Policy decisions about the human genome.

The application of genomic technology to drug development and therapy will lead to safer and more effective therapies. Elucidation of the human genome poses numerous social, ethical and legal issues, such as the possible misuse of genetic information to discriminate against a person in the workplace or through denial of insurance coverage. Pharmacists need to stay current with advances in genetic medicine and prepare themselves to use this information to optimize drug discovery and drug therapy.

Effects of comorbidity on health-related quality-of-life scores: an analysis of clinical trial data.

Coexisting diseases may have unforeseen yet clinically significant effects on patients' well-being. Both generic and disease-specific measures are frequently used to assess health-related quality of life (QOL). The present study assessed the effects of comorbidity on the results of QOL measures through an analysis of longitudinal data from 3 double-masked, randomized, placebo-controlled clinical trials dealing with heartburn, asthma, and ulcer. Patients were assigned to subgroups by comorbidity status: those with no comorbid diseases and those whose principal disease was heartburn, asthma, or ulcer and whose comorbid condition was chronic obstructive pulmonary disease, asthma, or chronic bronchitis; hypertension; migraine, coronary artery disease, or varicose veins; chronic gastrointestinal conditions; arthritis or back pain; diabetes; or depression. Multivariate analysis of covariance was used to test the study hypotheses. The study results suggest that comorbid conditions significantly and extensively affect patients' scores on generic QOL measures and estimation of treatment effect, whereas their influence on disease-specific QOL scores and estimation of treatment effect is considerably smaller. Further, the most important comorbidities in the 3 trial populations were arthritis or back pain and depression, which respectively accounted for 17% and 5% of the patient population. These findings have significant practical implications for the estimation of true treatment effects, control of comorbidity effects, and design of QOL trials.

Development and validation of the Migraine-Specific Quality of Life Questionnaire.

This paper describes the development and validation of a migraine-specific quality-of-life instrument that is capable of measuring health-related quality-of-life impairments attributed to migraine. Item selection, item reduction, and pretesting and finalization of items during the instrument development phase resulted in a total of 16 questions which were incorporated into the Migraine-Specific Quality of Life Questionnaire (Version 1:0) (1992 Glaxo Wellcome Inc). Three meaningful dimensions were hypothesized: Role Function-Restrictive, Role Function-Preventive, and Emotional Function. In the instrument validation phase, the Migraine-Specific Quality of Life Questionnaire was mailed to 1109 migraine patients, providing a response rate of 45% (n = 458). Initial psychometric evaluation of the questionnaire indicated that it possessed adequate reliability with Cronbach's alpha for the three dimensions ranging between 0.70 to 0.85. The Migraine-Specific Quality of Life Questionnaire possessed adequate content and criterion validity. All but three items satisfied the test of construct validity. In conclusion, the Migraine-Specific Quality of Life Questionnaire has acceptable psychometric properties and can be used to estimate the effect of migraine and its treatment on the patient's health-related quality of life.

Evaluation of a new quality of life questionnaire for patients with irritable bowel syndrome.

BACKGROUND: We describe the development and evaluation of a new disease-specific instrument, the Irritable Bowel Syndrome Quality of Life Questionnaire (IBSQOL), which was designed for use in patients with irritable bowel syndrome. The IBSQOL measures 10 domains found to be relevant to patients with irritable bowel syndrome: emotional health, mental health, health belief, sleep, energy, physical functioning, diet, social role, physical role, and sexual relations. METHODS: During its development and evaluation, the IBSQOL was administered to over 500 patients with irritable bowel syndrome--two groups of patients from tertiary care centres, three focus groups of 8-12 patients each, and 287 patients in a national irritable bowel syndrome support network. As a control, the IBSQOL was also administered to 37 patients who did not have irritable bowel syndrome but had other gastrointestinal disorders. Statistical analyses to test the reliability and validity of the IBSQOL were performed using Cronbach's alpha coefficient. RESULTS: Responses from the focus groups indicated that the IBSQOL was easy to complete and did not require too much time to fill out (approximately equal to 25 min). Statistical analyses of the final 30-item version of the IBSQOL demonstrated that it had both adequate validity and reliability (alpha > or = 0.60). A comparison of mean IBSQOL scores of persons with and without irritable bowel syndrome (but with other gastrointestinal conditions) showed no difference between the two groups with irritable bowel syndrome; however, scores for both irritable bowel syndrome groups were considerably lower than for the non-irritable bowel syndrome group, suggesting better health-related quality of life in patients who do not have irritable bowel syndrome. This further demonstrated the validity of the IBSQOL in targeting questions and domains specific to patients with irritable bowel syndrome. CONCLUSIONS: Evaluation of the IBSQOL included testing the questionnaire in a large number of patients, which resulted in a revised and well-constructed instrument that demonstrated both adequate validity and reliability. The IBSQOL is currently being used in large-scale clinical trials to measure changes in quality of life in patients with irritable bowel syndrome following treatment intervention.

