The patient-reported outcome (PRO) consortium: Filling measurement gaps for PRO end points to support labeling claims

Clinical Pharmacology and Therapeutics

Volumn 90, Issue 5, 2011, Pages 743-748

  Coons, S J ^a   Kothari, S ^b   Monz, B U ^c   Burke, L B ^d  

^a Patient Reported Outcome Consortium   (United States)

^b ASTELLAS PHARMA GLOBAL DEVELOPMENT   (United States)

^c BOEHRINGER INGELHEIM PHARMA GMBH AND CO KG   (Germany)

^d CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; CLINICAL TRIAL (TOPIC); COMPARATIVE EFFECTIVENESS; CONTENT VALIDITY; INFORMATION PROCESSING; LIBRARY SCIENCE; OUTCOME ASSESSMENT; PATIENT ASSESSMENT; PATIENT CARE; PATIENT DECISION MAKING; PATIENT EDUCATION; PATIENT INFORMATION; PATIENT REPORTED OUTCOME; PRESCRIPTION; PRIORITY JOURNAL; PSYCHOMETRY; RISK BENEFIT ANALYSIS; SELF REPORT;

CLINICAL TRIALS AS TOPIC; DATA COLLECTION; DRUG LABELING; ENDPOINT DETERMINATION; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE); REPRODUCIBILITY OF RESULTS; SELF REPORT;

EID: 80054980523     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2011.203     Document Type: Article

References (29)

