An estimated 48 percent of the population takes at least one prescription drug in a given month. Drugs provide great benefits to society by saving or improving lives. Many drugs are also associated with side effects or adverse events, some serious and some discovered only after the drug is on the market. The discovery of new adverse events in the postmarketing setting is part of the normal natural history of approved drugs, and timely identification and warning about drug risks are central to the mission of the Food and Drug Administration (FDA). Not all risks associated with a drug are known at the time of approval, because safety data are collected from studies that involve a relatively small number of human subjects during a relatively short period.


Written in response to a request by the FDA, Ethical and Scientific Issues in Studying the Safety of Approved Drugs discusses ethical and informed consent issues in conducting studies in the postmarketing setting. It evaluates the strengths and weaknesses of various approaches to generate evidence about safety questions, and makes recommendations for appropriate followup studies and randomized clinical trials. The book provides guidance to the FDA on how it should factor in different kinds of evidence in its regulatory decisions.


Ethical and Scientific Issues in Studying the Safety of Approved Drugs will be of interest to the pharmaceutical industry, patient advocates, researchers, and consumer groups.

Table of Contents


Front Matter
Abstract
Summary
1 Introduction
2 Incorporating Benefit and Risk Assessment and BenefitRisk Management into Food and Drug Administration Decision-Making
3 Evidence and Decision-Making
4 Selection and Oversight of Required Postmarketing Studies
5 Synthesis
Appendix A: Other Elements of the Food and Drug Administration Amendments Act
Appendix B: Committee's Letter Report
Appendix C: Open Session Agendas
Appendix D: Decision Conferencing and Multicriteria Decision Analysis
Appendix E: Benefit and Risk Assessment and Management Plan Document Template
Appendix F: Committee Biosketches