In January 2019, the Food and Drug Administration (FDA) released 2 draft guidance documents regarding assessment of Risk Evaluation and Mitigation Strategies (REMS). Both of these draft guidances are under a comment period through 02 April 2019. These documents are meant to provide industry additional information to more accurately assess the effectiveness of their REMS.
The Food and Drug Administration Amendments Act (FDAAA) created section 505-1 of the Food, Drug, and Cosmetic (FD&C) Act, which authorizes FDA to require Risk Evaluation and Mitigation Strategies for certain prescription drug and biologic products if the FDA determines that it is needed to ensure the benefits of the drug/biologic outweigh the risks (FDA REMS history webpage). REMS are required in cases where there are specific serious risks related to a product and are not meant as a means to mitigate all adverse events. Risk Evaluation and Mitigation Strategies can consist of a Medication Guide, a package insert, and/or a communication plan to inform key audiences about the risks of the product. A REMS should be designed with specific risk mitigation goals consisting of specific objectives and safety-related outcomes. The objectives should include metrics to indicate the program is meeting its goals when a goal cannot be measured directly. As of 05 March 2019, FDA has approved 76 REMS. Assessments evaluating the effectiveness of the REMS must be submitted 18 months, 3 years, and 7 years after the strategy is approved, or at other intervals specified in the strategy. While REMS assessments are required in accordance with 505-1 of the FD&C Act, it does not describe how to design and conduct these assessments.