Challenges in evaluating cancer as a clinical outcome in postapproval studies of drug safety

Simone P Pinheiro; Donna R Rivera; David J Graham; Andrew N Freedman; Jacqueline M Major; Lynne Penberthy; Mark Levenson; Marie C Bradley; Hui-Lee Wong; Rita Ouellet-Hellstrom

doi:10.1016/j.annepidem.2016.04.012

Challenges in evaluating cancer as a clinical outcome in postapproval studies of drug safety

Ann Epidemiol. 2016 Nov;26(11):735-740. doi: 10.1016/j.annepidem.2016.04.012. Epub 2016 Aug 31.

Affiliations

¹ 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.
² Clinical & Translational Epidemiology Branch, Epidemiology and Genomics Research, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD.
³ Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
⁴ Office of Biostatistics, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
⁵ 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.

Abstract

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.

Keywords: Cancer epidemiology; Drug safety; Pharmacoepidemiology.

Published by Elsevier Inc.

Publication types

Review

MeSH terms

Antineoplastic Agents / pharmacology*
Antineoplastic Agents / therapeutic use
Congresses as Topic
Controlled Clinical Trials as Topic
Drug Approval
Female
Humans
Male
National Cancer Institute (U.S.) / standards*
Neoplasms / drug therapy*
Neoplasms / pathology
Product Surveillance, Postmarketing*
Treatment Outcome*
United States
United States Food and Drug Administration / standards*

Substances

Antineoplastic Agents

Grants and funding

FD999999/Intramural FDA HHS/United States