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7.1. The FDA Approval Process 7.1. The FDA Approval Process
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7.2. Type I and II Errors in Drug Approval 7.2. Type I and II Errors in Drug Approval
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7.3. Errors Due to Statistical Imprecision and Wishful Extrapolation 7.3. Errors Due to Statistical Imprecision and Wishful Extrapolation
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7.4. FDA Rejection of Formal Decision Analysis 7.4. FDA Rejection of Formal Decision Analysis
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7.5. Adaptive Partial Drug Approval 7.5. Adaptive Partial Drug Approval
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Adaptive Limited-Term Sales Licenses Adaptive Limited-Term Sales Licenses
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Open Questions Open Questions
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7 Managing Uncertainty in Drug Approval
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Published:September 2019
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Abstract
This chapter considers management of uncertainty in drug approval. In the United States, the approval process of the Food and Drug Administration (FDA) determines whether a drug can legally be sold within the country. A similar process occurs in the European Union, with approval performed by the European Medicines Agency. To obtain approval for a new drug, a pharmaceutical firm must provide to the FDA information on treatment response through the performance of randomized trials that compare the new drug with an existing treatment or a placebo. The FDA makes a binary (yes/no) approval decision after reviewing the findings of these trials. Approval decisions are made with incomplete knowledge of the effectiveness and side effects of new drugs. The chapter describes how the FDA deals with uncertainty and suggests how the approval process might be improved.
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