Community-Based Participatory Research: Incorporating the Integral Voice of Community in Study Design


Impact of Elagolix on Workplace and Household Productivity Among Women with Moderate to Severe Pain Associated with Endometriosis: A Pooled Analysis of Two Phase III Trials

Abstract

Background

Endometriosis profoundly impairs women’s workplace and household productivity.

Objective

The aim of this study was to evaluate the impact of elagolix on endometriosis-related workplace and household productivity losses.

Methods

Data were pooled from two phase III trials of women aged 18–49 years with moderate to severe endometriosis-associated pain treated for 6 months with elagolix 150 mg daily (QD), 200 mg twice daily (BID), or placebo. The Health-Related Productivity Questionnaire was administered at baseline, Month 3, and Month 6 to determine workplace and household absenteeism and presenteeism. Productivity changes from baseline were compared between placebo and elagolix doses via analysis of covariance.

Results

Workplace analyses included 1270 employed women and household analyses included 1565 women. At baseline, women reported average weekly losses of 16 workplace hours, 8.3 household work hours, 45% of scheduled work, and 64% of planned household chores. At Month 6, treatment with elagolix 150 mg QD or 200 mg BID increased productive workplace hours by 1.7 (95% CI 0.1–3.4; p = 0.041) and 5.4 h (95% CI 3.7–7.1; p < 0.001) relative to placebo, corresponding to gains of 5.2% (95% CI 0.7–9.7; p = 0.022) and 14.6% (95% CI 10.0–19.1; p < 0.001) of scheduled work, respectively. Both elagolix doses improved household productivity at Month 6 by 1.7 (95% CI 0.7–2.7) and 3.1 (95% CI 2.1–4.0) hours relative to placebo (both p < 0.001), with increases of 8.8% (95% CI 3.5–14.1; p = 0.001) and 20.4% (95% CI 15.1–25.6; p < 0.001) of planned household work.

Conclusions

Treatment with elagolix improved endometriosis-related workplace and household productivity impairments.

Trial Registration

ELARIS EM-I (NCT01620528) and ELARIS EM-II (NCT01931670)

What Matters Most for Treatment Decisions in Hepatitis C: Effectiveness, Costs, and Altruism

Abstract

Objective

Comparative evaluations of innovations in hepatitis C virus (HCV) drug therapy typically focus on sustained virologic response (SVR) without addressing psychological and socioeconomic challenges that extend beyond virologic cure. This study aims to identify and prioritize variables important to patients when making the decision to start HCV treatment.

Methods

A three-round Delphi process was conducted with the first round derived from a systematic literature review and advisory board input, including patients who have been affected by HCV, physicians, pharmacists, and a patient group representative. Delphi panelists were HCV patients who had received treatment or were considering treatment. Panelists were asked about factors influencing their HCV treatment decisions. Thematic analysis of open-ended responses based on grounded theory was used. Agreement with each category and rankings based on order of importance from the patient perspective was reported.

Results

Treatment effectiveness (100% agreement), longer life (88%), fear of complications (84%), financial issues (80%), quality of life (100%), and impact on society (80%) were considered important factors to patients in decisions to seek treatment. A fear of harming others (87%) was considered more important than physical symptoms (83%) in terms of patient-reported problems caused by HCV. Medication costs (91%) were identified as the most important costs of having HCV, followed by doctor costs (77%).

Conclusions

In addition to treatment effectiveness, patient experiences with financial problems, quality of life, and altruistic desires impact HCV patients’ decisions. The risk of infecting others may motivate patients to seek treatment as much as personally experienced physical symptoms.

Does Device or Connection Type Affect Health Preferences in Online Surveys?

Abstract

Background and Objective

Recent evidence has shown that online surveys can reliably collect preference data, which markedly decrease the cost of health preference studies and expand their representativeness. As the use of mobile technology continues to grow, we wanted to examine its potential impact on health preferences.

