Pray, Leslie, Robinson, Sally. "6 Conducting Confirmatory Drug Safety and Efficacy Studies." Challenges for the FDA: The Future of Drug Safety, Workshop Summary. Washington, DC: The National Academies Press, 2007.
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Challenges for the FDA: The Future of Drug Safety - Workshop Summary
public–private surveillance system to improve the detection of safety signals, panelists went a step further during this session by considering the potential of such a system to be used not just for detection, but also as a tool for addressing the broad spectrum of safety science research questions that arise over the course of a drug’s lifetime. A collaborative effort to this end would be more cost-effective than multiple isolated efforts, as presenters in the previous session emphasized with regard to detection. It would give researchers access to a larger volume of information resources, and it would generate information of value to multiple stakeholders.
Dr. Dieck elaborated on earlier discussions regarding the use of public–private partnerships for establishing and conducting active surveillance studies. While industry is interested in supporting such partnerships because of their potential to lower the costs associated with larger safety studies and provide better benefit–risk information, costly studies for specialized populations unlikely to be included in an automated, linked database would still be necessary. Such specialized postmarket safety studies are expensive, costing from $500,000 to $110 million (i.e., the Exubera VOLUME LST study).
Dr. Califf identified two fundamental changes necessary to establish a large public–private partnership to prioritize, plan, and organize funding for confirmatory drug safety and efficacy studies of public health importance. First, stakeholders need to be proactive and take responsibility for establishing such a partnership. These stakeholders include pharmaceutical, biotechnology, and medical device companies; government agencies (the Department of Health and Human Services [DHHS], the National Institutes of Health [NIH], the Agency for Healthcare Research and Quality [AHRQ], the Centers for Medicare and Medicaid Services [CMS], and the Department of Veterans Affairs [VA]); private health plans; academic health centers (which have largely discouraged this kind of activity in the past); and consumer groups. Dr. Califf suggested that bringing these groups together would have not just an additive but a synergistic effect, particularly with regard to workforce standardization and interoperability. Second, it will be necessary to modernize the “incredibly inefficient” clinical research system to eliminate wasteful spending and build efficiency into the system.
Dr. Califf echoed statements made earlier by Drs. Krall and McClellan about what will happen if public–private partnerships and the associated lower costs are not achieved. If the various stakeholders developed their own systems, the resulting bureaucracy would be highly complex; moreover, it would be dangerous to have every health care organization publicizing results and making coverage decisions based on its own limited datasets. Dr. Califf suggested that the IOM conduct a study on the cost of developing such a partnership, hypothesizing that the total cost