First weigh the considerations, then take the risks.
-- Helmuth von Moltke
For you PDUFA junkies, some important reading prior to the upcoming E&C and HELP hearings.
Some snippets from a pre-publication copy …
A Risk-Characterization Framework for Decision-Making at the Food and Drug AdministrationCommittee on Ranking FDA Product Categories Based on Health Consequences, Phase II, Board on Environmental Studies and Toxicology Division on Earth and Life Studies, National Research Council, Institute of Medicine
FDA and the Department of Health and Human Services (DHHS) asked the National Research Council (NRC) to develop a conceptual model that could evaluate products or product categories that FDA regulates and provide information on the potential health consequences associated with them. As a result of that request, NRC formed the Committee on Ranking FDA Product Categories Based on Health Consequences.
Difficult decisions are common for the Food and Drug Administration (FDA). Whether it is allocating scarce resources, deciding how to mitigate newly found risks, or deciding what investments in human capital, facilities, data, or analytic methods would be most useful, decision-makers in the FDA often need to integrate data of varied quality, recognize uncertainties, and make trade-offs to arrive at a decision. Public-health and public-safety concerns must be balanced with the economic realities of budgets and the political constraints of imposing new regulations.How a program is presented by the media and understood by the public is an important determinant of the acceptance and success of the program. Science and public preferences and perceptions must be considered if one is to understand the potential outcomes of different decision options. To inform the decision-making process, data of differing degrees of quality and robustness must be used and sometimes fed through an array of models of varied sophistication. Expert opinion must be used to interpret the relevance of available data and to solve problems on which available data are weak or nonexistent. Immovable deadlines can thwart uncompromising reliance on the most thorough analysis based on detailed quantitative data for a given decision.
To succeed in such an environment, FDA needs a framework within which alternatives can be defined and evaluated systematically. Although it is beyond the scope of the present study to provide a comprehensive decision-making procedure for FDA, the committee proposes a general framework for thinking about and characterizing the human-health dimensions of FDA decisions. Health consequences are only a subset of the large array of factors that must be considered for any given problem. However, they constitute a reasonable place to start the process of developing a decision framework inasmuch as such factors loom large in most FDA decisions and substantial work has already been done on methods for estimating the human-health consequences associated with various risks, hazards, and decisions.
The framework offered here provides a common language for describing potential public-health consequences of decisions, is designed to have wide applicability among all FDA centers, and draws extensively on the well-vetted risk literature to define the relevant health dimensions for FDA decision-making.
This chapter first provides a brief description of the proposed framework and the risk and decision contexts that influenced the committee’s approach. Next, the basis and definition of the risk attributes that characterize the framework are provided, and then some approaches for estimating the outcomes of decisions using the risk attributes are described. The chapter concludes with a discussion of how the output of the framework can be used to support decision-making.
THE FRAMEWORK
The risk-characterization framework is designed to be as general as possible while providing consistent risk information in a way that can be used to support the wide variety of decisions that FDA faces. It is intended to supplement and augment other risk-based and risk-informed approaches that are in use and under development by FDA, not to be a replacement or a one-size-fits-all prescription for conducting all risk-informed decision-making.Indeed, the committee recognizes that the public-health consequence factors highlighted in this framework will seldom be the only important considerations in the decision-making process, but they are almost always some of the key considerations. The U.S. Nuclear Regulatory Commission has also recognized that risks are not the only factors that must be taken into account in regulatory decision-making. It recently embraced the concept of risk-informed decision-making, which it defines as “the use of risk insights, along with other important information, to help in making decisions” (USNRC 2008, page 1-1). The committee’s framework focuses on risk information but also recognizes that other information will be relevant for most FDA decisions.
The process is straightforward and involves three steps:Step 1. Identify and define the decision context: What decision options are being considered? What are the appropriate end points to evaluate and compare?
Step 2. Estimate or characterize the public-health consequences of each option by using the risk attributes that are described below. The values of the risk attributes should be summarized in a table to facilitate comparison of the options.
Step 3. Use the completed characterization as a way to compare decision options and to communicate their public-health consequences within the agency, to decision-makers, and to the public; use the comparison with other decision-relevant information to make informed decisions. Although the steps can be easily articulated, they involve thought and effort to complete.
The framework is not a cookbook and will require FDA to exercise judgment in how it is used.
The full report can be found here.
The policy of being too cautious is the greatest risk of all.
Jawaharlal Nehru