Methods

Two recently completed discrete choice experiments using members of the US general population (n = 15,292) included information on respondent device (cell phone, tablet, Mac, PC) and internet connection (business, cellular, college, government, residential). In this analysis, we tested for differences in respondent characteristics, participation, response quality, and utility values for the 5-level EQ-5D (EQ-5D-5L) by device and connection.

Results

Compared to Mac and PC users, respondents using a cell phone or tablet had longer completion times and were significantly more likely to drop out during the surveys (p < 0.001). Tablet users also demonstrated more logical inconsistencies (p = 0.05). Likewise, respondents using a cellular internet connection exhibit significantly less consistency in their health preferences. However, matched samples for tablets and cell phones produced similar EQ-5D-5L utility values (mean differences < 0.06 on a quality-adjusted life-year [QALY] scale for all potential health states).

Conclusion

Allowing respondents to complete online surveys using a cell phone or tablet or over a cellular connection substantially increases the diversity of respondents and the likelihood of obtaining a representative sample, as many individuals have cell phones but not a computer. While the results showed systematic variability in participation and response quality by device and connection type, this study did not show any meaningful changes in utility values.

Preferences for Use and Design of Electronic Patient-Reported Outcomes in Patients with Chronic Obstructive Pulmonary Disease

Abstract

Objectives

Collection of patient-reported outcome (PRO) measures is critical to fully understand chronic obstructive pulmonary disease (COPD) management and progression, as the impact on health-related quality of life is not well understood by objective measures alone. Electronic PROs (ePROs) are increasingly used because of their advantages over paper data collection, including elimination of transcription errors, increased accuracy and data quality, real-time data reporting, and increased compliance. The objective of this study was to characterize how patients with COPD prefer to use various types of technology to report disease symptoms, and their preferences for ePRO design and display.

Methods

The sample consisted of subjects with COPD (N = 103) who completed in-person surveys on their ePRO preferences.

Results

The majority of subjects prefer to use a form of electronic media over paper to report their disease symptoms. Of these electronic methods, subjects most often prefer to use a smartphone provided by their physician. Subjects were also interested in ePRO features, such as knowing estimated PRO completion time at the outset, tracking their progress in real time as they complete a questionnaire, seeing the data that they report in order to track their health status, being encouraged to complete their diary if they fall behind by positive messaging, and being thanked for their completion of a daily diary.

Conclusions

Investigators should consider including these preferences when designing ePRO assessments. Incorporating patient preferences for ePRO design can ultimately help reduce patient burden and increase engagement, compliance, and improve data quality.

Exploring the Impact of Infusion Frequency in Hemophilia A: Exit Interviews with Patients Participating in BAY 94-9027 Extension Studies (PROTECT VIII)

Abstract

Introduction

Prophylactic treatment of severe hemophilia A is burdensome, requiring frequent intravenous injections. Extended half-life (EHL) factor VIII replacement therapies offer longer intervals between infusions while still meeting efficacy and safety outcomes; however, patient perspectives following long-term use of such products in the real-world remain unknown.

Objective

We aimed to explore the importance of infusion frequency and the potential benefits of reduced infusion frequency among patients receiving prophylactic treatment with an EHL product (BAY 94-9027).

Methods

Patients with severe hemophilia A participating in the PROTECT VIII extension study were invited to participate in a semi-structured, concept elicitation ‘exit’ interview to discuss their experiences. Participants were recruited from Israel, The Netherlands, and the US. Interview transcripts were translated into English and analyzed using thematic analysis methods.

Results

Sixteen participants (29–68 years of age) infusing with BAY 94-9027 once every 7 days, once every 5 days, or twice weekly were interviewed. Participants reported infusion frequency (alongside efficacy) as the most important treatment attribute influencing their satisfaction with therapy. Patient-reported benefits of reduced infusion frequency and longer duration of factor coverage included greater ability to participate in physical activities; better vein health; less time to schedule and administer factor VIII; reduced impact on work; and improved emotional well-being.

