A Glossary of REMS-Related Terms
Abbreviated New Drug Application (ANDA)
The drug application submitted to the Food and Drug Administration to approve a generic version of a branded drug.
A part of a medicine that produces a biological effect.
Adverse Drug Event (ADE)
A reaction for which there is a reasonable possibility that the medical problem may have been caused by the drug.
An assessment of whether the therapeutic benefits of taking a medicine outweigh the risks involved.
A series of tests to determine that two or more drug products contain the same active ingredients and are equivalent in dosage, strength, route of administration, quality, performance, and intended use.
Warning text required by FDA that is placed at the top of the prescribing information to indicate that the drug carries a risk of a serious, possibly life-threatening adverse event.
A REMS program for a class of drugs that share a common set of risks where a single REMS system can be used by different manufacturers.
Developed by the drug’s sponsor to support implementation of a REMS program, a communications plan is intended to inform key audiences (health care providers) about the risks of the drug. The communication plan may include sending letters to healthcare providers; disseminating information about REMS elements to encourage implementation by health care providers or to explain certain safety protocols; and disseminating information to health care providers through professional societies about any serious risks of the drug and any protocol to assure safe use.
Consumer Medical Information (CMI)
Written information about prescription medicines developed by organizations or individuals other than a drug’s manufacturer that is intended for distribution to consumers at the time of drug dispensing. CMI is currently not reviewed or approved by FDA.
Elements to Assure Safe Use (ETASU)
The most extensive elements of a REMS, “Elements to Assure Safe Use” (ETASU) are carefully planned safety protocols required when the Food and Drug Administration (FDA) determines that a medication can only be marketed with stringent controls to ensure a serious or fatal risk can be avoided by proper use of the drug. ETASU elements include physician qualification and registration; patient enrollment; verification that certain conditions have been met, such as a negative pregnancy test, before the drug is dispensed; and certification and education of dispensing pharmacies.
Safety reports about serious adverse events associated with a drug that require reporting within a short timeframe to FDA.
FDA Adverse Event Reporting System (FAERS)
A database operated by FDA that contains information on adverse event and medication error reports submitted to the agency. FDA evaluates the reports in FAERS to monitor the safety of drugs and biologics after they are approved. Based on the potential safety risks identified through FAERS, FDA may require new information on the product’s labeling, restrict the use of the drug, and in rare cases, take the drug off the market.
Food and Drug Administration Amendments Act (FDAAA)
Legislation enacted in 2007 in which Congress gave FDA many new authorities and responsibilities to enhance drug safety, including the power to require a Risk Evaluation and Mitigation Strategy (REMS).
A drug that is the same as the brand name product in dosage, safety, strength, how it is taken, quality, performance, and intended use.
A description of how certain Elements to Assure Safe Use will be implemented.
Not usually required unless the REMS program includes Elements to Assure Safe Use, a Medication Guide contains information for patients written in non-technical language on how to take a drug safely. Healthcare providers are required to dispense a Medication Guide with each prescription as a review tool in counseling the patient on the risks of the prescribed medicine.
A system maintained by FDA for the voluntary reporting of adverse events, potential and actual medical product errors, and product quality problems associated with the use of drugs, biologics, devices, and dietary supplements.
New Drug Application (NDA)
The application submitted to the FDA when the sponsor of a new drug believes that enough evidence on the drug’s safety and effectiveness has been obtained to meet FDA’s requirements for marketing approval. The NDA must contain data from clinical studies as well as detailed information on the drug’s chemistry, pharmacology, and biopharmaceutics.
New Safety Information
Defined as a serious risk associated with use of a drug of which FDA has become aware since the drug was approved, since a REMS was required, or since the last assessment of the REMS.
Patient Package Insert (PPI)
Contains information for patients’ understanding of how to safely use a drug product. Today, the Medication Guide has largely replaced the PPI as an element of a REMS.
PDUFA (Prescription Drug User Fee Act)
First enacted in in 1992 (PDUFA I), this legislation gives FDA the authority to collect fees from industry to accelerate its drug evaluation process without compromising review quality. Reauthorized by the Congress every 5 years, the most recent PDUFA reauthorization (PDUFA V), the Food and Drug Administration Safety and Innovation Act (FDASIA), was enacted in2012.
Signed into law on July 9, 2012, the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 reauthorized PUDFA through September 30, 2017. FDASIA also specifies FDA’s performance goals in 5 general areas: (1) drug review performance goals, communication, and timing; (2) regulatory science; (3) risk-benefit assessment in decision-making; (4) drug safety; and (5) information technology and performance management.
Post Approval Study (PAS)
Under FDA’s REMS authority, the agency may require the manufacturer to conduct studies after the drug is approved to obtain further evidence of its safety and effectiveness. The studies are often required when newly acquired information raise concerns about potential unanticipated safety risks.
REMS (Risk Evaluation and Mitigation Strategy)
A REMS is a risk management plan that goes beyond the requirements in the drug prescribing information to manage serious risks associated with a drug and to enable patients to have continued access to such medicines by managing their safe use.
REMS Supporting Document
Includes a thorough explanation of the rationale for, and supporting information about, the content of the REMS.
Although rarely required by the FDA, restricted distribution systems may be necessary to ensure a medicine known to cause birth defects, organ damage and other serious or life-threating events is only acquired and used by patients under carefully controlled conditions. These systems may require physician qualification and registration, patient informed consent, pharmacist distribution limitations, controls to ensure the safe return and disposal of unused medications, patients enrolling in a registry and patients undergoing routine testing, such as women having a monthly pregnancy test.
The application of scientifically-based methodologies to identify, assess, communicate and minimize the risks of a medicine in order to maintain the drug’s favorable benefit-risk profile in patients.
Risk Management Plan (RMP)
A set of activities designed to identify, characterize, prevent, or minimize the risks of medicine use; to assess the effectiveness of these interventions; and to communicate the risks to patients and health care providers.
Risk Minimization Action Plan (Risk MAP)
A strategic safety program designed to meet specific goals and objectives in minimizing known risks of a product while preserving its benefits. FDA issued guidances for developing a RiskMAP in 2005. RiskMAPs were replaced by REMS after the program became effective in March, 2008.
Serious Adverse Event (Serious Adverse Experience)
Any unfavorable and unintended sign, symptom, or medical problem occurring at any dose of a drug that results in death, a life-threatening adverse drug experience, hospitalization, a significant disability/incapacity, or a congenital anomaly/birth defect.
Timetable for Submission of Assessments
The schedule for evaluating the effectiveness of a brand manufacturer’s REMS safety measures. FDA requires that, at a minimum, assessments be conducted at 18 months, 3 years, and 7 years after the REMS is approved with the results reported to the agency. Results may be used to modify the REMS, or even eliminate it, if the assessment shows changes are needed or that the REMS has met its goals.
The capability of a drug or other substance to cause death or injury. The level of toxicity associated with a medicine may vary with the condition for which the medicine is used, as well as the dosage.
Unsuspected Serious Risk
A serious adverse event that is not listed in the labeling of a drug or differs from the adverse drug experience identified in the labeling due to greater severity, specificity, or prevalence.