Conclusions, Recommendations, and Options
Medical products provide great benefit to the public, but they can also cause injury. FDA and the many other participants in healthcare delivery act to maximize the benefits and minimize the risks associated with using medical products, but often the actions of the participants are insufficiently integrated. The Task Force believes that the common goal of maximizing benefits and minimizing risks could be greatly advanced if the participants in the system worked together to gain an understanding of these activities within a systems framework. To achieve such a framework, we need a better understanding of the risks involved and their sources, and we need to clarify our individual roles and ensure that our individual roles are well integrated. Only then can we plan effective risk management strategies.
The Task Force also examined in detail FDA's role in the overall system. We find that the Agency's pre- and postmarketing risk assessment systems are performing well. Nonetheless, we believe that additional emphasis should be placed on the quality assurance of our premarketing review programs. In addition, the Task Force finds that program expansion is needed to ensure that our postmarketing programs are able to meet the challenges of the current regulatory and healthcare environment.
The Task Force is making a number of recommendations as a result of its review. Most recommendations center around ways that FDA, within the confines of the current system, can further improve its risk management activities. The Agency intends to implement these recommendations. Many of these improvements already are underway, and the Task Force recommends that ongoing enhancements be aggressively pursued. Specifics can be found at the end of Parts 2, 3, and 4 of the report, but these recommendations generally include:
- Initiate steps to have each Center establish separate quality assurance/quality control units.
- Ensure and document ongoing professional education and core competency training for all reviewers.
- Complete the good review practice documents and keep them current.
- Rapidly complete AERS and enhance MAUDE adverse event reporting systems for pharmaceutical products and medical devices.
- Integrate existing postmarketing systems so analytical tools, data entry, and editing can be uniformly applied, and all information is readily available to every reviewer.
- Enhance and intensify surveillance of newly marketed products.
- Develop new methodological tools for inference from available datasets.
The Task Force also identified a number of options for consideration, which, if adopted, might contribute to improved risk management. These ideas need full public policy analysis and review to understand their potential value, costs, and acceptability to the various stakeholders in medical product risk management. Some of the options would require significant new resources and legislative changes. Input from stakeholders on these options and their prioritization is needed. For these reasons, the Task Force's key recommendation is that:
- FDA join in or convene a meeting, or series of meetings, with stakeholders to discuss the current system for managing risks. As part of this meeting, FDA should consult stakeholders about the options identified in detail in the report and summarized below.
The Task Force identified a number of options that we believe may improve the FDA's risk management activities as well as improve the overall system of managing the risks from medical products. These options should be evaluated in the context of the stakeholder risk confrontation meeting(s) recommended above. Only by working with all other participants in the overall risk management system for medical products can the Agency arrive at the most effective approach for managing those risks.
Details of the options for public consideration can be found in the relevant chapters of this report. In summary, these options might include:
- Examine and evaluate mechanisms designed to address the inherent limits of premarketing development (e.g., wider use of large, community-based simple trials, restricting exposure during the early postmarketing period).
- Design and implement additional mechanisms to obtain postmarketing information (e.g., sentinel sites, prospective product use registries, enhanced links to external databases).
- Enhance Agency epidemiological and methodological research activities.
- Enhance the Agency's role and responsibilities in risk communication.
- Increase the number of postmarketing risk interventions for products with special risks, such as restricting distribution of products or requiring mandatory educational programs for healthcare professionals and patients.
- Seek legislative changes for other types of risk intervention, such as suspension authority for drugs.
4 A number of terms are used to describe an adverse event, including adverse drug reaction (ADR), adverse experience, and adverse effect. In this report, the term adverse event is used in most cases to avoid confusion.
5 Through the Prescription Drug User Fee Act of 1992 (PDUFA) and the Food and Drug Administration Modernization Act of 1997, Congress has encouraged the FDA to act more rapidly in making decisions on whether new medical products may enter the marketplace.
8 Redux, Pondimin, Seldane, Duract, and Posicor were withdrawn from the market in 1997 and 1998; Seldane and Pondimin were approved prior to PDUFA. For a full discussion, see Friedman, M.A., J. Woodcock, M. Lumpkin, J. Shuren et al., "The Safety of Newly Approved Medicines: Do Recent Market Removals Mean There is a Problem?" JAMA, Vol. 281, No. 18, May 12, 1999.