Conclusions

This study provides rich insights into the experiences of patients with EHL products and the value of reduced infusion frequency. Such data could be of value to a range of stakeholders (e.g. regulators, payers) and facilitate patient–clinician discussions to promote tailored treatment decisions.

Effect of Treatment Preference in Randomized Controlled Trials: Systematic Review of the Literature and Meta-Analysis

Abstract

Background

A significant limitation of the traditional randomized controlled trials is that strong preferences for (or against) one treatment may influence outcomes and/or willingness to receive treatment. Several trial designs incorporating patient preference have been introduced to examine the effect of treatment preference separately from the effects of individual interventions. In the current study, we summarized results from studies using doubly randomized preference trial (DRPT) or fully randomized preference trial (FRPT) designs and examined the effect of treatment preference on clinical outcomes.

Methods

The current systematic review and meta-analysis was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Studies using DRPT or FRPT design were identified using electronic databases, including PubMed, Cochrane Library, EMBASE, and Google Scholar between January 1989 and November 2018. All studies included in this meta-analysis were examined to determine the extent to which giving patients their preferred treatment option influenced clinical outcomes. The following data were extracted from included studies: study characteristics, sample size, study duration, follow-up, patient characteristics, and clinical outcomes. We further appraised risk of bias for the included studies using the Cochrane Collaboration’s risk of bias tool.

Results

The search identified 374 potentially relevant articles, of which 27 clinical trials utilized a DRPT or FRPT design and were included in the final analysis. Overall, patients who were allocated to their preferred treatment intervention were more likely to achieve better clinical outcomes [effect size (ES) = 0.18, 95% confidence interval (CI) 0.10–0.26]. Subgroup analysis also found that mental health as well as pain and functional disorders moderated the preference effect (ES = 0.23, 95% CI 0.11–0.36, and ES = 0.09, 95% CI 0.03–0.15, respectively).

Conclusions

Matching patients to preferred interventions has previously been shown to promote outcomes such as satisfaction and treatment adherence. Our analysis of current evidence showed that allowing patients to choose their preferred treatment resulted in better clinical outcomes in mental health and pain than giving them a treatment that is not preferred. These results underline the importance of incorporating patient preference when making treatment decisions.

Behavioral Economic Insights to Improve Medication Adherence in Adults with Chronic Conditions: A Scoping Review

Abstract

Background and Objective

Medication adherence is poor in patients with chronic conditions. Behavioral economic interventions may reduce biases that are associated with poor adherence. The objective of this review is to map the available evidence on behavioral economic interventions to improve medication adherence in adults with chronic conditions in high-income settings.

Methods

We conducted a scoping review and reported the study using the Joanna Briggs Institute Reviewers’ Manual and the PRISMA Extension for Scoping Review checklist. We searched PubMed, EMBASE, SCOPUS, PsycINFO, EconLit, and CINAHL from database inception to 29 August, 2018 for peer-reviewed studies and included a search of the gray literature. Data on study characteristics, study design, and study outcomes were extracted by one reviewer. Twenty-five percent of the studies were verified by a second reviewer.

Results

Thirty-four studies, targeting diabetes mellitus, human immunodeficiency virus, and cardiovascular and renal diseases met our inclusion criteria. All but two studies were from the USA. The majority of interventions used financial incentives, often in conjunction with other behavioral economic concepts. Non-financial interventions included framing, social influences, reinforcement, and feedback. The effectiveness of interventions was mixed.

Conclusions

Behavioral economic informed interventions show promise in terms of improving medication adherence. However, there is no single simple intervention. This review highlighted the importance of targeting non-adherent patients, understanding their reasons for non-adherence, providing reminders and feedback to patients and physicians, and measuring clinical outcomes in addition to medication adherence. Further research in settings that differ from the US health system is needed.

Advancing the Use of Patient Preference Information as Scientific Evidence in Medical Product Evaluation: A Summary Report of the Patient Preference Workshop