JCL roundtable: Risk evaluation and mitigation strategy.

Many factors enter into the decision by the Food and Drug Administration (FDA) to approve a new drug for use by physicians and other health care providers in treating diseases. Initially, the FDA authority was restricted to issues of safety and only later did the documentation of efficacy become part of the review process required for approval. However, all drugs have the potential for causing harm at some dose level to all and at lower doses in certain patients with vulnerability to the particular pharmacology of the agent. As new drugs have been designed to manage disorders that are uncommon, but of significant consequence, they may have adverse effects that are acceptable only because they are so uniquely beneficial to these specific conditions. The risk of these adverse effects may be acceptable since the benefit can outweigh the harm in most patients and the adversity can be predicted and managed. The approval of this category of drugs has grown rapidly since definition of a mechanism of action to manage and modify the risk has been provided by a process known as known as Risk Evaluation and Mitigation Strategy or "REMS." In 2007, the Food and Drug Administration Amendments Act (FDAAA) allowed the FDA to require postmarketing studies and the authority to mandate the implementation of a REMS for drugs with efficacy but documented potential for harm. Two relatively new drugs useful in the management of severe elevations of low-density lipoprotein cholesterol have been approved under a requirement for a REMS. These are lomitapide, an inhibitor of microsomal triglyceride transfer protein and mipomersen, an antisense oligonucleotide which reduces the synthesis of apolipoprotein B.

Atherosclerosis and teen eating study.

BACKGROUND: Low-density lipoprotein, high-density lipoprotein, serum triglyceride concentrations, blood pressure, obesity, and blood glucose are all known to contribute to risk for development of atherosclerosis. Research demonstrates that dietary modifications, which reduce saturated fat intake, impact the likelihood of development of atherosclerosis. OBJECTIVE: The Atherosclerosis and Teen Eating Study was designed to determine whether a short-term health educational program, complemented by availability of alternative low-fat school lunches, could result in favorable changes to healthier eating patterns. Additionally, the study was designed to measure whether changes in eating behaviors were sufficient to result in risk-factor reduction. METHODS: The 6-week study consisted of a defined educational curriculum in addition to the availability of alternative low-fat cafeteria meals. Six 1-hour educational sessions on heart-healthy nutrition and exercise were presented over a period of 2 weeks. Self-perceived nutritional intake was assessed at baseline and the conclusion of the trial via a documented method, the Eating Pattern Assessment Tool. Routine laboratory work and blood pressure were measured at baseline and conclusion of the study. RESULTS: Eating Pattern Assessment Tool score decreased by 14 points (P < 0.001), indicating a significant fall in fat consumption. Fasting glucose and diastolic blood pressure were also reduced. Mean fasting glucose decreased by 3.1 mg/dL (P = 0.041) and mean diastolic blood pressure fell by 6.6 mmHg (P < 0 .001). The percentage reduction in low-density lipoprotein, the primary endpoint, showed a trend downward but did not achieve statistical significance (P = 0.075). CONCLUSION: School-based educational programs and improved choice of foods focused on cardiovascular risk reduction have the capacity to positively influence eating patterns and risk factors associated with future development of atherosclerosis.