Documenting the rationale and psychometric characteristics of patient reported outcomes for labeling and promotional claims: the PRO Evidence Dossier
Rent the article at a discountRent now
* Final gross prices may vary according to local VAT.Get Access
The Food and Drug Administration (FDA) and European Medicines Agency (EMEA) are willing to consider including information on patient reported outcomes (PROs) in product labeling and advertising. Pharmaceutical industry researchers must provide sufficient evidence supporting PRO benefit before an approval may be granted. This report describes the purpose and content of a PRO Evidence Dossier, which consists of important information supporting PRO claims. The dossier should be completed by pharmaceutical industry or other researchers to document the planning of the PRO assessment strategy, psychometric evidence, desired target labeling statements, and the clinical trial evidence of PRO benefits. The systematic reporting and documentation of information on the rationale for including PROs, rationale for the selection of specific PRO instruments, evidence on the psychometric qualities of the PRO measures, and guidelines for interpreting PRO findings will facilitate achieving a PRO labeling or promotional claim. Combining all the relevant information into a single document will facilitate the review and evaluation process for clinical and regulatory reviewers. The PRO Evidence Dossier may also be helpful to industry and academic researchers in identifying further information that will need to be developed to support the clinical development program and the PRO endpoints.
- Wilke, R. J., Burke, L. B., & Erickson, P. (2004). Measuring treatment impact: A review of patient-reported outcomes and other efficacy endpoints in approved labels. Control Clinical Trials, 25, 535–52. CrossRef
- Food and Drug Administration. (February 2006). Guidance for industry –patient-reported outcome measures: Use in medical product development to support labeling claims. Silver Spring, MD: FDA.
- Committee for Medicinal Products for Human Use. (July 2005). Reflection Paper on the regulatory guidance for the use of Health-Related Quality of Life (HRQL) measures in the evaluation of medicinal products. London: EMEA.
- Food and Drug Administration Modernization Act, 1997. Available at http://www.fda.gov/cder/guidance. Accessed September 9, 2004.
- Lohr, K. (2002). Assessing health status and quality-of-life instruments: Attributes and review criteria. Quality of Life Research, 11, 193–05. CrossRef
- Patrick, D. L., & Chiang, Y. P. (2000). Measurement of health outcomes in treatment effectiveness evaluations: Conceptual and methodological challenges. Medical Care, 38, (9 Suppl): II14–5.
- Hays, R., & Revicki, D. A. (2005). Reliability and validity, including responsiveness. In P. Fayers, & R. Hays (Eds.), Assessing quality of life in clinical trials (2nd ed.). New York: Oxford University Press.
- Fayers, P., & Hays, R. (Eds.), (2005). Assessing quality of life in clinical trials (2nd ed.). New York: Oxford University Press.
- Leidy, N. K., Revicki, D. A., & Geneste, B. (1999). Recommendations for evaluating the validity of quality of life claims for labeling and promotion. Value in Health, 2, 113–27. CrossRef
- Revicki, D. A., Osoba, D., Fairclough, D., Barofsky, I., Berzon, R., Leidy, N. K., & Rothman M. (2000). Recommendations on health related quality of life research to support labeling and promotional claims in the United States. Quality of Life Research, 9, 887–00. CrossRef
- Acquadro, C., Berzon, R., Dubois, D., Kline Leidy, N., Marquis, P., Revicki, D., & Rothman, M. (2003). Incorporating the patient’s perspective into drug development and communication: An ad hoc task force report of the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001. Value in Health, 6, 522–31. CrossRef
- Wilson, I. B. & Cleary, P. D. (1995). Linking clinical variables with health-related quality of life A conceptual model of patient outcomes. JAMA, 273, (1):59–5. CrossRef
- Lynn, M. R. (1986). Determination and quantification of content validity. Nursing Research, 35, 382–85. CrossRef
- Guilford, J. P. (1954). Psychometric methods New York: McGraw-Hill.
- Jones, P. W., Quirk, F. H., Baveystock, C. M., & Littlejohns, P. A. (1992). Self-completed measure for chronic airflow limitation –the St George’s Respiratory Questionnaire. American Review of Respiratory Disease, 145, 1321–327.
- Ware, J. E., Snow, K. K., Kosinski, M., & Gandek, B. (1993). SF-36 Health survey: Manual and interpretation guide. Boston, Massachusetts: The Health Institute, New England Medical Center.
- Guyatt, G., Osoba, D., Wu, A., Wyrwich, K., & Norman, G. (2002) Methods to explain the clinical significance of health status measures. Mayo Clinic Proceedings, 77, 371–83. CrossRef
- Sloan, J. A., Cella, D., & Hays, R. D. (2005). Clinical significance of patient-reported questionnaire data: Another step toward consensus. Journal of Clinical Epidemiology, 58, 1217–219. CrossRef
- Revicki, D. A., Cella, D., Hays, R. D., Sloan, J. A., Lenderking, W. R., & Aaronson, N. K. (2006). Responsiveness and minimal important differences for patient reported outcomes. Health Quality of Life Outcomes, 4, 70. CrossRef
- Wyrwich, K. W., Bullinger, M., Aaronson, N., Hays, R. D., Patrick, D. L., Symonds, T., & Sloan, J. A. (2005). Estimating clinically significant differences in quality of life outcomes. Quality of Life Research, 14, 285–95. CrossRef
- Revicki, D. A. (2000). Reporting analyses for clinical trials. In P. Fayers, & R. Hays, (Eds.), Assessing quality of life in clinical trials 2nd ed.). New York: Oxford University Press.
- Efficace, F., Bottomly, A., Osoba, D., Gotay, C., Flechtner, H., D’Haese, S., & Zurlo, A. (2003). Beyond the development of health-related quality-of-life (HRQOL) measures: A checklist for evaluating HRQOL outcomes in cancer clinical trials–does HRQOL evaluation in prostate cancer research inform clinical decision making? Journal of Clinical Oncology, 21, 3502–511. CrossRef
- Szende, A., Leidy, N. K., & Revicki, D. A. (2005). Health-related quality of life and other patient-reported outcomes in the European centralized drug regulatory process: A review of guidance documents and performed authorizations of medicinal products 1995 to 2003. Value in Health, 8, 534–48. CrossRef
- Academy of Managed Care Pharmacy. (2005). The AMCP format for formulary submissions version 2.1. Alexandria, VA: Academy of managed Care Pharmacy.
- National Institute for Clinical Excellence (2004). A guide for manufacturers and sponsors contributing to a technology appraisal London: National Institute for Clinical Excellence.
- Documenting the rationale and psychometric characteristics of patient reported outcomes for labeling and promotional claims: the PRO Evidence Dossier
Quality of Life Research
Volume 16, Issue 4 , pp 717-723
- Cover Date
- Online ISSN
- Springer Netherlands
- Additional Links
- Evidence dossier
- Patient reported outcomes
- Regulatory agencies
- Pharmaceutical industry
- Industry Sectors
- Author Affiliations
- 1. Center for Health Outcomes Research, United Biosource Corporation, 7101 Wisconsin Ave., Suite 600, Bethesda, MD, 20814, USA
- 2. Novartis Pharmaceuticals, East Hanover, NJ, USA
- 3. Duke University, Durham, NC, USA