Statistical challenges in drug approval trials that use patient-reported outcomes∗

Statistical Methods in Medical Research

Volumn 23, Issue 5, 2014, Pages 398-408

  Izem, Rima ^a   Kammerman, Lisa A ^a   Komo, Scott ^a  

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

Author keywords

Patient reported outcome; statistical review

Indexed keywords

ADVISORY COMMITTEE; ALLERGIC CONJUNCTIVITIS; ARTICLE; CLINICAL ASSESSMENT TOOL; CLINICAL PRACTICE; CLINICAL STUDY; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; DRUG APPROVAL; DRUG SCREENING; DYSPNEA; FOOD AND DRUG ADMINISTRATION; HEALTH STATUS; HUMAN; MAINTENANCE THERAPY; OUTCOME ASSESSMENT; PATIENT REPORT OUTCOME; ST. GEORGE RESPIRATORY QUESTIONNAIRE; TRANSITIONAL DYSPNEA INDEX; STANDARDS; STATISTICS; UNITED STATES;

CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; HUMANS; PATIENT OUTCOME ASSESSMENT; STATISTICS AS TOPIC; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84907542701     PISSN: 09622802     EISSN: 14770334     Source Type: Journal    
DOI: 10.1177/0962280213476376     Document Type: Article

References (19)

1. United States 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/UCM071975.pdf (2009, accessed 15 May 2012).
2. United States Food and Drug Administration. Guidance for Industry: Qualification process for drug development tools, http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf (2010, accessed 1 May 2012).
3. Rothman M, Burke L, Erickson P, et al. 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. 2009 ; 12 (8). 1075-1083
4. United States Food and Drug Administration / Center for Drug Evaluation and Research: Meeting minutes of the Pulmonary Allergy Drugs Advisory Committee, Gaithersburg, Maryland, September 6, 2002, http://www.fda.gov/ohrms/dockets/ac/02/minutes/3890M1.htm (2002, accessed in January 2013).
5. Mahler DA, Weinberg DH, Wells CK, et al. The measurement of dyspnea. Contents, interobserver agreement, and physiologic correlates of two new clinical indexes. Chest. 1984 ; 85 (6). 751-758
6. United States Food and Drug Administration / Center for Drug Evaluation and Research: Statistical Review for NDA 21-395, http://www.accessdata.fda.gov/drugsatfda-docs/nda/2004/21-395.pdf-Spiriva-Statr.pdf (2002, accessed 1 May 2012).
7. Quittner AL, Buu A, Messer MA, et al. Development and validation of the Cystic Fibrosis Questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis. Chest. 2005 ; 128: 2347-2354
8. Goss CH, Quittner AL. Patient-reported outcomes in cystic fibrosis. Proc Am Thorac Soc. 2007 ; 4: 378-386
9. Offen W, Chuang-Stein C, Dmitrienko A, et al. Multiple co-primary endpoints: medical and statistical solutions. A report from the Multiple Endpoints Expert Team of the Pharmaceutical Research and Manufacturers of America. Drug Infor J. 2007 ; 41: 31-46
10. McDonald RP Test theory: a unified treatment. London: Erlbaum ; 1999 :
11. Duda RO, Hart PE, Stork DG Pattern classification. 2 nd edn. New York: Wiley-Interscience, 2000. New York: Wiley-Interscience ; 2000 :
12. Rosenfeld M, Emerson J, Williams-Warren J, et al. Defining a pulmonary exacerbation in cystic fibrosis. J Pediatrics. 2001 ; 139 (3). 359-365
13. Bretz F, Maurer W, Brannath W, et al. A graphical approach to sequentially rejective multiple test procedures. Statist Med. 2009 ; 28: 586-604
14. Huque MF, Alosh M, Bhore R. Addressing multiplicity issues of a composite endpoint and its components in clinical trials. J Biopharma Stat. 2011 ; 21: 610-634
15. United States Food and Drug Administration/ Center for Drug Evaluation and Research: Reproductive Health Drugs Advisory Committee Meeting, Gaithersburg, Maryland, June 18, 2010, http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/ReproductiveHealthDrugsAdvisoryCommittee/ucm210869.htm (2010, accessed January 2013).
16. The prevention and treatment of missing data in clinical trials. Washington, DC: National Academies Press ; 2010 :
17. Curran D, Bacchi M, Hsu Schmitz SF, et al. Identifying the types of missingness in quality of life data from clinical trials. Statist Med. 1998 ; 17: 739-756
18. Mainpour CM, Lovato LC. Ensuring the quality of quality of life data: the Southwest Oncology Group experience. Statist Med. 1998 ; 17: 641-651
19. United States Food and Drug Administration. Guidance for Industry: Clinical Studies Section of Labeling for Human Prescription Drug and Biological Products - Content and Format, http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM075059.pdf (2006, accessed on 1 June 2012).