JCL roundtable: Risk evaluation and mitigation strategy.

Area of Science:

• Pharmacology and Drug Approval Processes
• Cardiovascular Disease Management
• Regulatory Science

Background:

• Drug approval initially focused on safety, later incorporating efficacy.
• All drugs carry risks, with some patients being more vulnerable to adverse effects.
• Novel therapies for rare, significant conditions may have acceptable risks if benefits outweigh harms.

Purpose of the Study:

• To explain the role of Risk Evaluation and Mitigation Strategies (REMS) in drug approval.
• To highlight the FDA's authority to mandate REMS for drugs with potential harm.
• To introduce new drugs for severe high cholesterol approved with REMS.

Main Methods:

• Review of FDA drug approval processes and regulatory frameworks.
• Examination of the Risk Evaluation and Mitigation Strategy (REMS) mechanism.
• Identification of specific drugs (lomitapide, mipomersen) approved under REMS for hypercholesterolemia.

Main Results:

• The FDAAA of 2007 granted the FDA authority to require postmarketing studies and REMS.
• REMS allows for the management and modification of risks associated with beneficial drugs.
• Lomitapide and mipomersen are examples of drugs approved with REMS for managing severe low-density lipoprotein cholesterol elevations.

Conclusions:

• REMS is a critical tool for balancing drug efficacy with patient safety.
• The use of REMS has expanded for drugs treating serious conditions with inherent risks.
• Lomitapide and mipomersen represent advancements in managing severe hypercholesterolemia under regulatory oversight.