Patient-reported outcomes in cancer drug development and US regulatory review: Perspectives from industry, the Food and Drug Administration, and the patient

JAMA Oncology

Volumn 1, Issue 3, 2015, Pages 375-379

  Basch, Ethan ^a   Geoghegan, Cindy ^b   Coons, Stephen Joel ^c   Gnanasakthy, Ari ^d   Slagle, Ashley F ^e   Papadopoulos, Elektra J ^e   Kluetz, Paul G ^e  

^a UNIVERSITY OF NORTH CAROLINA   (United States)

^b Patient and Partners LLC   (United States)

^c Patient Reported Outcome Consortium   (United States)

^d NOVARTIS PHARMA AG   (Switzerland)

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

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT;

ATTITUDE TO HEALTH; COOPERATION; DRUG APPROVAL; DRUG DEVELOPMENT; DRUG INDUSTRY; FOOD AND DRUG ADMINISTRATION; HUMAN; INTERDISCIPLINARY COMMUNICATION; LEGISLATION AND JURISPRUDENCE; NEOPLASMS; PATHOLOGY; PATIENT; PATIENT PARTICIPATION; PATIENT SATISFACTION; PERCEPTION; PSYCHOLOGY; TREATMENT OUTCOME; UNITED STATES;

ANTINEOPLASTIC AGENTS; COOPERATIVE BEHAVIOR; DRUG APPROVAL; DRUG DISCOVERY; DRUG INDUSTRY; HEALTH KNOWLEDGE, ATTITUDES, PRACTICE; HUMANS; INTERDISCIPLINARY COMMUNICATION; NEOPLASMS; PATIENT PARTICIPATION; PATIENT SATISFACTION; PATIENTS; PERCEPTION; TREATMENT OUTCOME; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84965093502     PISSN: 23742437     EISSN: 23742445     Source Type: Journal    
DOI: 10.1001/jamaoncol.2015.0530     Document Type: Article

References (15)

1. Coons SJ, Kothari S, Monz BU, Burke LB. The Patient-Reported Outcome (PRO) Consortium: filling measurement gaps for PRO endpoints to support labeling claims. Clin Pharmacol Ther. 2011;90:743-748.
2. Basch E. Toward patient-centered drug development in oncology. N Engl J Med. 2013;369(5):397-400.
3. Gnanasakthy A, Mordin M, Clark M, DeMuro C, Fehnel S, Copley-Merriman C. A review of patient-reported outcome labels in the United States: 2006 to 2010. Value Health. 2012;15(3):437-442.
4. US Food and Drug Administration. Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. December 2009. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed March 15, 2015.
5. Fehnel S, DeMuro C, McLeod L, Coon C, Gnanasakthy A. US FDA patient-reported outcome guidance: great expectations and unintended consequences. Expert Rev Pharmacoecon Outcomes Res. 2013;13(4):441-446.
6. Revicki DA; Regulatory Issues and Patient-Reported Outcomes Task Force for the International Society for Quality of Life Research. FDA draft guidance and health-outcomes research. Lancet. 2007;369(9561):540-542.
7. Clark MJ, Harris N, Griebsch I, Kaschinski D, Copley-Merriman C. Patient-reported outcome labeling claims and measurement approach for metastatic castration-resistant prostate cancer treatments in the United States and European Union. Health Qual Life Outcomes. 2014;12:104.
8. Gnanasakthy A, DeMuro C, Boulton C. Integration of patient-reported outcomes in multiregional confirmatory clinical trials. Contemp Clin Trials. 2013;35(1):62-69.
9. Aaronson NK, Ahmedzai S, Bergman B, et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst. 1993;85(5):365-376.
10. Doward LC, Gnanasakthy A, Baker MG. Patient reported outcomes: looking beyond the label claim. Health Qual Life Outcomes. 2010;8:89.
11. Bergmann L, Enzmann H, Broich K, et al. Actual developments in European regulatory and health technology assessment of new cancer drugs: what does this mean for oncology in Europe? Ann Oncol. 2014;25(2):303-306.
12. Food and Drug Administration Safety and Innovation Act, S 3187, 112th Cong (2012). http://www.gpo.gov/fdsys/pkg/PLAW-112publ144/pdf/PLAW-112publ144.pdf. Accessed June 17, 2014.
13. Adequate and Well-controlled Studies, 21 CFR §314.126 (2014).
14. Ellis LM, Bernstein DS, Voest EE, et al. American Society of Clinical Oncology perspective: raising the bar for clinical trials by defining clinically meaningful outcomes.JClin Oncol. 2014;32(12):1277-1280.
15. US Food and Drug Administration. Guidance for industry and FDA staff: qualification process for drug development tools. January 2014. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf. Accessed March 15, 2015.