Understanding the Patient Perception of Statin Experience: A Qualitative Study.

INTRODUCTION: Statin intolerance (SI) occurs in patients with dyslipidemia treated with statins. Statin-associated symptoms have been reported, but the overall patient experience is poorly understood. No instruments are available to collect this patient experience. Our aim is to develop a patient survey to define SI from the patient's perspective, inform clinical practice, and identify potential patient characteristics and barriers associated with discontinuing treatment when statin-related difficulties are encountered. METHODS: We conducted qualitative concept elicitation interviews with 65 patients across 12 European study sites. A semi-structured qualitative interview guide was developed based on literature review and clinician interviews. Concept elicitation interviews with patients were used to describe the patient experience and develop the conceptual framework for the survey. RESULTS: Symptoms experienced by patients included muscle and non-muscle-related pain and discomfort; other muscle-related symptoms; gastrointestinal, cardiovascular, cold-like, fatigue-related, and sensory and systems symptoms; mood changes; and cognitive and memory problems. Impacts included limitations on general physical functioning; physical activities; social functioning; emotional impacts; sleep disturbances; decreased productivity; and increased healthcare use. Conceptual framework elements to support survey goals include demographic and clinical characteristics, health information and beliefs, statin side-effect history, symptom severity, and impact severity. CONCLUSIONS: Symptoms and impacts described by patients showed a wider range of symptoms and impacts than usually discussed clinically. The patient survey is designed to capture information from patients who experience difficulties with statin therapy and may be useful in identifying patients who are at higher risk for giving up or discontinuing their treatment. FUNDING: Amgen Inc.

The psychometric validation of a US English satisfaction measure for patients with benign prostatic hyperplasia and lower urinary tract symptoms.

BACKGROUND: The purpose of the current study was to validate the US English Patient Perception of Study Medication (PPSM) questionnaire, which measures patient satisfaction with Benign Prostatic Hyperplasia (BPH) treatment and was administered to men with BPH lower urinary tract symptoms (LUTS) enrolled in a multi-national clinical trial. METHODS: Patients with moderate to severe BPH symptoms completed three disease-specific measures: The International Prostate Symptom Score (IPSS), the BPH Impact Index (BII) and the PPSM, at baseline (after completion of the placebo run-in period) and at every 13-week clinic visit thereafter for the duration of the study treatment period. The PPSM was analysed to assess its variability, reliability and validity. RESULTS: There were 879 patients included in the analyses, with a mean age of 66.7 years. The PPSM was found to comprise two factors - PPSM-Global and PPSM-Pain, with a Total Score ranging from 7 to 49. It demonstrated good internal consistency (Cronbach's alpha ranged from .95 to .97) and also demonstrated convergent validity through significant correlations with the IPSS (.48 to .58), IPSS Quality of Life (QoL) item (.41 to .63) and BII (.31 to .45) and known-groups validity against the IPSS, IPSS QoL item and BII. CONCLUSION: Results support the use of the PPSM as a measure of satisfaction in BPH patient groups.

Principles of Good Practice for the Translation and Cultural Adaptation Process for Patient-Reported Outcomes (PRO) Measures: report of the ISPOR Task Force for Translation and Cultural Adaptation.

In 1999, ISPOR formed the Quality of Life Special Interest group (QoL-SIG)--Translation and Cultural Adaptation group (TCA group) to stimulate discussion on and create guidelines and standards for the translation and cultural adaptation of patient-reported outcome (PRO) measures. After identifying a general lack of consistency in current methods and published guidelines, the TCA group saw a need to develop a holistic perspective that synthesized the full spectrum of published methods. This process resulted in the development of Translation and Cultural Adaptation of Patient Reported Outcomes Measures--Principles of Good Practice (PGP), a report on current methods, and an appraisal of their strengths and weaknesses. The TCA Group undertook a review of evidence from current practice, a review of the literature and existing guidelines, and consideration of the issues facing the pharmaceutical industry, regulators, and the broader outcomes research community. Each approach to translation and cultural adaptation was considered systematically in terms of rationale, components, key actors, and the potential benefits and risks associated with each approach and step. The results of this review were subjected to discussion and challenge within the TCA group, as well as consultation with the outcomes research community at large. Through this review, a consensus emerged on a broad approach, along with a detailed critique of the strengths and weaknesses of the differing methodologies. The results of this review are set out as "Translation and Cultural Adaptation of Patient Reported Outcomes Measures--Principles of Good Practice" and are reported in this document.