1
Introduction

Throughout its 100-year history,1 the U.S. Food and Drug Administration (FDA) has sought to protect the public’s health by “assuring that safe and effective drugs are available to the American people” (FDA 2007). Over time, a large segment of the public has developed the belief that FDA- approved drugs carry no risk.2 Yet all drugs have inherent risks3 and one of the functions of the FDA is to evaluate and balance these potential risks against potential benefits. Assessing and managing the risks of modern medical products is a complex and nuanced scientific, political, and sociological challenge that includes not only the assessment of risks and benefits, but also how we communicate them and make healthcare decisions based on them.

To explore these issues, the Forum on Drug Discovery, Development, and Translation conducted a public workshop entitled Understanding the Benefits and Risks of Pharmaceuticals, with the broad goals of gaining a better understanding of the current system for evaluating benefit and risk and of identifying opportunities for improvement. This workshop was held in Washington, D.C., on May 30–31, 2006.

1

The FDA originated in June 1906, when President Teddy Roosevelt signed the Food and Drugs Act, entrusting its implementation to the U.S. Department of Agriculture’s Bureau of Chemistry. The bureau eventually became the FDA.

2

This section is based largely on introductory comments by workshop co-chairs Steven Galson, FDA and Jeff Leiden, Abbott Laboratories.

3

The FDA defines a safe product as “one with reasonable risks given the magnitude of the expected benefit and the available alternatives.”



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OCR for page 7
Understanding the Benefits and Risks of Pharmaceuticals: Workshop Summary 1 Introduction Throughout its 100-year history,1 the U.S. Food and Drug Administration (FDA) has sought to protect the public’s health by “assuring that safe and effective drugs are available to the American people” (FDA 2007). Over time, a large segment of the public has developed the belief that FDA- approved drugs carry no risk.2 Yet all drugs have inherent risks3 and one of the functions of the FDA is to evaluate and balance these potential risks against potential benefits. Assessing and managing the risks of modern medical products is a complex and nuanced scientific, political, and sociological challenge that includes not only the assessment of risks and benefits, but also how we communicate them and make healthcare decisions based on them. To explore these issues, the Forum on Drug Discovery, Development, and Translation conducted a public workshop entitled Understanding the Benefits and Risks of Pharmaceuticals, with the broad goals of gaining a better understanding of the current system for evaluating benefit and risk and of identifying opportunities for improvement. This workshop was held in Washington, D.C., on May 30–31, 2006. 1 The FDA originated in June 1906, when President Teddy Roosevelt signed the Food and Drugs Act, entrusting its implementation to the U.S. Department of Agriculture’s Bureau of Chemistry. The bureau eventually became the FDA. 2 This section is based largely on introductory comments by workshop co-chairs Steven Galson, FDA and Jeff Leiden, Abbott Laboratories. 3 The FDA defines a safe product as “one with reasonable risks given the magnitude of the expected benefit and the available alternatives.”

OCR for page 7
Understanding the Benefits and Risks of Pharmaceuticals: Workshop Summary The benefit–risk profiles of pharmaceuticals are constantly evolving as new data are collected throughout the life cycle of a drug. Discussions during the workshop focused on the following: (1) premarket assessment by the FDA and/or industry, during which clinical trial data are used to assess benefit and risk; (2) communication of that information to prescribing physicians and their patients; (3) healthcare decisions made by prescribing physicians and their patients; and (4) the accumulation of benefit–risk information from postmarketing experience, which feeds back into the other phases (Figure 1-1). The workshop considered the role of multiple stakeholders in benefit–risk assessment throughout the development process, including the FDA, a key player in benefit–risk analyses and decision making; academia, where much of the early discovery takes place; industry, where most of the development process occurs and where marketing plays an ever-increasing role in benefit–risk communication; and physicians and patients, where final decisions about drug use are made. This workshop summary is organized into 6 sections. Section 2 focuses on the regulatory assessment phase of the benefit–risk assessment process. It identifies challenges and potential solutions in premarket assessment of benefits and risks, including approaches to evaluating drug safety. It also examines methods used by other industries for assessing benefits and risks. Section 3 addresses communication issues such as how to quantitatively communicate information about risk to the public, and possible solutions for providing user-friendly information to physicians and patients. Section 4 focuses on healthcare decision making by providers and patients. It discusses how individuals acquire information and make judgments about benefits and risks, how decisions depend on subtle contextual factors, and ways to help patients make informed decisions. Section 5 discusses patient experience with drugs over time and the limitations of postmarketing surveillance. Section 6 concludes with actionable next steps identified by workshop participants.

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Understanding the Benefits and Risks of Pharmaceuticals: Workshop Summary FIGURE 1-1 Constraints and limitations of the four-part life cycle of the pharmaceutical drug benefit–risk assessment process. SOURCE: Adapted from Jeffrey Leiden’s presentation.