FDA issues new guidance for Safety Trials for Diabetes medicines

The U.S. Food and Drug Administration today issued a new draft guidance, "Type 2 Diabetes Mellitus: Evaluating the Safety of New Drugs for Improving Glycemic Control," to solicit public comment on its proposals for broad safety evaluations before drug approval that look beyond ischemic (reduced blood flow) cardiovascular disease, as well as for the inclusion of a broader scope of human subjects, such as older subjects and those with chronic kidney disease who may be more vulnerable to drug-related side effects.

The new draft guidance does not contain the recommendation that sponsors of all new therapies for type 2 diabetes uniformly rule out a specific degree of risk for ischemic cardiovascular adverse outcomes, which was recommended in previous guidance and has typically been done through cardiovascular outcome trials.

"The FDA continues to review new clinical trial evidence and update its recommendations for drug development to reflect emerging scientific information," said Lisa Yanoff, M.D., acting director of the Division for Metabolism and Endocrinology Products in the FDA's Center for Drug Evaluation and Research. "By following previous FDA recommendations, sponsors have shown that new type 2 diabetes drugs do not have excess ischemic cardiovascular risk, which has provided reassuring cardiovascular safety information for millions of diabetes patients. Now, with this proposed approach, we will have broader, valuable safety information for these medications."

The 2020 draft guidance stems from the ongoing evaluation of cardiovascular outcomes trials and recommendations from the FDA's Endocrinologic and Metabolic Drugs Advisory Committee, which noted safety data beyond ischemic cardiovascular safety data was desired to evaluate the safety profile of antidiabetic drugs before they were approved.

More than 30 million Americans have diabetes, and 90% to 95% of them have type 2 diabetes. People with type 2 diabetes can suffer from serious medical complications, such as nerve damage, kidney and eye damage, sleep apnea, and heart and blood vessel disease, as a result of high glucose (blood sugar) levels in the blood. These patients may also be more likely to die prematurely compared with people without type 2 diabetes.

While a healthy lifestyle may slow or even stop the disease, many patients also take medications to improve glycemic (blood sugar) control. It is important that companies robustly evaluate the medications' safety in clinical trials to identify risks. This knowledge can help patients and their health care providers make the most informed treatment decisions.

The 2020 draft guidance will replace the FDA guidances for industry, "Diabetes Mellitus – Evaluating Cardiovascular Risk in New Antidiabetic Therapies to Treat Type 2 Diabetes," published in December 2008, and "Diabetes Mellitus: Developing Drugs and Therapeutic Biologics for Treatment and Prevention," published in February 2008.

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.