Interpreting patient-reported outcome results: US FDA guidance and emerging methods

Expert Review of Pharmacoeconomics and Outcomes Research

Volumn 11, Issue 2, 2011, Pages 163-169

  McLeod, Lori D ^a   Coon, Cheryl D ^a   Martin, Susan A ^a   Fehnel, Sheri E ^a   Hays, Ron D ^b  

^a RTI HEALTH SOLUTIONS   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

individual change; minimal important differences; reliable change index; responder

Indexed keywords

ANALYTIC METHOD; AREA UNDER THE CURVE; CLINICAL RESEARCH; CONTENT VALIDITY; DOCUMENTATION; EFFECT SIZE; ENVIRONMENTAL PROTECTION; FEEDBACK SYSTEM; FOOD AND DRUG ADMINISTRATION; HUMAN; METHODOLOGY; OUTCOME ASSESSMENT; PRACTICE GUIDELINE; RECEIVER OPERATING CHARACTERISTIC; REVIEW; SENSITIVITY AND SPECIFICITY; UNITED STATES;

CLINICAL TRIALS AS TOPIC; GUIDELINES AS TOPIC; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE); UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 79953887793     PISSN: 14737167     EISSN: 17448379     Source Type: Journal    
DOI: 10.1586/erp.11.12     Document Type: Review

References (14)

1. Juniper ER, Guyatt GH, Willan A, Griffth LE. Determining a minimal important change in a disease-specifc quality of life questionnaire. J. Clin. Epidemiol. 47(1), 81-87 (1994). (Pubitemid 24034293)
2. Hays RD, Farivar SS, Liu H. Approaches and recommendations for estimating minimally important differences for health-related quality of life measures. COPD: J. Chron. Obstruct. Pulmon. Dis. 2, 63-67 (2005). (Pubitemid 40776225)
3. Walters SJ, Brazier JE. What is the relationship between the minimally important difference and health state utility values? The case of the SF-6D. Health Qual. Life Outcomes 1, 4 (2003).
4. Wyrwich KW, Tierney WM, Wolinsky FD. Further evidence supporting an SEM-based criterion for identifying meaningful intra-individual changes in health-related quality of life. J. Clin. Epidemiol. 52(9), 861-873 (1999). (Pubitemid 29385600)
5. Farrar JT, Dworkin RH, Max MB. Use of the cumulative proportion of responders ana lysis graph to present pain data over a range of cutoff points: making clinical trial data more understandable. J. Pain Symptom Manage. 31(4), 369-377 (2006).
6. Riffenburgh RH. Statistics in Medicine. Elsevier Academic Press, NY, USA (1999).
7. Hanley JA, McNeil BJ. A method of comparing the areas under receiver operating characteristic curves derived from the same cases. Radiology 148(3), 839-843 (1983). (Pubitemid 13033817)
8. Diaz-Ramos S, Stevens DL Jr, Olsen AR. EMAP statistical methods manual. EPA/620/R-96/002. US Environmental Protection Agency Offce of Research and Development, National Health Effects and Environmental Research Laboratory Western Ecology Division, Corvallis, OR, USA (1996).
9. Gagnon DD. Some logical problems with tests for clinically important changes in HRQoL. Presented at: 8th Annual Conference of the International Society for Quality of Life Research. Amsterdam, The Netherlands, 7-10 November 2001.
10. Farrar JT, Young JP, LaMoreaux L et al. Clinical importance of changes in chronic pain intensity measured on an 11-point numerical pain rating scale. Pain 94, 149-158 (2001). (Pubitemid 33016608)
11. Hays RD, Brodsky M, Johnston MF, Spritzer KL, Hui K. Evaluating the statistical signifcance of health-related quality of life change in individual patients. Eval. Health Prof 28, 160-171 (2005). (Pubitemid 40628905)
12. Martin S, Stanford R, Dale P, Fehnel S. What represents a meaningful improvement in SFD and RFD? The patient's perspective. Presented at: The 2010 European Respiratory Society Meeting. Barcelona, Spain, 18-22 September 2010
13. Brandon P Conclusions about frequently studied modifed Angoff standard-setting topics. Appl. Meas. Educ. 17(1), 59-88 (2004). (Pubitemid 38202857)
14. McLeod LD, Fehnel SE, Brandman J, Symonds T Evaluating minimal clinically important differences (MCID) for the acne-specifc quality of life questionnaire (Acne-QoL). Pharmacoeconomics 21(15), 1069-1079 (2003). (Pubitemid 37330548)