Issues in applying recent CPMP ‘Points to Consider’ and FDA guidance documents with biostatistical implications

The International Conference on Harmonisation guideline ‘Statistical Principles for Clinical Trials’ was adopted by the Committee for Proprietary Medicinal Products (CPMP) in March 1998, and consequently is operational in Europe. Since then more detailed guidance on selected topics has been issued by the CPMP in the form of ‘Points to Consider’ documents. The intent of these was to give guidance particularly to non‐statistical reviewers within regulatory authorities, although of course they also provide a good source of information for pharmaceutical industry statisticians. In addition, the Food and Drug Administration has recently issued a draft guideline on data monitoring committees. In November 2002 a one‐day discussion forum was held in London by Statisticians in the Pharmaceutical Industry (PSI). The aim of the meeting was to discuss how statisticians were responding to some of the issues covered in these new guidelines, and to document consensus views where they existed. The forum was attended by industry, academic and regulatory statisticians. This paper outlines the questions raised, resulting discussions and consensus views reached. It is clear from the guidelines and discussions at the workshop that the statistical analysis strategy must be planned during the design phase of a clinical trial and carefully documented. Once the study is complete the analysis strategy should be thoughtfully executed and the findings reported. Copyright © 2003 John Wiley & Sons, Ltd.     

Risk of Disclosure, Perceptions of Risk, and Concerns about Privacy and Confidentiality as Factors in Survey Participation

This article reports on a web-based vignette experiment investigating how likely subjects would be to participate in surveys varying in topic sensitivity and risk of disclosure. A total of 3,672 participants each responded to a series of eight vignettes, along with a variety of background questions, concerns about confidentiality, trust in various institutions, and the like.Vignettes were randomly assigned to respondents, such that each respondent was exposed to four levels of disclosure risk for each level of topic sensitivity (high versus low). Half the sample was assigned to receive a confidentiality statement for all eight vignettes, while the other half received no mention of confidentiality in the vignettes. The order of presentation of vignettes was randomized for each respondent.Respondents were also asked for their subjective perceptions of risk, harm, and social as well as personal benefits for one of the eight vignettes. Adding these questions permits us to examine how objective risk information presented by the researcher relates to the subjective perception of risk by the participant, and to assess the importance of both for their willingness to participate in the surveys described.Under conditions resembling those of real surveys, objective risk information does not affect willingness to participate. On the other hand, topic sensitivity does have such effects, as do general attitudes toward privacy and survey organizations as well as subjective perceptions of risk, harm, and benefits. We discuss the limitations and implications of these findings.     

Energy and the economy— A reply to Smil and Kuz

In the June 1976 edition of this journal Smil and Kuz discussed the case for saying that there are quite firm relationships between energy consumption and GDP. They argued broadly that there was no general relationship, but, instead, the individual countries are locked into individual patterns of energy consumption. The proponents of the ‘there is a relationship’ school of thought include the author of this reply who is employed in the Economics and Programmes Branch of the U.K. Atomic Energy Authority.     

Child Welfare in the United States: Policy, Practice and Innovations in Service Delivery

Child welfare in the United States is characterized by numerous and complex problems. By reviewing child welfare policy and trends in the United States, this article aims to stimulate discussion about innovative approaches that could allow child welfare professionals, both in the US and internationally, to better meet the needs of children and families.     

Eye-Tracking Data: New Insights on Response Order Effects and Other Cognitive Shortcuts in Survey Responding

Survey researchers since Cannell have worried that respondents may take various shortcuts to reduce the effort needed to complete a survey. The evidence for such shortcuts is often indirect. For instance, preferences for earlier versus later response options have been interpreted as evidence that respondents do not read beyond the first few options. This is really only a hypothesis, however, that is not supported by direct evidence regarding the allocation of respondent attention. In the current study, we used a new method to more directly observe what respondents do and do not look at by recording their eye movements while they answered questions in a Web survey. The eye-tracking data indicate that respondents do in fact spend more time looking at the first few options in a list of response options than those at the end of the list; this helps explain their tendency to select the options presented first regardless of their content. In addition, the eye-tracking data reveal that respondents are reluctant to invest effort in reading definitions of survey concepts that are only a mouse click away or paying attention to initially hidden response options. It is clear from the eye-tracking data that some respondents are more prone to these and other cognitive shortcuts than others, providing relatively direct evidence for what had been suspected based on more conventional measures.     

Children in self-care: An examination of research confounds

Families using self-care, in-home, and out-of-home child care arrangements were compared on a number of demographic variables. Certain variables (e.g., family income; marital status) were, as expected, related to the type of child care in use. However, one unexpected trend also was identified: regardless of the type of child care arrangement reported by parents, as the age of children increased across the out-of-home, in-home, and self-care family groups so too did the number of hours children spent at home alone. This trend is discussed in relation to two confounding areas of which researchers should be aware when conducting school-age child care needs assessment surveys and conducting self-care studies: (a) the context within which the self-care decision is made; and (b) the reasoning that goes into making the self-care decision.     

Narratives of Socialization: Perceptions of Parental Influence After Childhood Divorce

Despite substantial research on the consequences of parental divorce, no studies have investigated offspring discourse about parents’ influences. This study draws on a sample of university students and uses a grounded theory approach to identify four types of socialization narratives. These narratives include (a) fear of unintentional modeling, (b) absence of effective modeling, (c) determination to avoid parental mistakes, and (d) fear of self- or partner change. For the first three types, young adults link perceptions of relationship socialization to a specific parental trait or relationship pattern. The fourth type reflects generalized conclusions about human change over the life course.     

Cross-Cultural Analyses of Determinants of Quality of Life and Mental Health: Results from the Eurohis Study

Recent projects on international instrument development have produced a wide array of health indicators that may be used for cross-cultural field-testing, however more information on their cross-cultural performance in relation to health determinants is necessary. The current study approaches one step for international conceptual validation by analysing the association between various health determinants and different types of health outcomes (mental health, quality of life) across a range of countries or geographical areas. The current study is based on the EUROHIS project that has been conducted in a sample of 4849 adults across 10 European countries. Results highlight that interactions between health determinants with subjective mental health, general health and quality of life (QoL) differ between Western European countries, Eastern European countries and the Newly Associated States as well as Israel. Using a MIMIC model approach, we were able to show that the impact of each of the sociodemographic variables cannot be interpreted on the basis of its single loading but only seeing the interacting with other indicators. Future studies should include sociodemographics in MIMIC models in each latent factor before carrying out regressions on a larger scale. Future investigations will require larger and representative samples to (a) test models on latent factors of mental health and QoL and (b) on the basis of these findings test overall structural models across countries.