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FDA Statement

FDA statement from Commissioner Scott Gottlieb, M.D. announcing efforts to improve the quality of the information used to assess the effectiveness of REMS programs in supporting the safe use of medications

For Immediate Release:
Statement From:

In making decisions about whether the benefits of a drug outweighs its risks, there are many times when a drug provides important benefits, but also has serious risks. In some cases, if there is a greater assurance that those risks can be properly managed, then the benefits can support approval. This is the field of risk management, and it’s a critical function here at the agency.

Today, we’re announcing the availability of two draft guidances that will help improve our ability to ensure that the risk mitigation programs put in place for certain drugs and biologics as a requirement of their approval are working. These risk mitigation programs can help make sure that benefits of these products continue to outweigh the risks.

Risk management involves four key areas: assessing a drug’s benefit-risk balance; developing and implementing tools to minimize risks while preserving benefits; evaluating the effectiveness of such tools and re-assessing the drug’s benefit-risk balance; and making adjustments, as appropriate, to risk minimization tools to further improve the benefit-risk balance. Our primary risk management tool is communicating through FDA-approved product labeling, often referred to as the “package insert” or the “prescribing information,” which includes a summary of the essential information needed by health care providers for the safe and effective use of the drug. Labeling is sufficient for most drugs to ensure that the benefits outweigh the risks. But in a limited number of cases – 75 currently – the FDA may determine that a Risk Evaluation and Mitigation Strategy (REMS) will also be needed to help ensure that the benefits of the drug outweigh its risks. A REMS is a drug safety program that the FDA can require for certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks. REMS are designed to reinforce medication use behaviors and actions that support the safe use of that medication.

Most REMS include a requirement to foster communication between a doctor and patient, for example, about the specific safety risk or risks that the REMS is intended to mitigate. Some REMS also include additional elements that may ensure providers have, or provide to patients, all the appropriate information to appropriately prescribe or dispense the medication. This may include limiting access until health care providers complete an educational program, assess certain confirmatory laboratory tests, or document that they counseled patient about the potential risks.

But just as important as setting the strategy for managing the risk is established, and setting clear goals for the program, is ensuring it is having the desired impact. From the onset of the REMS, the FDA works closely with companies that sell these products to determine the best way to assess its effectiveness in meeting its goals and maintaining the benefit-risk balance of the product. There’s no “one-size-fits-all” approach to assess REMS programs, as each REMS assessment  is specifically designed for the specific REMS program. As with any public health initiative, evaluating the effectiveness of a REMS is an iterative process consisting of  periodic assessment and REMS modification if necessary.

To that end, today we’re advancing new guidance on the assessment of REMS programs. The draft guidances (“REMS Assessment: Planning and Reporting Guidance for Industry” and “Survey Methodologies to Assess REMS Goals That Relate to Knowledge Guidance for Industry”) provide a framework for companies to develop a REMS Assessment Plan at the same time that the REMS program is being developed in order to 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.

The REMS Assessment draft guidance describes how to develop a REMS Assessment Plan by specifically considering how the REMS program goals, objectives and REMS 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. The draft guidance 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. 

Examples of metrics and data sources to evaluate the performance of the REMS are provided in the guidance. For example, drug utilization data, surveys of stakeholders, postmarket adverse event data, observational or epidemiological data as well as stakeholder input may all be helpful in assessing a REMS program. We’ve also outlined considerations for assessing the impact of REMS on patient access to the drug or the burden that the REMS impose on the health care delivery system. These considerations are critical implications of instituting REMS and can impact patient care. Properly assessing these isues can help in the design and implementation of more efficient, effective REMS.

The second guidance we are issuing today, the REMS Survey Methodologies draft guidance, provides recommendations on conducting 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 discusses general principles and recommendations related to conducting REMS assessment knowledge surveys, including study design, survey data collection and processing and data analysis.

During this period without a Fiscal Year 2019 appropriation for the FDA, the agency has been focused on making sure it continues critical aspects of our work, to the extent permitted by law. At this time, for products covered by a user fee program, such as those under a REMS, our review of existing medical product applications and associated policy development is funded by limited carryover user fee balances. The FDA will continue to update the public on how we’re approaching our work.

It’s critical that we develop and implement rigorous ways to assess the effectiveness of REMS in real-world settings. We need to make sure that the REMS are achieving their public health goals, and that we’re designing and implementing these approaches in ways that minimize burdens to patients and providers. We know there’s still much to learn about the science of risk management. Approaches and scientific methods for assessing the effectiveness of REMS continue to evolve. Our goal in providing this latest thinking on assessing REMS programs is to ensure we’re constantly improving the programs we have in place today, and establishing the most efficient and effective programs moving forward, to optimize patient care and keep Americans safe and healthy.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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Sarah Peddicord

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