Tara Symonds

The impact of measuring patient-reported outcomes in clinical practice: a systematic review of the literature

ObjectiveThe purpose of this paper is to summarize the best evidence regarding the impact of providing patient-reported outcomes (PRO) information to health care professionals in daily clinical practice.MethodsSystematic review of randomized clinical trials (Medline, Cochrane Library; reference lists of previous systematic reviews; and requests to authors and experts in the field).ResultsOut of 1,861 identified references published between 1978 and 2007, 34 articles corresponding to 28 original studies proved eligible. Most trials (19) were conducted in primary care settings performed in the USA (21) and assessed adult patients (25). Information provided to professionals included generic health status (10), mental health (14), and other (6). Most studies suffered from methodologic limitations, including analysis that did not correspond with the unit of allocation. In most trials, the impact of PRO was limited. Fifteen of 23 studies (65%) measuring process of care observed at least one significant result favoring the intervention, as did eight of 17 (47%) that measured outcomes of care.ConclusionsMethodological concerns limit the strength of inference regarding the impact of providing PRO information to clinicians. Results suggest great heterogeneity of impact; contexts and interventions that will yield important benefits remain to be clearly defined.

Estimating clinically significant differences in quality of life outcomes

Objective: This report extracts important considerations for determining and applying clinically significant differences in quality of life (QOL) measures from six published articles written by 30 international experts in the field of QOL assessment and evaluation. The original six articles were presented at the Symposium on Clinical Significance of Quality of Life Measures in Cancer Patients at the Mayo Clinic in April 2002 and subsequently were published in Mayo Clinic Proceedings. Principal findings: Specific examples and formulas are given for anchor-based methods, as well as distribution-based methods that correspond to known or relevant anchors to determine important differences in QOL measures. Important prerequisites for clinical significance associated with instrument selection, responsiveness, and the reporting of QOL trial results are provided. We also discuss estimating the number needed to treat (NNT) relative to clinically significant thresholds. Finally, we provide a rationale for applying group-derived standards to individual assessments. Conclusions: While no single method for determining clinical significance is unilaterally endorsed, the investigation and full reporting of multiple methods for establishing clinically significant change levels for a QOL measure, and greater direct involvement of clinicians in clinical significance studies are strongly encouraged.

Practical Guidelines for Assessing the Clinical Significance of Health-Related Quality of Life Changes within Clinical Trials

Health-related quality of life (HRQOL) assessment is becoming common practice in many clinical trials. There is much debate over how to determine the clinical significance of changes in HRQOL scores. A number of techniques have been used to address this issue. This paper reviews the most popular of these approaches for use in a clinical trial setting. More specifically, the anchor-based “minimal clinically important difference” technique is described and critiqued, as is the more traditional distribution-based effect size technique. A novel application of effect size, which applies a common statistical premise known as the empirical rule, is also presented. The review of these techniques indicates that there is no single, optimal solution to determining clinical significance of changes in HRQOL scores. However, it is encouraging to note that they ail suggest a similar criterion of a half-standard deviation for whether or not a change in HRQOL score is clinically significant. Recommendations are given for reporting the clinical significance of HRQOL assessments in clinical trials.

How does premature ejaculation impact a man s life

No systematic study has examined the psychological impact of premature ejaculation (PE) on the man and his partner. This study explores this vital issue by reporting on interviews of 28 men with self-diagnosed PE. From a qualitative perspective, these interviews assess whether these men had concerns about their PE and, if so, what they were. These men focused on two major themes: impact on self-confidence and future/current relationships. This suggests that PE has a similar qualitative impact on the individual as erectile dysfunction. Further investigation will need to determine how prevalent these concerns are in the PE population and also to delineate the impact on the men’s partners.

