Patient-reported outcomes for US oncology labeling: review and discussion of score interpretation and analysis methods

References

• Brook RH, Ware JE Jr, Rogers WH, et al. Does free care improve adults’ health? Results from a randomized controlled trial. N Engl J Med. 1983;309(23):1426–1434. DOI:10.1056/nejm198312083092305.
   PubMed Web of Science ®Google Scholar
• Brook RH, Ware JE Jr., Davies-Avery A, et al. Overview of adult health measures fielded in Rand’s health insurance study. Med Care. 1979;17(7 Suppl):iii-x, 1-131.
   Web of Science ®Google Scholar
• Tarlov AR, Ware JE Jr., Greenfield S, et al. The Medical Outcomes Study. An application of methods for monitoring the results of medical care. JAMA. 1989;262(7):925–930.
   PubMed Web of Science ®Google Scholar
• U.S. Department of Health and Human Services, & Food and Drug Administration. Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Silver Spring, MD: U.S. Food and Drug Administration; 2009.
   Google Scholar
• U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research, & Center for Biologics Evaluation and Research. Guidance for Industry: Clinical Trial Endpoints for the Approval of Non-Small Cell Lung Cancer Drugs and Biologics. Silver Spring, MD: U.S. Food and Drug Administration; 2015.
   Google Scholar
• European Medicines Agency. Reflection paper on the Regulatory Guidance for the Use of Health-related Quality of Life (HRQL) measures in the evaluation of medicinal products. London: European Medicines Agency; 2005.
   Google Scholar
• European Medicines Agency. Guideline on the evaluation of anticancer medicinal products in man. 2012.
   Google Scholar
• Zagadailov E, Fine M, Shields A. Patient-reported outcomes are changing the landscape in oncology care: challenges and opportunities for payers. American Health Drug Benefits. 2013;6:264–274.
   PubMedGoogle Scholar
• Willke RJ, Burke LB, Erickson P. Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels. Control Clin Trials. 2004;25(6):535–552. DOI:10.1016/j.cct.2004.09.003.
   PubMedGoogle Scholar
• Marquis P, Caron M, Emery M, et al. The role of health-related quality of life data in the drug approval processes in the US and Europe. Pharmaceut Med. 2011;25(3):147–160.
   Google Scholar
• Gnanasakthy A, Mordin M, Clark M, et al. A review of patient-reported outcome labels in the United States: 2006 to 2010. Value Health. 2012;15(3):437–442.
   PubMed Web of Science ®Google Scholar
• Basch E, Geoghegan C, Coons S, et al. Patient-reported outcomes in cancer drug development and us regulatory review: perspectives from industry, the food and drug administration, and the patient. JAMA Oncol. 2015. DOI:10.1001/jamaoncol.2015.0530.
   Google Scholar
• U.S. Food and Drug Administration. Drug Development Tools (DDT) Qualification Programs. Silver Spring, MD: U.S. Food and Drug Administration; 2014.
   Google Scholar
• Wyrwich KW, Norquist JM, Lenderking WR, et al. Methods for interpreting change over time in patient-reported outcome measures. Qual Life Res. 2013;22(3):475–483. DOI:10.1007/s11136-012-0175-x.
   PubMed Web of Science ®Google Scholar
• Revicki D, Hays RD, Cella D, et al. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol. 2008;61(2):102–109.
   PubMed Web of Science ®Google Scholar
• Revicki DA, Erickson PA, Sloan JA, et al. Interpreting and reporting results based on patient-reported outcomes. Value Health. 2007;10 (Suppl 2):S116–S124.
   PubMed Web of Science ®Google Scholar
• Uryniak T, Chan ISF, Fedorov VV, et al. Responder analyses — a PhRMA position paper. Stat Biopharm Res. 2011;3(3):476–487. DOI:10.1198/sbr.2011.10070.
   Web of Science ®Google Scholar
• Guyatt G, Walter S, Norman G. Measuring change over time: assessing the usefulness of evaluative instruments. J Chronic Dis. 1987;40(2):171–178.
   PubMedGoogle Scholar
• Jaeschke R, Singer J, Guyatt GH. Measurement of health status: ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10(4):407–415.
   PubMedGoogle Scholar
• Guyatt GH, Osoba D, Wu AW, et al. Methods to explain the clinical significance of health status measures. Mayo Clin Proc. 2002;77(4):371–383.
   PubMed Web of Science ®Google Scholar
• Schunemann HJ, Guyatt GH. Commentary–goodbye M(C)ID! Hello MID, where do you come from? Health Serv Res. 2005;40(2):593–597. DOI:10.1111/j.1475-6773.2005.00374.x.
