Regulatory Issues for Health-Related Quality of Life— PhRMA Health Outcomes Committee Workshop, 1999

Abstract

HRQL provides a patient perspective on the impact of disease and therapy on patients' daily life and functioning. Including HRQL information in promotion could be beneficial to decision making on the use of therapies. HRQL is a measure of effectiveness, not safety, and should be treated as any other clinical end point.

Development and validation of the Patient Assessment of Constipation Quality of Life questionnaire

“…Its content was derived directly from individuals affected by the condition and from clinicians involved in the treatment of gastrointestinal disorders. Furthermore, the results of validation studies conducted in North America, Europe, and Australia provide evidence that the PAC-QOL meets or exceeds standards for reliability, validity, and responsiveness that have been suggested for regulatory review of QOL findings reported for new therapies [16,17].…”

Poor Reporting of Quality of Life Outcomes in Dermatology Randomized Controlled Clinical Trials

“…Several recommendations for optimal use of QOL questionnaires in therapeutic trials have recently been published[3,4,5,6]. Those recommendations were intended to raise the status of QOL evaluation to that of a pertinent endpoint by improving the quality of use and reports on RCTs using a QOL questionnaire as an evaluation criterion just as Consolidated Standards for Reporting of Trials (CONSORT) use has helped to improve the quality of RCT reports [74].…”

“…Although QOL is a patient-reported criterion, the scientific principles of clinical trials should be applied whether it is a primary or secondary endpoint to make it a judgment endpoint. To achieve this objective, several groups have published recommendations [4, 5] concerning the validation of the questionnaires to be used and their rigorous application during the trials. Furthermore, the role of the QOL in the evaluation of therapies was clearly established in the recent general recommendations issued by the European Agency for the Evaluation of Medicinal Products and moreover by the guidance on patient-reported outcomes released by the Food and Drug Administration [75, 76].…”

“…Several published recommendations on the use of QOL questionnaires in RCTs are available [3,4,5,6]. The objective of this investigation was to gather the reports on RCTs measuring QOL in dermatology, and analyze them based on the main elements of the checklist of The European Regulatory Issues on Quality of Life Assessment (ERIQA) Working Group.…”

Patient reported outcomes in gastroesophageal reflux disease: An overview of available measures

“…The FDA is expected to release a guidance document in the near future. Meanwhile, several publications have discussed the requirements for inclusion of PROs in clinical trials and their use in promotional and labelling claims [23,24,[26][27][28].…”