Patient-perceived severity of irritable bowel syndrome in relation to symptoms, health resource utilization and quality of life.

AIM: In this study of patients with irritable bowel syndrome (IBS), we evaluated the relationship between patient-rated severity of IBS and patients' physical and psychological symptoms, health care resource use and quality of life. METHODS: One hundred and twenty-six patients diagnosed with IBS were administered a series of questionnaires, including the Bowel Symptom Checklist, the Symptom Checklist-90-R (a psychological symptom checklist), the IBSQOL (a disease-specific quality of life instrument), the SF-36 (a general health status instrument), and a health resource utilization assessment that measured health care use, time loss from work, impact on productivity, and days worked with symptoms. RESULTS: No relationship was found between IBS severity and gastrointestinal symptoms, except for a feeling of unpassed stool. IBS severity was also not related to psychological symptom severity. Direct traditional indicators of resource use (e.g. physician visits, hospital admissions and emergency room visits) were not significantly associated by severity level; however, indirect measures of resource use (e.g. number of days with pain, productivity and number of bed days) were related to severity. Quality of life was clearly associated with perceived IBS severity. Patients who rated themselves as very severe reported the lowest scores and had the poorest health for all quality of life dimensions measured. CONCLUSIONS: These findings suggest that perceived IBS severity is defined by the limitations the disease imposes, rather that by the symptoms. Patients with reduced productivity and decreased functioning for most of the quality of life indicators were those who rated their IBS as very severe.

Clinical effectiveness and quality of life with ranitidine vs placebo in gastroesophageal reflux disease patients: a clinical experience network (CEN) study.

BACKGROUND: Gastroesophageal reflux disease (GERD), often characterized as heartburn, is a highly common presenting complaint to family physicians. This study is the first large, prospective, nationwide family practice outpatient evaluation of the effectiveness of the histamine (H2)-receptor antagonist ranitidine as medical therapy for this disorder. METHODS: This randomized, double-blind, placebo-controlled, parallel group, 6-week study was designed to evaluate the effect of ranitidine on clinical outcomes and quality of life in patients with GERD. Eligible patients included those who were at least 18 years old and had at least a 3-month history of heartburn or heartburn therapy and a minimum of 4 days with at least one heart-burn episode in the week preceding the baseline visit. Quality-of-life effects were measured using a general health status instrument and a previously validated heartburn-specific questionnaire. RESULTS: Ranitidine treatment conferred clinically and statistically significant reductions in mean heartburn pain scores within the first 24 hours (P < or = .001) and mean number of heartburn episodes within the first 48 hours (P < or = .001). These reductions were maintained throughout the 6-week trial, during both daytime and nighttime. Compared with patients receiving placebo, patients treated with ranitidine also used significantly fewer doses of antacids (P < or = .003). Further, both ranitidine-treated patients' and their physicians' global assessments of decreases in heartburn severity, as well as clinical improvement on ranitidine, proved superior to those of controls (P < or = .001). The rate of adverse events associated with ranitidine and placebo was low and similar. Ranitidine-treated patients had more favorable scores on the general health status dimensions of physical functioning, bodily pain, and vitality (P < .05), and more favorable scores on all dimensions of the heartburn-specific questionnaire (P < .05). CONCLUSIONS: Twice-daily treatment with ranitidine 150 mg is a valuable therapy for GERD in a typical family practice setting. It reduces the frequency and severity of symptoms within the first 24 to 48 hours of treatment and diminishes the use of nonprescription antacids while improving the quality of life as measured by both a general health status instrument and a disease-specific instrument.

Obtaining pharmacoeconomic data in health care organizations.

The article illustrates the process and techniques of obtaining or collecting pharmacoeconomic data in various health care organizations, focusing on hospitals, physicians' offices, and pharmacies as the research settings. The role that pharmacoeconomic data have in the decision-making process as well as the perspective of the decision maker are also discussed. The three primary components needed to conduct a complete pharmacoeconomic analysis (clinical outcomes, humanistic outcomes, and economic outcomes) are described in relation to the health care organization. The strengths, weaknesses, advantages, and disadvantages of such data are discussed. Various databases that are accessible within each organization are also outlined.