1. European Medicines Agency. Committee for Medical Products for Human Use. Reflection Paper on the Regulatory Guidance for the Use of Health-Related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products 〈http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/ 2009/09/WC500003637.pdf〉 (2005).
2. US Food and Drug Administration. Guidance for Industry (Draft): Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (2006).
3. Chassany, O., Sagnier, P., Marquis, P., Fullerton, S. & Aaronson, N. Patientreported outcomes: 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. Drug Inf. J. 36, 209-239 (2002).
4. Acquadro, C. et al. 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 Health 5, 521-533(2003).
5. Gondek, K., Sagnier, P.P., Gilchrist, K. & Woolley, J.M. Current status of patientreported outcomes in industry-sponsored oncology clinical trials and product labels. J. Clin. Oncol. 25, 5087-5093 (2007). (Pubitemid 350232288)
6. Burke, L.B. & Walton, M. Outcomes Targeted for labeling: What Works and What Doesn't? Presented at Second Annual Patient-Reported Outcome Consortium Workshop, Silver Spring, MD 〈http://c-path.org/PROSlides/Workshop2/ PROsTargetedforLabeling.pdf〉 (2011).
7. US Food and Drug Administration. Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims 〈http://www.fda.gov/downloads/Drugs/ guidancecomplianceregulatoryinformation/Guidances/UCM193282.pdf〉 (2009).
8. Willke, R.J., Burke, L.B. & Erickson, P. Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels. Control. Clin. Trials 25, 535-552 (2004). (Pubitemid 39592859)
9. Rock, E.P., Kennedy, D.L., Furness, M.H., Pierce, W.F., Pazdur, R. & Burke, L.B. Patient-reported outcomes supporting anticancer product approvals. J. Clin. Oncol. 25, 5094-5099 (2007). (Pubitemid 350232289)
10. Mordin, M. et al. PRO label claims: an analysis based on a review of PROs among NMEs and BLAs 2006-2010. Value Health 14, A29 (2011).
11. Rothman, M.L., Beltran, P., Cappelleri, J.C., Lipscomb, J. & Teschendorf, B.; Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Patientreported outcomes: conceptual issues. Value Health 10 (suppl. 2), S66-S75 (2007).
12. Snyder, C.F., Watson, M.E., Jackson, J.D., Cella, D. & Halyard, M.Y.; Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Patient-reported outcome instrument selection: designing a measurement strategy. Value Health 10 (suppl. 2), S76-S85 (2007). (Pubitemid 350098924)
13. Turner, R.R., Quittner, A.L., Parasuraman, B.M., Kallich, J.D. & Cleeland, C.S.; Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Patientreported outcomes: instrument development and selection issues. Value Health 10 (suppl. 2), S86-S93 (2007). (Pubitemid 350098925)
14. Frost, M.H., Reeve, B.B., Liepa, A.M., Stauffer, J.W. & Hays, R.D.; Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. What is sufficient evidence for the reliability and validity of patient-reported outcome measures? Value Health 10 (suppl. 2), S94-S105 (2007). (Pubitemid 350098926)
15. Sloan, J.A., Dueck, A.C., Erickson, P.A., Guess, H., Revicki, D.A. & Santanello, N.C.; Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Analysis and interpretation of results based on patient-reported outcomes. Value Health 10 (suppl. 2), S106-S115 (2007). (Pubitemid 350098927)
16. Revicki, D.A., Erickson, P.A., Sloan, J.A., Dueck, A., Guess, H. & Santanello, N.C.; Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Interpreting and reporting results based on patient-reported outcomes. Value Health 10 (suppl. 2), S116-S124 (2007). (Pubitemid 350098928)
17. Patrick, D.L. et al. Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health 10 (suppl. 2), S125-S137 (2007). (Pubitemid 350098929)
18. Revicki, D.A., Gnanasakthy, A. & Weinfurt, K. Documenting the rationale and psychometric characteristics of patient reported outcomes for labeling and promotional claims: the PRO Evidence Dossier. Qual. Life Res. 16, 717-723 (2007). (Pubitemid 46474951)
19. Coons, S.J. et al. ISPOR ePRO Task Force. Recommendations on evidence needed to support measurement equivalence between electronic and paper-based patient-reported outcome (PRO) measures: ISPOR ePRO Good Research Practices Task Force report. Value Health 12, 419-429 (2009).
20. Rothman, M., Burke, L., Erickson, P., Leidy, N.K., Patrick, D.L. & Petrie, C.D. Use of existing patient-reported outcome (PRO) instruments and their modification: the ISPOR Good Research Practices for Evaluating and Documenting Content Validity for the Use of Existing Instruments and Their Modification PRO Task Force Report. Value Health 12, 1075-1083 (2009).
21. Patrick D.L., Burke L.B., Gwaltney C.J., Leidy, N.K., Martin, M.L. & Ring, L. Content validity-establishing and reporting the evidence in newlydeveloped patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: part 1-eliciting concepts for a new PRO instrument. Value Health (2011); e-pub ahead of print September 2011
22. US Food and Drug Administration, US Department of Health and Human Services. Critical Path Initiative 〈http://www.fda.gov/scienceresearch/ specialtopics/criticalpathinitiative/default.htm〉.
23. Wu, A.W., Snyder, C., Clancy, C.M. & Steinwachs, D.M. Adding the patient perspective to comparative effectiveness research. Health Aff. 29,1863-1871 (2010).
24. US Food and Drug Administration. US Department of Health and Human Services. Innovation or Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products 〈http://www.fda.gov/downloads/scienceresearch/ specialtopics/criticalpathinitiative/criticalpathopportunitiesreports/ucm113411. pdf〉 (2004).
25. US Food and Drug Administration, US Department of Health and Human Services. Making medical products better, faster, and cheaper: what FDA's "Critical Path" initiatives means to consumers 〈http://www.fda. gov/forconsumers/consumerupdates/ucm061234.htm〉 (2007).
26. US Food and Drug Administration, US Department of Health and Human Services. Critical Path Opportunities List 〈http://www.fda.gov/downloads/ scienceresearch/specialtopics/criticalpathinitiative/ criticalpathopportunitiesreports/UCM077258. pdf〉 (2006).
27. US Food and Drug Administration, US Department of Health and Human Services. Guidance for Industry (Draft): Qualification Process for Drug Development Tools. 〈http://www.fda.gov/downloads/Drugs/ guidancecomplianceregulatoryinformation/Guidances/UCM230597.pdf〉 (2010).
28. Shiffman, S., Hufford, M.R. & Paty, J. Subject experience diaries in clinical research, Part 1: the patient experience movement. Appl. Clin. Trials. 10, 46-56 (2001).
29. Wiklund, I. Assessment of patient-reported outcomes in clinical trials: the example of health-related quality of life. Fundam. Clin. Pharmacol. 18, 351-363 (2004). (Pubitemid 38758704)