NIAAA UPDATE: FOCUS ON THE MEDICATIONS DEVELOPMENT PROGRAM

Alcohol Use Disorder (AUD) is a prevalent and complex disorder that results in a variety of medical, psychological, social, and economic problems. Approximately 27.9 million (10%) Americans are diagnosed with AUD each year, resulting in over 178,000 deaths from alcohol-related causes. Alcohol misuse costs American society more than $249 billion annually. To date, the Food and Drug Administration (FDA) has approved three medications for AUD treatment: disulfiram, acamprosate, and naltrexone, which is available in both oral and long-acting injectable formulations. In addition, several other repurposed compounds have shown efficacy for treating AUD, including nalmefene, baclofen, topiramate, gabapentin, and varenicline. Unfortunately, these medications do not work for everyone, and their effect sizes are modest, due in part to considerable heterogeneity in AUD. To provide more treatment options for AUD, it is critical to identify new medications that are efficacious for reducing alcohol consumption. This presentation will focus on the National Institute on Alcohol Abuse and Alcoholism’s (NIAAA) Medications Development program, highlighting priority areas of interest and providing an overview of the AUD pharmacotherapy portfolio. Grant and contract mechanisms that may be of interest to the ASCP community will be described. These include NIAAA’s IND-enabling program, Alcohol Pharmacotherapy Evaluation Program (APEP), and preclinical program. Other priority areas of interest will be described, including precision medicine, endpoints for Phase III pharmacotherapy trials, and NIAAA-sponsored public resources related to the treatment of AUD (NIAAA Data Archive, Rethinking Drinking, NIAAA Treatment Navigator, and NIAAA Health Care Professional’s Core Resource). More information about NIAAA’s Medication Development program is available at: https://www.niaaa.nih.gov/research/extramural-research/division-treatment-and-recovery-dtr.

Learning Objective 1: To understand NIAAA’s Medications Development Program, including the AUD medication pipeline, current portfolio, and programmatic priorities.

Learning Objective 2: To understand NIAAA-sponsored public resources related to the treatment of AUD.

References

Soyka M, Rösner S. Modern pharmacotherapy guidance for treating alcohol use disorders. Expert Opin Pharmacother. 2025 Feb;26(2):147-156. doi: 10.1080/14656566.2024.2445734. Epub 2024 Dec 30. PMID: 39702947. Litten RZ, Falk DE, Ryan ML, Fertig J, Leggio L. Five priority areas for improving medications development for alcohol use disorder and promoting their routine use in clinical practice. Alcohol Clin Exp Res. 2020 Jan;44(1):23-35. doi: 10.1111/acer.14233. Epub 2019 Dec 5. PMID: 31803968.