3.5: To restore appropriate balance between the FDA’s dual goals of speeding access to innovative drugs and ensuring drug safety over the product’s lifecycle, the committee recommends that Congress should introduce specific safety-related performance goals in the Prescription Drug User Fee Act IV in 2007. (See Chapter 3 for suggested goals.)

Science and Expertise

FDA’s Adverse Event Reporting System (AERS) is outdated and inefficient, and although CDER has begun a technological overhaul of the system, more work is needed to improve its usefulness in postmarketing surveillance.


4.1: The committee recommends that in order to improve the generation of new safety signals and hypotheses, CDER (a) conduct a systematic, scientific review of the AERS system, (b) identify and implement changes in key factors that could lead to a more efficient system, and (c) systematically implement statistical-surveillance methods on a regular and routine basis for the automated generation of new safety signals.

In addition, CDER’s ability to test drug safety hypotheses is limited.


4.2: The committee recommends that in order to facilitate the formulation and testing of drug safety hypotheses, CDER (a) increase their intramural and extramural programs that access and study data from large automated healthcare databases and (b) include in these programs studies on drug utilization patterns and background incidence rates for adverse events of interest, and (c) develop and implement active surveillance of specific drugs and diseases as needed in a variety of settings.


The report makes several recommendations (4.3, 4.5, 4.8, and 5.4 below) intended to help CDER develop a more structured way to determine the level of postmarketing scrutiny and data requirements, in other words, to match the evaluation of drugs with the way that they will be used in the population. Short-term preapproval trials do not provide adequate information about the balance of risks and benefits of drugs that are used by many people for many years.

Various public- and private-sector organizations possess increasingly high-quality data resources and scientific capacity, and a concerted effort is



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