Challenges in evaluating cancer as a clinical outcome in postapproval studies of drug safety.
Pharmaceuticals approved in the United States are largely not known human carcinogens. However, cancer signals associated with pharmaceuticals may be hypothesized or arise after product approval. There are many study designs that can be used to evaluate cancer as an outcome in the postapproval setting. Because prospective systematic collection of cancer outcomes from a large number of individuals may be lengthy, expensive, and challenging, leveraging data from large existing databases are an integral approach. Such studies have the capability to evaluate the clinical experience of a large number of individuals, yet there are unique methodological challenges involved in their use to evaluate cancer outcomes. To discuss methodological challenges and potential solutions, the Food and Drug Administration and the National Cancer Institute convened a two-day public meeting in 2014. This commentary summarizes the most salient issues discussed at the meeting.
Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD. Electronic address: simone.pinheiro@fda.hhs.gov.
Simone P
SP
Pinheiro
Clinical & Translational Epidemiology Branch, Epidemiology and Genomics Research, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD.
Donna R
DR
Rivera
Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
David J
DJ
Graham
Clinical & Translational Epidemiology Branch, Epidemiology and Genomics Research, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD.
Andrew N
AN
Freedman
Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Jacqueline M
JM
Major
Clinical & Translational Epidemiology Branch, Epidemiology and Genomics Research, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD.
Lynne
L
Penberthy
Office of Biostatistics, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Mark
M
Levenson
Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD; Clinical & Translational Epidemiology Branch, Epidemiology and Genomics Research, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD.
Marie C
MC
Bradley
Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Hui-Lee
HL
Wong
Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Rita
R
Ouellet-Hellstrom
D000970:Antineoplastic Agents / Q000494:pharmacology* / Q000627:therapeutic use; D003226:Congresses as Topic; D018849:Controlled Clinical Trials as Topic; D017277:Drug Approval; D005260:Female; D006801:Humans; D008297:Male; D054547:National Cancer Institute (U.S.) / Q000592:standards*; D009369:Neoplasms / Q000188:drug therapy* / Q000473:pathology; D011358:Product Surveillance, Postmarketing; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration / Q000592:standards*
D000970:Antineoplastic Agents
Cancer epidemiology; Drug safety; Pharmacoepidemiology
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