Public health and safety is the highest priority at the U.S. Food and Drug Administration. We maintain a robust practice of postmarket surveillance and risk evaluation programs to identify new adverse events that did not appear during the product development process, or to learn more about known adverse events. Evaluations occur on more than two million adverse event reports submitted every year to the FDA Adverse Event Reporting System (FAERS) through the MedWatch Program and to the Vaccine Adverse Event Reporting System (VAERS) by patients, family members, and health care providers, as well as adverse event reports submitted by regulated industry. Staff in our Office of Surveillance and Epidemiology and CBER’s Office of Biostatistics and Epidemiology use this information to identify safety concerns and recommend actions to improve product safety and protect the public.
Recently, we’ve been working to enhance the efficiency of our postmarket drug safety surveillance practices. In 2016, the 21st Century Cures Act (Cures Act) amended the Federal Food, Drug, and Cosmetic Act to eliminate the requirement for FDA to prepare a summary analysis of adverse drug reaction reports received for a drug by 18 months following approval of the drug or after 10,000 individuals have used the drug, whichever occurs later. A 2016 agency study to assess the impact of these analyses on our regulatory actions found that these assessments were largely redundant to the FDA’s current surveillance practices and were not an efficient use of agency resources. For example, many drugs and biologics for rare diseases never met the 10,000-individual use threshold.
The Cures Act included a new requirement that the FDA make our best practices for drug safety surveillance publicly available on the web. Following the passage of the Cures Act we posted a statement announcing the risk-based principles we use for postmarket safety surveillance. Today, we’re announcing the availability of our draft document, “Best Practices in Drug and Biological Product Postmarket Safety Surveillance for FDA Staff,” which expands upon those principles. This draft best practice document outlines our approach for timely postmarket analyses of drugs and biologics, and includes a high-level overview of tools, methods, and signal detection and evaluation activities, using varied data sources, for drug safety surveillance to provide a broader context and a general overview of our overarching effort and commitment in this area.
For prompt signal detection and evaluation of safety concerns using a risk-based approach, our best practices document addresses how we take into consideration the product’s characteristics and use. For example, products that generally are subject to more extensive monitoring include: NDAs that are new molecular entities, original biological license applications, biosimilar biological products, first-in-class approvals, newly approved formulation(s), newly approved indication(s), extension into new patient populations, products with complex pharmacokinetic or pharmacodynamic characteristics, and products with complex compositions or manufacturing processes. FDA staff also monitor the safety of compounded products, even though they are not subject to FDA premarket review and approval, as well as homeopathic products.
Our best practices document incorporates the guiding principle that postmarket safety surveillance is a dynamic and constantly evolving field. By using a risk-based approach, the FDA takes into account the nature of the drug, its potential adverse events, the intended population, and the potential for serious outcomes, as well as the impact on individuals and the overall potential impact on the health of the public. When information is uncovered that may change the benefit-risk profile of a product, we will investigate the issue and consider appropriate action, including labeling changes, issuing drug safety communications, requiring postmarketing studies or requiring a Risk Evaluation and Mitigation Strategy. Even when we do not have enough evidence to establish a causal association between a drug and adverse event, we continue monitoring the safety of the product.
In addition to FDA’s surveillance, postmarketing studies are important to patient safety and public health . We have been reporting the progress of postmarketing studies and holding companies accountable for completing these important studies on time to ensure that our understanding of a drug’s safety and efficacy keeps pace with its uses. As the agency continues to commit to its safety assessments, product sponsors must continue to do their part, and we will take appropriate action when requirements are missed. The FDA will continue to provide guidance and oversight to ensure product sponsor studies are carried out in a complete and timely manner.
We are constantly seeking new methods for improving our surveillance practices, and we invite the public to comment on the draft document released today. Through new data systems, tools, applications and partnerships, we will continue our efforts and commitment to exploring new and updated approaches to postmarket safety surveillance of all drugs and biologic products as part of our uncompromising efforts to protect the American public and ensure the products they take are safe and effective.