Assessing clinical significance in measuring oncology patient quality of life: Introduction to the symposium, content overview, and definition of terms

The Clinical Significance Consensus Meeting Group of the Symposium on the Clinical Significance of Quality-of-Life Measures in Cancer Patients produced 6 articles regarding the clinical significance of quality of life (QOL) assessments in oncology. The 6 articles deal with the methods used to date: group-vs-individual clinical significance; single items vs summated scores; patient, clinician, and population perspectives; assessment of changes over time; and communication of results. The articles were produced by a team of 30 QOL research experts assembled in a consensus writing meeting held at the Mayo Clinic, Rochester, Minn, October 6 and 7, 2000. This introduction describes the need for the articles, definitions of key terms, and plans for the future. It is hoped that this series of articles will serve as a resource for individuals conducting cancer QOL research and for clinicians considering incorporation of QOL assessment in the treatment of patients with neoplastic diseases. A secondary goal is to stimulate further discussion and research in the interpretation of QOL assessments.

The clinical significance of quality of life assessments in oncology: a summary for clinicians

BackgroundA series of six manuscripts with an introduction appeared in the Mayo Clinic Proceedings, based upon the collective effort of 30 individuals with an interest and expertise in assessing the clinical significance of quality of life (QOL) assessments. The series of manuscripts described the state of the science of QOL assessments in oncology clinical research and practice and included extensive literature and theoretical justification for the continued inclusion of QOL in oncology clinical research and practice.ObjectivesThe purpose of this paper is to produce a summary of these articles and to supplement these works with additional information that was gleaned from subsequent meetings and discussions of this material. The primary aim of this paper is to present a cogent and concise description for clinicians to facilitate the incorporation of QOL assessments into oncology clinical research and practice. The theoretical discussion is supplemented with an example of how the various ideas can be operationalized in an oncology clinical trial.

Exploration of the Value of Health-Related Quality-of-Life Information From Clinical Research and Into Clinical Practice

Quality-of-life (QOL) instruments used in clinical research can provide important evidence to inform decisions about alternative treatments. This is particularly true when patients, such as those with cancer who are contemplating toxic chemotherapy, face tradeoffs between quantity of life and QOL or when the primary goal of therapy is to improve how patients feel. Surrogate measures (cardiac function, exercise capacity, bone density, tumor size) are inadequate substitutes for direct measurement of QOL. Quality-of-life measures will be most valuable when they comprehensively measure aspects of QOL that are both important to patients and likely to be influenced by therapy, when the QOL measurement instruments are valid (measuring what is intended) and responsive (able to detect all important changes, even if small), and when the results are readily interpretable (determining whether treatment-related changes are trivial, small but important, or large). Researchers are finding new, imaginative ways to help clinicians understand the magnitude of treatment impact on QOL. Additionally, QOL measures may be useful in clinical practice. Recent results from well-designed randomized controlled trials suggest that information on patient QOL provided to clinicians might, in some circumstances, result in benefits for these patients. Further investigation is warranted to confirm these observations and to define the particular combination of methods and settings most likely to yield important benefits.

Development of a questionnaire on sexual quality of life in women

The Sexual Quality of Life–Female (SQOL-F) questionnaire has been developed to assess the impact of female sexual dysfunction (FSD) on a womans sexual quality of life. SQOL-F items were developed through interviews with 82 women. Three data sets from womens health surveys in the United Kingdom and the United States generated data for scale validation. The SQOL-F showed good psychometric properties: convergent validity, discriminant validity, and test-retest reliability. The SQOL-F is a valid instrument for assessing the impact of FSD on quality of life and as an adjunct in evaluating FSD in clinical trials. The SQOL-F sensitivity to changes in sexual function needs confirmation.

