On January 24, 2019, the United States Food and Drug Administration (FDA) released two draft guidances focused on improving the Agency’s ability to ensure that the risk mitigation programs in place for drugs and biologics as a requirement of their approval are working without limiting patient access or increasing burdens on the health care system.
The draft guidances are expected to provide a framework for companies to develop a Risk Evaluation and Mitigation Strategy (REMS) Assessment Plan at the same time the REMS program is being developed. This will help improve the quality of the information used to assess the effectiveness of a REMS program and provide post-market evidence that the REMS is meeting its risk mitigation goals.
REMS Assessment: Planning and Reporting Guidance for Industry
The first guidance, titled, “REMS Assessment: Planning and Reporting Guidance for Industry,” explains how to develop a REMS Assessment Plan by considering how the program goals, objectives and design may impact the types of metrics and data sources that could be used to assess whether the program is meeting its risk mitigation goals. It further recommends incorporating both process indicators (i.e., the number of patients or health care providers who are enrolled or certified in the REMS) and outcome measures (i.e., the number of prevented fetal exposures to a teratogenic drug) in the REMS Assessment Plan.
Included in the guidance are examples of metrics and data sources to evaluate the performance of the REMS, as well as considerations for assessing the impact of REMS on patient access to the drug or the burden that the REMS imposes on the health care delivery system. According to the FDA’s press release on the subject, “[t]hese considerations are critical implications of instituting REMS and can impact patient care. Properly assessing these issues can help in the design and implementation of more efficient, effective REMS.”
Survey Methodologies to Assess REMS Goals That Relate to Knowledge Guidance for Industry
The second guidance, “Survey Methodologies to Assess REMS Goals That Relate to Knowledge Guidance for Industry,” provides recommendations on how to conduct REMS assessment surveys to evaluate patient or health care provider knowledge of REMS-related information, such as the serious risks and safe use of a medication. The draft guidance also discusses general principles and recommendations related to conducting REMS assessment knowledge surveys, including study design, survey data collection and processing and data analysis.
Conclusion
FDA Commissioner Scott Gottlieb, MD has repeatedly indicated that REMS reform is one of his top priorities and his belief that it is necessary to prevent anticompetitive behavior in the prescription drug market. These guidances are the latest in a series of changes the FDA is making to improve the effectiveness of REMS. The release of these draft guidances follow two other draft guidance documents released in May 2018 that are designed to improve the clarity and efficiency of requirements for developing shared system REMS.