Identify, develop, and evaluate data sources and efficient techniques for data mining, data linkage, and large data set analysis that can be used to assess the postmarket toxicity or the safety and effectiveness of FDA-regulated products
Clinical trials for product approval or marketing authorization are conducted in controlled settings. As a result, they cannot uncover or predict all adverse events that might occur during broader clinical use. Some toxicity and safety issues are revealed only after a product is marketed and used in a larger, more diverse population under uncontrolled, variable clinical conditions (real world). FDA is committed not only to support research that facilitates the premarket review of medical products, but also to fund research on marketed products to assist in assessing whether these products are promoting and protecting public health for the diverse subpopulations of women who are using them.
Relevant research OWH has supported in the past includes improving safety signal40 detection, data mining capabilities, and data standardization to enable the use of postmarket data to evaluate the toxicity or the safety and effectiveness of FDA-regulated products. Identifying and using various postmarket data sources is especially critical for gathering informative evidence for special populations of women like pregnant or post-menopausal women using regulated products.
Advances in this area promise numerous benefits. The capability to integrate and interpret diverse data should enable FDA to use its considerable repositories of data from product submissions and adverse event reports more efficiently, in combination with data from electronic medical records, claims data, registries, and basic science research to identify potential efficacy and safety or toxicity issues for women related to the use of regulated products. Research in this area should give FDA additional tools to help identify adverse events in women and predict and minimize adverse events through regulatory actions, such as labeling changes or considerations of product withdrawal from the market. A real benefit would be the identification of data that are useful to support label expansions or changes in practice patterns when groups of patients outside the initial labeled indication are identified for whom benefits outweigh risks.
The potential public health benefits of advancements in evaluating the toxicity or the safety, effectiveness, and security of FDA-regulated products in the postmarket setting are substantial. Advances should provide the scientific data and the tools needed to strengthen existing surveillance networks and help identify and understand possible security, safety, and toxicity issues of importance for the diverse subpopulations of women and for all demographic groups. These strategies will continue to be translated into tools that improve surveillance for all Americans.
5.1 Evaluate and validate U.S. and international postapproval or postmarket data sources for use in the identification of the potential effects of sex differences on the toxicity or the safety, effectiveness, and security of FDA-regulated products used by women:
- Electronic health records
- Surveillance databases
- Clinical outcomes data
- Medicare and other claims data
- Information gathered in pragmatic trials
- Adverse event reports
5.2 Evaluate methodologies for the identification of clinically relevant sex differences, using a variety of postmarket data sources
5.3 Conduct studies using postapproval or postmarket data sources to evaluate the toxicity or the safety, effectiveness, and health effects of FDA-regulated products used by women, including the examination of sex differences
5.4 Develop efficient data mining and analysis techniques that can be used on data sets that can specifically identify women’s health issues on a larger scale. Such techniques have the added value of being beneficial when analyzing premarket as well as postmarket data sources, like clinical data repositories.
40 The phrase safety signal refers to a concern about an excess of adverse events compared to what would be expected to be associated with a product's use. Safety signals can arise from postmarket data, preclinical data, and events associated with other products in the same pharmacologic class. For more, see FDA Guidance Good Pharmacovigilance Practices and Pharmacoepidemiologic Assessment. Accessed March 2015.