions relevant to the purchasing of home ware. The results indicated clear differences as well as similarities in home ware consumers' decision‐making process. It was evident that the decision‐making process was a result of their specific need for a home ware product. Consumers evaluated home ware based on their needs, values and personal style, while making their final product choice based on product quality, appearance and price. It was also possible to identify home ware consumer profiles based on an evaluation of their decision‐making process. Four profiles were identified, namely, the quality‐conscious, the fashion‐conscious, the lifestyle‐orientated and the impulsive home ware consumer. The characteristics associated with each profile should provide the growing home ware market with insight regarding the specific needs and preferences of the consumer. Seeing that this study was only exploratory in nature, it is recommended that further research regarding the purchasing of home ware should be done.     

The gravity model and trade in intermediate inputs

Is the gravity model as applicable to trade in intermediate inputs as it is to trade in final goods? One of the contributions of this paper is that we explicitly account for the dual nature of products that can be used as either intermediate inputs or final goods. We find that the structural gravity model performs extremely well for describing bilateral trade in final goods and in intermediate inputs. Moreover, this continues to hold even when we focus on a subset of countries in which intermediate inputs trade accounts for a growing share of trade, namely "Factory Asia." However, the gravity model may perform poorly due to model misspecification (i.e., exclusion of intranational trade) and/or sample selection, even after the model considers the dual nature of products. We demonstrate that the poor performance of the gravity model is not attributable to the large trade flow of intermediate inputs, which supports the continued use of the model as these trade flows continue to grow in importance worldwide.     

An economic evaluation of area yield insurance for small-scale cane growers

In the past, crop insurance schemes have based indemnity payments on individual producers' yields. Insurance of individual yields does not cope efficiently with systemic risk and therefore fails in areas where crop yields are correlated across space. Area yield insurance provides an alternative and eliminates the problems of moral hazard and adverse selection associated with individual yield insurance. Area yield insurance therefore effectively copes with systemic risk. In this study, principles of area yield insurance were applied to yield data on small-scale cane growers in KwaZulu-Natal and used to calculate pure premium rates. The viability of a government-subsidised area yield insurance scheme for small-scale cane farmers was assessed in terms of affordability to the government, the farmers and private insurance companies. The empirical results obtained from this study indicate that such a scheme may pose great expense to the government and, as a result, may not be viable in South Africa. This topic needs further study, while other risk management strategies should also be considered.     

Vertical Pay Dispersion,Peer Observability,and Misreporting in a Participative Budgeting Setting

In this study, we examine the joint effect of vertical pay dispersion and peer observability on subordinates' misreporting choices. We adopt a participative budgeting setting in which two subordinates report to one superior, and we manipulate vertical pay dispersion (low/high) and peer observability (absent/present). Subordinates have private information about actual project costs and can over-report project costs to the superior without detection and thus create budgetary slack. When a peer's reporting choices are observable, we predict and find that peer reporting choices have an asymmetric influence on the focal subordinates' reporting choices, and this asymmetric influence depends on the level of vertical pay dispersion. Specifically, we find that when vertical pay dispersion is low, subordinates who observe peer reports containing low slack misreport less, whereas observing peer reports that contain high slack has no significant effect. However, when vertical pay dispersion is high, subordinates who observe peer reports containing high slack misreport more, whereas observing peer reports that contain low slack has no significant effect. Driven by these asymmetric effects, subordinates misreport less in the presence of peer observability than in its absence when vertical pay dispersion is low and misreport more in the presence of peer observability than in its absence when vertical pay dispersion is high. Overall, our findings suggest that when a firm has a more egalitarian pay structure (i.e., low vertical pay dispersion), an open information policy is conducive to a more honest reporting environment, whereas under a more hierarchical pay structure (i.e., high vertical pay dispersion), open information policies can compromise the honesty of subordinates' reports.