Ensuring that pharmaceutical and biotechnology products are safe for patient use is an important responsibility health care providers, patients, caregivers, product manufacturers and the U.S. Food and Drug Administration (FDA) and other regulatory agencies share.

It begins in the pre-approval phase with years of laboratory research and clinical studies and continues in the post-approval phase with education, patient follow-up and pharmacovigilance, including the reporting and tracking of adverse events (AEs) and serious adverse events (SAEs). The primary goal of these activities is to ensure that the benefits of each product outweigh the risks in the person receiving the treatment.

Prior to 2007, the FDA had extensive authority to regulate patient safety during the research phase of a product life cycle, but the options for acting on information gathered after the product was marketed were limited.

The passage of the Food and Drug Administration Amendments Act of 2007 (FDAAA) provided the FDA expanded authority to improve patient safety, including a new authority to require certain products to have Risk Evaluation and Mitigation Strategies (REMS) programs.

In the simplest terms, a REMS is an FDA-mandated program, generally developed by a product manufacturer and approved by the FDA that incorporates one or more tools to help ensure the benefits of a specific drug outweigh its risks. The tools included in a REMS vary depending on the risks targeted for minimization. They can include – but are not limited to – a patient medication guide or a health care provider communication plan. A REMS can also include strict limitations on what types of patients are allowed to have access to the product or in what types of health care settings they may be treated.

The intended recipient or responsible party (stakeholder) targeted with each tool can include, among others, patients, caregivers, distributors and/or multiple types of health care providers. Regardless of the tools and/or stakeholders, each REMS is required to have a timetable for assessing whether it is meeting its goals and for evaluating the performance of the individual tools. At each assessment point, the manufacturer may propose – and the FDA can approve – changes to the program to better target the specific patient safety goals for that REMS.

With the introduction of FDAAA and REMS, the FDA has more powerful tools than before to actively address patient safety issues that arise with pharmaceutical and biotechnological products. In fact, a manufacturer with a product requiring a REMS must maintain the approved program or the product will be considered misbranded, incurring monetary and/or civil penalties.

(Editor’s Note: This article provides a brief overview of REMS; future articles will go into greater detail about the specifics of REMS, such as the successful implementation of various types of REMS programs into the pharmacovigilance and commercialization plans of a product.)