Commentary on using the SF-36 or MOS-HIV in studies of persons with HIV disease

The purpose was to compare and comment on use of the SF-36 and MOS-HIV instruments in studies of persons with HIV disease. Three medical information databases were searched to identify examples of HIV studies that included the MOS-HIV or SF-36. Thirty-nine and 14 published articles were identified for illustration in comparing the use of the MOS-HIV and SF-36 in HIV disease, respectively. Support for the reliability and construct validity of the MOS-HIV and SF-36 was found. Ceiling and floor effects were reported for both the MOS-HIV and SF-36; however, ceiling effects were more common for the MOS-HIV, in part due to fewer items in the physical, social, and role functioning domains. The MOS-HIV measures three domains hypothesized to be associated with the health deterioration of HIV disease not measured by the SF-36; however, these domains may not assess aspects of HIV disease that typify the majority of the persons with HIV disease today. National norms for the U.S. adult population (and other nations) are available for the SF-36. In addition, the SF-36 has been used in a wide variety of patient populations, enabling comparisons of HIV-infected persons with persons with other health conditions. No national norms for the MOS-HIV are available. We conclude that there is currently insufficient evidence in the literature to recommend the use of the MOS-HIV over the SF-36 in HIV-infected persons. Although the SF-36 is not targeted at HIV, it may be preferable to use the SF-36 over the MOS-HIV due to fewer ceiling effects, availability of national norms, and the vast amount of data for other populations in the U.S. and around the world. Head-to-head comparisons demonstrating the unique value of the MOS-HIV over the SF-36 are clearly needed. More importantly, additional work needs to be directed at comparing the MOS-HIV and other putatively HIV-targeted instruments to one another to help demarcate aspects of HRQOL that are truly generic versus specific to HIV disease. Using both a generic and targeted HRQOL measure is a good general strategy, but this has not been a typical practice in studies of HIV because the MOS-HIV is so similar in content to the SF-36

PRO Data Collection in Clinical Trials Using Mixed Modes: Report of the ISPOR PRO Mixed Modes Good Research Practices Task Force

AbstractThe objective of this report was to address the use and mixing of data collection modes within and between trials in which patient-reported outcome (PRO) end points are intended to be used to support medical product labeling. The report first addresses the factors that should be considered when selecting a mode or modes of PRO data collection in a clinical trial, which is often when mixing is first considered. Next, a summary of how to "faithfully" migrate instruments is presented followed by a section on qualitative and quantitative study designs used to evaluate measurement equivalence of the new and original modes of data collection. Finally, the report discusses a number of issues that must be taken into account when mixing modes is deemed necessary or unavoidable within or between trials, including considerations of the risk of mixing at different levels within a clinical trial program and mixing between different types of platforms. In the absence of documented evidence of measurement equivalence, it is strongly recommended that a quantitative equivalence study be conducted before mixing modes in a trial to ensure that sufficient equivalence can be demonstrated to have confidence in pooling PRO data collected by the different modes. However, we also strongly discourage the mixing of paper and electronic field-based instruments and suggest that mixing of electronic modes be considered for clinical trials and only after equivalence has been established. If proceeding with mixing modes, it is important to implement data collection carefully in the trial itself in a planned manner at the country level or higher and minimize ad hoc mixing by sites or individual subjects. Finally, when mixing occurs, it must be addressed in the statistical analysis plan for the trial and the ability to pool the data must be evaluated to then evaluate treatment effects with mixed modes data. A successful mixed modes trial requires a "faithful migration," measurement equivalence established between modes, and carefully planned implementation to minimize the risk of increased measurement error impacting the power of the trial to detect a treatment effect

Patient-Reported Outcomes in Cancer Drug Development and US Regulatory Review: Perspectives From Industry, the Food and Drug Administration, and the Patient

Data reported directly by patients about how they feel and function are rarely included in oncology drug labeling in the United States, in contrast to Europe and to nononcology labeling in the United States, where this practice is more common. Multiple barriers exist, including challenges unique to oncology trials, and industry's concerns regarding cost, logistical complexities, and the Food and Drug Administration's (FDA's) rigorous application of its 2009 guidance on the use of patient-reported outcome (PRO) measures. A panel consisting of representatives of industry, FDA, the PRO Consortium, clinicians, and patients was assembled at a 2014 workshop cosponsored by FDA to identify practical recommendations for overcoming these barriers. Key recommendations included increasing proactive encouragement by FDA to clinical trial sponsors for including PROs in drug development programs; provision of comprehensive PRO plans by sponsors to FDA early in drug development; promotion of an oncology-specific PRO research agenda; development of an approach to existing ("legacy") PRO measures, when appropriate (focused initially on symptoms and functional status); and increased FDA and industry training in PRO methodology. FDA has begun implementing several of these recommendations

Complications among colorectal cancer survivors: SF-6D preference-weighted quality of life scores