   PubMed Web of Science ®Google Scholar
• Norman GR, Sloan JA, Wyrwich KW. Interpretation of changes in health-related quality of life: the remarkable universality of half a standard deviation. Med Care. 2003;41(5):582–592.
   PubMed Web of Science ®Google Scholar
• King MT. A point of minimal important difference (MID): a critique of terminology and methods. Expert Rev Pharmacoecon Outcomes Res. 2011;11(2):171–184. DOI:10.1586/erp.11.9.
   PubMed Web of Science ®Google Scholar
• U.S. Department of Health and Human Services. Draft guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. Silver Spring, MD: U.S. Food and Drug Administration; 2006.
   Google Scholar
• Burke LB. Clinical trial outcome assessments: concept definition and context of use. Silver Spring, MD: U.S. Food and Drug Administration; 2011.
   Google Scholar
• Temple R. Context of use for COA’s [Internet]. 2012 Apr 25 [cited 2015 Oct 1]. Available from: http://www.fda.gov/ucm/groups/fdagov-public/@fdagov-afda-orgs/documents/document/ucm303246.pdf.
   Google Scholar
• Slagle AF. Context of use: an FDA Perspective [Internet]. 2014 Mar 18 [cited 2015 Oct 1]. Available from: http://www.diaglobal.org/en/conference-listing/meetings/2014/03/beginning-with-the-end-in-mind-study-endpoints-targeting-patient-centered-outcomes/agenda/spoke-1-defining-the-clinical-trial-context-of-use.
   Google Scholar
• McLeod L, Coon C, Martin S, et al. Interpreting patient-reported outcome results: US FDA guidance and emerging methods. Expert RevPharmacoeconomics Outcomes Res. 2011;11(2):163–169.
   PubMed Web of Science ®Google Scholar
• Werble C. Provectus breakthrough denial is symptom of a different breakthrough at FDA [Internet]. 2014 [cited 2015 Oct 1]. Available from: https://www.pharmamedtechbi.com/publications/rpm-report/10/7/provectus-breakthrough-denial-is-symptom-of-a-different-breakthrough-at-fda.
   Google Scholar
• Cohen J. The Cost of Dichotomization. Appl Psychol Meas. 1983;7(3):249–253. DOI:10.1177/014662168300700301.
   Web of Science ®Google Scholar
• Del Priore G, Zandieh P, Lee MJ. Treatment of continuous data as categoric variables in obstetrics and gynecology. Obstet Gynecol. 1997;89(3):351–354. DOI:10.1016/s0029-7844(96)00504-2.
   PubMed Web of Science ®Google Scholar
• MacCallum RC, Zhang S, Preacher KJ, et al. On the practice of dichotomization of quantitative variables. Psychol Methods. 2002;7(1):19–40.
   PubMed Web of Science ®Google Scholar
• Altman DG, Royston P. The cost of dichotomising continuous variables. BMJ. 2006;332(7549):1080. DOI:10.1136/bmj.332.7549.1080.
   PubMed Web of Science ®Google Scholar
• Bernhard J, Cella DF, Coates AS, et al., et al. Missing quality of life data in cancer clinical trials: serious problems and challenges. Stat Med. 1998;17(5–7):517–532.
   PubMed Web of Science ®Google Scholar
• Fairclough DL. Design and analysis of quality of life studies in clinical trials. 2nd ed. London: CRC Press; 2010.
   Google Scholar
• U.S. Food and Drug Administration. JAKAFI™ (ruxolitinib) - Label and Approval History. Silver Spring, MD: U.S. Food and Drug Administration; 2011.
   Google Scholar
• McCallister E, Usdin S (2011). A PROfessional trial. The Bernstein report on BioBusiness: BioCentury.
   Google Scholar
• Mesa RA, Gotlib J, Gupta V, et al. Effect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trial. J Clin Oncol. 2013;31(10):1285–1292. DOI:10.1200/JCO.2012.44.4489.
   PubMed Web of Science ®Google Scholar
• Paty J. Measurement is Strategic. Applied Clinical Trials. 2010;19:10. Special Section p6.
   Google Scholar
• Bedard G, Zeng L, Zhang L, et al. Minimal important differences in the EORTC QLQ-C30 in patients with advanced cancer. Asia Pac J Clin Oncol. 2014;10(2):109–117. DOI:10.1111/ajco.12070.
   PubMed Web of Science ®Google Scholar
• Cella D, Hahn EA, Dineen K. Meaningful change in cancer-specific quality of life scores: differences between improvement and worsening. Qual Life Res. 2002;11:207–221.
   PubMed Web of Science ®Google Scholar