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Patient-Reported Outcomes: The Example of Health-Related Quality of Life—a European Guidance Document for the Improved Integration of Health-Related Quality of Life Assessment in the Drug Regulatory Process

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Abstract

The added value for assessing the health-related quality of life (HRQOL) in chronic conditions is now well documented for evaluation of treatment effectiveness in clinical trials and as a criterion for licensing new medications and in policy decisions. However, European standards still need to be developed for the measurement and reporting of HRQOL in clinical trials. This is one of the objectives of the European Regulatory Issues on Quality of Life Assessment (ERIQA) Working Group. This document reviews the major issues arising from the selection of an HRQOL instrument; the integration of HRQOL assessment into the research protocol (methodological design, practicalities of HRQOL administration and collection, prevention and handling of missing data); the statistical analysis plan; and the presentation and interpretation of the results. Finally, to gain wider acceptance, whether HRQOL is considered as a primary or secondary endpoint, the scientific principles of clinical trial design should apply to HRQOL.

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Correspondence to Dr. Olivier Chassany MD, PhD.

Additional information

Members of the European Regulatory Issues on Quality of Life Assessment (ERIQA) Group: Dr. Neil Aaronson, The Netherlands Cancer Institute, The Netherlands; Dr. Catherine Acquadro, Coordinator of the ERIQA Group, Mapi Research Institute, France; Dr. Giovanni Apolone, Mario Negri Institute, Italy; Dr. Harry Burns, Dalian House, Glasgow, UK; Dr. Olivier Chassany, Hopital Saint-Louis, France; Dr. Gianfranco De Carli, GlaxoWellcome, Italy; Dr. Dominique Dubois, Janssen Pharmaceutica N.V., Belgium; Steve Fullerton, Cedars-Sinai Health System/Zynx Health Incorporated, US; Dr. Bernard Geneste, Aventis, France; Dr. Asha Hareendran, Pfizer Central Research, UK; Bernard Jambon, Mapi Group, France; Dr. Patrick Marquis, Mapi Values, US; Dr. Pauline McNulty, ICOM Health Economics, Johnson & Johnson, US; Dr. Caroline Miltenburger, SCHERING AG, Germany; Dr. Annoesjka Novak, NV Organon, The Netherlands; Dr. Margaret L. Rothman, Janssen Research Foundation, US; Dr. Pierre Philippe Sagnier, Bayer plc, Medical Affairs, UK; Dr. François Schubert, GlaxoWellcome Research & Development, UK; Dr. Soren E. Skovlund, Novo Nordisk, Denmark; Dr. Marianne Sullivan, Goteborg University, Sweden; Dr. Marc Tomas, Outcomes Research BMS, Belgium; Dr. Suzanne Wait, Bristol Myers Squibb, UK; Dr. Ingela Wiklund, AstraZeneca R&D, Sweden; and Dr. G. Rhys Williams, Knoll Pharmaceutical Company, US.

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Chassany, O., Sagnier, P., Marquis, P. et al. Patient-Reported Outcomes: The Example of Health-Related Quality of Life—a European Guidance Document for the Improved Integration of Health-Related Quality of Life Assessment in the Drug Regulatory Process. Ther Innov Regul Sci 36, 209–238 (2002) doi:10.1177/009286150203600127

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Key Words

  • Health-related quality of life (HRQOL)
  • Randomized clinical trials
  • Patient-reported outcomes
  • Missing data
  • Guidelines