Outcome measurement in Female Sexual Dysfunction clinical trials: Review and recommendations

Abstract Defining and measuring Female Sexual Dysfunction (FSD) is a complex and challenging task. Several factors have confounded the theory and measurement of FSD including: the use of an inappropriate male paradigm; difficulty in capturing the complexity of womens sexual response; an evolving but presently untested nosology; and the relative independence between subjective and objective aspects of womens sexual response. Each of these factors have contributed to the difficulty in developing meaningful and valid endpoints for clinical trials. The Food and Drug Administrations (FDA) 2000 draft guidance document for female sexual dysfunction clinical trials recommended the use of daily diary measures as primary and self-administered questionnaires (SAQs) as secondary endpoints. Event logs or diary measures may be adequate for assessing aspects of male sexual performance (e.g., erectile function), or in other therapeutic areas with discrete and readily observable endpoints (e.g., incontinence). However, psychometric theory suggests that for female sexual dysfunction clinical trials, SAQ instruments may provide more sensitive and reliable measures of outcome. We offer an alternative set of recommendations in the hope that the FDA will reconsider its position and to serve as potential guidelines for non-industry sponsored research on female sexuality as well. First, we propose that SAQs be elevated from their current status as secondary endpoints to be considered as potential primary endpoints in clinical trials of FSD. Second, we recommend that depending on the trial design and intervention under study, either an SAQ or diary measure (typically one or the other, and not both), might serve as a primary endpoint in a clinical trial. Third, SAQs and diaries should be employed, analyzed and interpreted in their particular areas of strength. Diaries are most useful for enumerating events and/or counting frequencies. SAQs are superior at gathering subjective data related to womens sexual function. Fourth, we believe there is a theoretical basis for considering SAQs to be superior measurement tools compared to diaries in assessing sexual dysfunction in women. At present, however there is insufficient objective data to fully support this opinion. Conversely, we do not anticipate either theoretical or objective evidence to support the alternative hypothesis (that diaries are superior to SAQs). If this proves to be correct in the future, diary measures may no longer be considered as primary endpoints for FSD clinical trials. Finally, we recommend that the FDA and/or other regulatory agencies reconsider the emphasis given to the number of successful or satisfactory sexual events over time as primary endpoints because they do not definitively demonstrate whether there has or has not been any improvement in the FSD endpoint under study (e.g., sexual desire). Successful and satisfactory encounters represent an amalgam of subjective assessments that are too far removed from the essential FSD component.

Psychometric validation of a sexual quality of life questionnaire for use in men with premature ejaculation or erectile dysfunction.

INTRODUCTION An instrument that can systematically capture the impact of sexual dysfunction on quality of life (QoL) in men is needed. AIMS To psychometrically validate a sexual QoL instrument for men (SQOL-M) with premature ejaculation (PE) or erectile dysfunction (ED). METHODS The main assessment populations were men participating in clinical trials of treatments for PE or ED. Men with PE had a confirmed intravaginal ejaculatory latency time of < or = 2 minutes in > or = 70% of attempts. Men with ED had a score of > 21 on the International Index of Erectile Function (IIEF). Confirmatory psychometric testing was conducted in further groups of men with PE. MAIN OUTCOME MEASURES The internal consistency, convergent and discriminant validity, test-retest reliability, and known-groups validity of the instrument were assessed. RESULTS An 11-item version of the SQOL-M was produced following factor analyses on men with either PE or ED. Psychometric testing showed no overlap between items and good item-total correlations. Factor analysis confirmed a one-factor solution. Excellent internal consistency was demonstrated, with a Cronbachs alpha of > or = 0.82 in all groups. In men reporting no change in their symptoms, the SQOL-M showed excellent test-retest reliability: the intraclass correlation coefficient was 0.77 for men with PE, and 0.79 for men with ED. Convergent validity was also good. In men with PE, the SQOL-M correlated with the satisfaction and distress domains of the Index of Premature Ejaculation. In men with ED, the SQOL-M correlated with the overall satisfaction domain of the IIEF. The measure also demonstrated excellent discriminant validity between men with PE or ED and men with no sexual dysfunction (P < 0.0001). CONCLUSIONS The SQOL-M instrument is a useful tool for evaluating sexual QoL in men with PE and ED.