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.     

Did the Proposed Country-of-Origin Law Affect Product Choices? The Case of Salmon

The 2002 Farm Securit and Rural Investment Act mandated that country-of-origin labeling (COOL) be applied to specific food commodities. Using revealed preference data for 1998–2006, a nonlinear AIDS model is used to estimate the demand for uncooked frozen, uncooked fresh, and precooked salmon to assess the impact of the legislation on consumer demand patterns. Findings from a non parametric analysis suggest a possible rotation in the demand curve for fresh salmon that may be linked with the quality signal associated with COOL. However, our findings indicate that COOL had no significant impact on overall consumer demand for the three products. All three were found to be inelastic, with uncooked fresh salmon being slightly more price sensitive than uncooked frozen and precooked salmon. Given the expenditure elasticities, the fresh salmon segment of the industry appear to be more vibrant than the other salmon segments and would likely influence investment decisions in the salmon industry.     

Developing a Marketing Strategy for Nonprofit Organizations: An Exploratory Study

Nonprofit organizations have grown tremendously in the last three decades. With this growth has come a greater interest from the nonprofit sector in the importance of marketing. Nonprofits did not apply marketing techniques until 1960–1970, but it is now a well accepted practice. Traditional marketing strategies do not work for nonprofit organizations, and this study proposes the development of a new marketing strategy specifically for this sector. Through the use of interviews and surveys, the authors examine issues of marketing strategy that are distinct for nonprofits. Unlike previous studies, this study examines these issues from the viewpoint of the nonprofit organization. The perception of marketing is different in nonprofit organizations, and the strategic implications of this finding are discussed.     

Effect of Approved Destination Status on Mainland Chinese Travel Abroad

Approved Destination Status (ADS) agreements facilitate international leisure travel by Chinese citizens, allowing authorized travel agencies to offer group tour arrangements, including visa applications. The number of destinations with ADS agreements increased from 2 in 1983 to 114 in 2012. Using panel data for Chinese visitor arrivals in 58 countries from 1985 to 2005, we estimate a semi‐parametric matched difference‐in‐differences model of the impact of ADS on overseas departures from mainland China. Treatment with ADS increased Chinese visitor arrivals in the 3‐year period following ADS designation by more than 10 percent annually, with results varying substantially across countries.     

Three types of organizational boundary spanning: Predicting CSR policy extensiveness among global consumer products companies

As part of the rise of a worldwide corporate social responsibility (CSR) movement, companies have increasingly incorporated social and environmental concerns into their policies. This paper examines the extensiveness of these policies, proposing that an underappreciated contributor is the degree of organizational boundary spanning. The paper is novel in integrating multiple types of boundary spanning into a single empirical framework, including product, sub‐unit, and national boundary spanning. The paper adds complexity to the literature by theorizing that different types of boundary spanning associate with CSR policy extensiveness in different issue areas. The results show that product spanning associates with CSR policy extensiveness in the area of consumers, sub‐unit spanning in the areas of workers, and nation‐state spanning in all issue areas. A unique, comprehensive, and global data set of 2,714 prominent consumer goods companies in the GoodGuide database underpins these findings.     

TESTING THE FISHERIAN HYPOTHESIS: SOME METHODOLOGICAL ISSUES AND FURTHER EVIDENCE FOR THE UK

The relationship between common stock returns and actual, expected and unexpected inflation has been subject to considerable scrutiny recently. The objectives of this paper are threefold. First, it is suggested that the Fisherian hypothesis of anticipated stock market returns has been subjected to inappropriate tests in some previous work. Second, the Fisherian hypothesis is developed into an ex post form which is suitable for estimation and which will avoid the difficulties in previous work. Thirdly, estimates of the model are reported using UK data which give some support to the hypothesis.     

Buyer information and the hedonic: The impact of a seller disclosure on the implicit price for airport noise

Revealed preference methods like the hedonic model generally assume economic agents have access to publicly available information and use it effectively. In the housing market, the recent proliferation of seller disclosure laws suggests that policymakers perceive buyers to be less than “fully informed,” presumably since they face higher information acquisition costs than sellers. The introduction of an airport noise disclosure in the residential housing market surrounding the Raleigh–Durham International Airport is used as a quasi-random experiment to analyze the impact of this type of information asymmetry between buyers and sellers on housing prices. The results from a regression analysis that controls for potential spatial and temporal confounders, suggest that the airport noise disclosure reduced the value of houses most heavily impacted by airport noise by 2.9 percent. This represents approximately a 37 percentage point increase in the implicit price of airport noise. The results provide evidence that publicly available information, such as that available for airport noise, may not be adequately considered by all buyers. They also suggest that the information environment should be carefully considered when using housing data and the hedonic model to value urban amenities and disamenities.