Assessing the Effectiveness of REMS: FDA Drafts Two New Guidances

Regulatory NewsRegulatory News | 24 January 2019 |  By 

The US Food and Drug Administration (FDA) on Thursday released two new draft guidance documents aiming to help improve the agency’s ability to ensure that Risk Evaluation and Mitigation Strategies (REMS) put in place for certain drugs and biologics are working properly.

The guidances provide industry with a framework to develop a REMS Assessment Plan to better develop a REMS program and improve the quality of the information used to assess the effectiveness of it, and to provide postmarket evidence that the REMS is meeting its risk mitigation goals.


A REMS is a drug safety program that FDA can require for certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks. Currently, FDA has determined that a REMS is necessary for 75 medicines.

Most REMS require communication between a doctor and patient, for example, about specific safety 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 a patient about the potential risks.

From the onset of the REMS, FDA works closely with companies that sell these products to determine the best way to assess its effectiveness in ensuring the benefit-risk balance of the product.

There’s no “one size fits all” approach to assess REMS programs, FDA says, as each assessment is specifically designed for the specific REMS program. And as with any public health initiative, evaluating the effectiveness of a REMS is an iterative process consisting of periodic assessment and modification, if necessary, the agency adds.


The 23-page REMS assessment draft guidance describes how to develop a REMS Assessment Plan by considering how the REMS program goals, objectives and REMS design may impact the types of metrics and data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.

“For example, if the REMS includes a goal to mitigate the risk of a drug and an objective to inform or educate patients about the safe use of that drug, prescribers could be required to counsel their patients using a REMS material such as a patient-prescriber agreement,” the draft says.

The draft also discusses how to assess the impact of REMS on patient access to a drug and its burden to the health care delivery system.

It 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, but can also include unintended outcomes (i.e., barriers to patient access).

Examples of metrics and data sources to evaluate the performance of the REMS are provided in the guidance, including: drug utilization data, surveys of stakeholders, postmarket adverse event data, observational or epidemiological data as well as stakeholder input. These may all be helpful in assessing a REMS program.

The other draft guidance, a 23-page document on REMS survey methodologies, 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 such assessment knowledge surveys, including study design, survey data collection and processing and data analysis.

“The endpoints of interest are measurements of knowledge of the REMS key messages in a target population of interest (e.g., patients, prescribers, dispensers, or others, as appropriate). At the subject level, the knowledge endpoint is a binary outcome indicating whether the subject knows or does not know the key message. In a target population, the endpoint is the knowledge rate of a REMS key message. The knowledge rate is the proportion of subjects who know the key message out of all subjects; it is also the chance that a given subject in the target population knows the key message,” the draft explains.

REMS Assessment: Planning and Reporting Guidance for Industry

Survey Methodologies to Assess REMS Goals That Relate to Knowledge Guidance for Industry


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Tags: FDA, guidance, REMS

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