Background Societal preference-weighted health-related quality of life (HRQOL) scores enable comparing multi-dimensional health states across diseases and treatments for research and policy. Objective To assess the effects of living with a permanent intestinal stoma, compared to a major bowel resection, among colorectal cancer (CRC) survivors. Research Design Cross-sectional multivariate linear regression analysis to explain preference-weighted HRQOL scores. Subjects Six-hundred-forty CRC survivors (≥5 years) from three group-model HMOs; ostomates and non-ostomates with colorectal resections for CRC were matched on gender, age (±5 years), time since diagnosis, and tumor site (rectum vs. colon). Measures SF-6D scoring system applied to Medical Outcomes Study Short Form-36 version 2 (SF-36v2); City of Hope Quality of Life-Ostomy (mCOH-QOL-O); Charlson-Deyo comorbidity index. Methods Survey of CRC survivors linked to respondents’ clinical data extracted from HMO files. Results Response rate was 52%. Ostomates and non-ostomates had similar sociodemographic characteristics. Mean SF-6D score was 0.69 for ostomates, compared to 0.73 for non-ostomates (p <.001), but other factors explained this difference. Complications of initial cancer surgery, and prior-year comorbidity burden and hospital use were negatively associated with SF-6D scores, while household income was positively associated. Conclusions CRC survivors’ SF-6D scores were not associated with living with a permanent ostomy after other factors were taken into account. Surgical complications, comorbidities, and metastatic disease lowered the preference-weighted HRQOL of CRC survivors with and without ostomies. Further research to understand and reduce late complications from CRC surgeries as well as associated depression is warranted

THE EFFECT OF SELF-CARE INFORMATION ON HEALTH-RELATED ATTITUDES AND BELIEFS.

Health-case costs in the United States have been rising at an alarming pace. Therefore, a great deal of emphasis has been placed on finding more cost-effective methods of providing care and maintaining health. An area that warrants investigation is the potential for reducing inappropriate utilization of medical-care resources for relatively minor self-limiting problems. One method of accomplishing this is to provide the proper encouragement, skills, and resources to enable individuals to appropriately involve themselves in self-care activities. A study was conducted to determine if an intervention involving self-care information would change participants' beliefs and attitudes concerning responsibility and involvement in their own health care. Students entering the Student Health Service at the University of Arizona during the study period were randomly assigned to the treatment group or control group. Members of the treatment group received the intervention and were asked to complete a survey instrument. Members of the control group were asked to complete the survey instrument only. The survey instrument consisted of a measurement of attitudes toward information and behavioral involvement in health care (i.e., Krantz Health Opinion Survey) and a measure of beliefs regarding one's ability to exert control over their health (i.e., Multidimensional Health Locus of Control). Also on the instrument was a measure of the individual's behavioral intentions regarding projected use of medical-care practitioners. Results of the study indicated that the intervention was able to change the treatment group's attitudes regarding a greater preference for more active involvement in their health care. Also subsequent to the intervention, the treatment group's responses reflected less of a belief that health was outside of their control. No difference was found between the treatment and control group in regard to behavioral intentions. These results demonstrate that a positive change in health-related beliefs and attitudes can come about as a result of a relatively uncomplicated and inexpensive intervention

Commentary on using the SF-36 or MOS-HIV in studies of persons with HIV disease.

The purpose was to compare and comment on use of the SF-36 and MOS-HIV instruments in studies of persons with HIV disease. Three medical information databases were searched to identify examples of HIV studies that included the MOS-HIV or SF-36. Thirty-nine and 14 published articles were identified for illustration in comparing the use of the MOS-HIV and SF-36 in HIV disease, respectively. Support for the reliability and construct validity of the MOS-HIV and SF-36 was found. Ceiling and floor effects were reported for both the MOS-HIV and SF-36; however, ceiling effects were more common for the MOS-HIV, in part due to fewer items in the physical, social, and role functioning domains. The MOS-HIV measures three domains hypothesized to be associated with the health deterioration of HIV disease not measured by the SF-36; however, these domains may not assess aspects of HIV disease that typify the majority of the persons with HIV disease today. National norms for the U.S. adult population (and other nations) are available for the SF-36. In addition, the SF-36 has been used in a wide variety of patient populations, enabling comparisons of HIV-infected persons with persons with other health conditions. No national norms for the MOS-HIV are available. We conclude that there is currently insufficient evidence in the literature to recommend the use of the MOS-HIV over the SF-36 in HIV-infected persons. Although the SF-36 is not targeted at HIV, it may be preferable to use the SF-36 over the MOS-HIV due to fewer ceiling effects, availability of national norms, and the vast amount of data for other populations in the U.S. and around the world. Head-to-head comparisons demonstrating the unique value of the MOS-HIV over the SF-36 are clearly needed. More importantly, additional work needs to be directed at comparing the MOS-HIV and other putatively HIV-targeted instruments to one another to help demarcate aspects of HRQOL that are truly generic versus specific to HIV disease. Using both a generic and targeted HRQOL measure is a good general strategy, but this has not been a typical practice in studies of HIV because the MOS-HIV is so similar in content to the SF-36