FDA Statement on Clinical Trials for Drugs to Potentially Treat Alcoholism
Media attention to FDA’s process for reviewing investigational drugs to treat patients with alcohol addiction provides the Agency an opportunity to correct the misconception that abstinence alone would be the only valuable endpoint the FDA considers when evaluating a new treatment for alcoholism.
In fact, FDA has accepted that drinking patterns other than abstinence can be valid predictors of clinical benefit for patients in drug studies. For example, analyses provided by the National Institutes on Alcohol Abuse and Alcoholism (NIAAA), as well as other data sources, support the conclusion that patients with alcohol use disorders can largely avoid the known consequences of drinking if they never have a heavy drinking day, while patients who violate the “heavy drinking” limit, even rarely, are at higher risk of poor clinical outcomes. These analyses support the use of a “no heavy-drinking days” endpoint, which FDA has used as a basis for approval.
In addition, FDA is engaged in cooperative efforts with academia, industry, NIAAA, and the European Medicines Agency to explore whether other patterns of drinking can be used in clinical trials to predict a clinical benefit for people who want to stop drinking.