Gender similarities and differences in the treatment, relapse, and recovery cycle

This study explores the influence of gender on changes in recovery status among participants in a longitudinal study. The study sample (N = 1,202; 60% female) is recruited on referral to treatment, and annual interviews are conducted from Years 2 to 6 following intake. At each annual observation, participants are classified into one of four statuses (recovery, treatment, incarcerated, and using), and the transitional probabilities and correlates of transitioning from one status to another are estimated. About 80% of the participants changed status at least once over the follow-up period. Women are one third less likely to transition from recovery to using; the predictors of transitioning to different statuses vary by gender. The implications of gender as a moderator of the recovery process are discussed.     

Subgroup analyses of clinical effectiveness to support health technology assessments

Subgroup analysis is an integral part of access and reimbursement dossiers, in particular health technology assessment (HTA), and their HTA recommendations are often limited to subpopulations. HTA recommendations for subpopulations are not always clear and without controversies. In this paper, we review several HTA guidelines regarding subgroup analyses. We describe good statistical principles for subgroup analyses of clinical effectiveness to support HTAs and include case examples where HTA recommendations were given to subpopulations only. Unlike regulatory submissions, pharmaceutical statisticians in most companies have had limited involvement in the planning, design and preparation of HTA/payers submissions. We hope to change this by highlighting how pharmaceutical statisticians should contribute to payers' submissions. This includes early engagement in reimbursement strategy discussions to influence the design, analysis and interpretation of phase III randomized clinical trials as well as meta-analyses/network meta-analyses. The focus on this paper is on subgroup analyses relating to clinical effectiveness as we believe this is the first key step of statistical involvement and influence in the preparation of HTA and reimbursement submissions.     

A case study comparing a randomized withdrawal trial and a double-blind long-term trial for assessing the long-term efficacy of an antidepressant

Assessing long-term efficacy in psychiatric drugs involves a number of complex questions, and the priaority of these questions is different for different disorders and for different stakeholders. Therefore, it is essential that we not adopt a one-method-fits-all approach, but rather adapt the specific details of the designs and analysis of data from long-term trials to individual disease states. Randomized withdrawal (RW) designs, even though addressing a specific question of particular interest, face some difficult methodological and ethical challenges. A less common alternative design, termed the double-blind long-term efficacy (DBLE) design, is logistically similar to traditional responder extension designs. However, use of an analytic approach that includes all randomized patients rather than only the selected subset that continued beyond acute treatment avoids the major criticism of the extender design. The present paper illustrates the attributes of the RW and DBLE designs by analyzing data from trials adopting these designs. The RW and DBLE designs address different questions, and are thus not directly comparable. Potential benefits of the DBLE design include: (1) the parsimonious use of patients and the resultant reduced exposure to placebo as each patient can contribute to multiple developmental objectives; (2) the results are generalizable to actual clinical practice as the design matches treatment guidelines; and, (3) results of safety assessments are meaningful as they involve all randomized patients. Our case study suggests that the DBLE design can provide definitive answers to important questions and may be a useful design for assessing long-term treatment effects.     

Lessons from meta‐analyses of randomized clinical trials for analysis of distributed networks of observational databases

Networks of constellations of longitudinal observational databases, often electronic medical records or transactional insurance claims or both, are increasingly being used for studying the effects of medicinal products in real‐world use. Such databases are frequently configured as distributed networks. That is, patient‐level data are kept behind firewalls and not communicated outside of the data vendor other than in aggregate form. Instead, data are standardized across the network, and queries of the network are executed locally by data partners, and summary results provided to a central research partner(s) for amalgamation, aggregation, and summarization. Such networks can be huge covering years of data on upwards of 100 million patients. Examples of such networks include the FDA Sentinel Network, ASPEN, CNODES, and EU‐ADR. As this is a new emerging field, we note in this paper the conceptual similarities and differences between the analysis of distributed networks and the now well‐established field of meta‐analysis of randomized clinical trials (RCTs). We recommend, wherever appropriate, to apply learnings from meta‐analysis to help guide the development of distributed network analyses of longitudinal observational databases.     

Savings Program Participation and Outcomes for Hispanics

Low- and moderate-income Hispanics in the United States have traditionally experienced few institutional incentives for savings and asset development and have instead encountered many disincentives. The authors provide a brief review of the various enticements and deterrents for savings and wealth development for traditionally underrepresented groups, with a focus on Hispanics. One of the most recent savings initiatives for low- and moderate-income individuals involves dedicated savings accounts, also known as Individual Development Accounts (IDAs). This study was designed to examine whether IDA program completion supports asset development over time for Hispanics. Results indicate that while there are no statistically significant differences in asset growth between IDA completers and non-completers, IDA program completers did experience more growth on all asset measures. The authors provide suggestions on how to close the racial and ethnic wealth gap for low- and moderate-income Hispanic individuals and households.     

Willingness to have unprotected sex

Little is known about people's willingness to engage in sex without protection from unwanted pregnancy. This study surveyed 1,497 women and men at 75 clinics and physician offices across California after their reproductive health care visits in late 2007 and early 2008. When asked if they would have sex without contraception, 30% said definitively that yes, they would have unprotected sex, and 20% indicated they would "sometimes" or "maybe" engage in unprotected sex. In multivariate models, compared to non-Latino White respondents, Latinos who responded to the survey in English were 52% more likely and African Americans were 75% more likely to report willingness to have unprotected intercourse. Wanting a child within the next three years was associated with increased willingness to have unprotected sex. Age, gender, parity, and relationship status were not significant in multivariate models. A considerable proportion of women and men may be willing to have unprotected sex, even with access to subsidized contraceptive services and even when recently counseled about birth control. The dominant behavioral models of contraceptive use need to acknowledge the widespread likelihood of occasional unprotected sex, even among people motivated to usually use contraceptives. Findings underscore the need to make contraceptive methods accessible, easy to use, and even pleasurable.     

"If I can't have you, no one can": development of a Relational Entitlement and Proprietariness Scale (REPS)

Relational proprietariness and entitlement have been theoretically related to partner violence following the threat of relationship dissolution. To date, however, no measure has been employed to verify such accounts. A multistage item pool development and refinement strategy was employed, resulting in a 32-item measure with strong construct validity. An online survey administered to 279 students resulted in an interpretable factor structure of sexual proprietariness and entitlement, consisting of social, behavioral, and information control, and a potential factor of face threat reactivity. These factors added unique variance to the prediction of instrumental and expressive aggression, were related to self-esteem and attachment, and were not contaminated by social desirability. Recommendations for bolstering the face threat reactivity factor and future studies are suggested. This measure provides a new tool that contributes to the prediction of intimate partner violence.