Today, the U.S. Food and Drug Administration published a new draft guidance to assist sponsors in developing treatments for stimulant use disorders. The guidance, Stimulant Use Disorders: Developing Drugs for Treatment, when finalized, will be the first to provide the FDA’s current thinking on the overall development program and clinical trial design to develop drugs and biologics to support treatment of moderate to severe cocaine use disorder, methamphetamine use disorder and prescription stimulant use disorder. 

“Currently there is no FDA-approved medication for stimulant use disorder. When finalized, we hope that the guidance will support the development of novel therapies that are critically needed to address treatment gaps," said Marta Sokolowska, Ph.D., deputy center director for Substance Use and Behavioral Health in FDA’s Center for Drug Evaluation and Research. “The guidance is one of the actions within the agency’s Overdose Prevention Framework, which includes appropriate prescribing of prescription stimulants as well as development of evidence-based treatments for stimulant use disorder.”

Among other things, the draft guidance contains recommendations regarding clinical trial design related to evaluating stimulant use disorder treatments. Designing clinical studies to evaluate the safety and effectiveness of stimulant use disorder treatments presents a number of unique challenges—from selecting appropriate populations for studies to determining the most appropriate clinical endpoints—that require careful review. However, there are opportunities to improve clinical trial design and develop trials that are more person-centered, which may result in increased sensitivity to detect a treatment effect, with the potential for treatment effects to be linked to meaningful long-term outcomes. 

The draft guidance also describes basic considerations throughout the drug development process including trial conduct, data collection, methods to assess treatment response, subject safety and new drug application requirements. For example, the guidance incorporates lessons learned about approaches that are unlikely to be successful and reflects current recommendations about approaches for treating stimulant use disorders and evaluating response to treatment.

Stimulant use disorder describes a range of symptoms associated with the use of stimulant drugs, including methamphetamine, cocaine, and amphetamines, but not including caffeine or nicotine. A diagnosis of stimulant use disorder is made when a clinician identifies a pattern of use of an amphetamine-type substance, cocaine, or other stimulant that leads to clinically significant impairment or distress, including an inability to reduce or control consumption, cravings to use a stimulant, continued use of a stimulant despite it causing negative consequences, and the need to use increased amounts of a stimulant to achieve the desired effect. 

The agency has taken action to promote safe use and appropriate prescribing of prescription stimulants by requiring sponsors to update and standardize prescribing information for medications used to treat attention deficit/hyperactivity disorder (ADHD) and other disorders where stimulants are prescribed. Additionally, the FDA awarded the National Academies of Sciences, Engineering, and Medicine a grant to convene a workshop on the diagnosis and treatment of ADHD in adults. It has also funded several research projects to inform prevention of prescription stimulant misuse, addiction and overdose.

The FDA continues to encourage the development of treatments for stimulant use disorder and novel trial designs. Over the last several years, the agency has held workshops and public meetings with patients and patient advocates, researchers, industry and other stakeholder groups to better understand the stimulant use disorder landscape and inform the FDA’s understanding of the clinical context for drug review and regulatory decision making. 

Comments on the draft guidance are requested within 60 days to ensure the agency considers them.