New FDA Draft Guidance Pushes for Development of Stimulant Use Disorder Treatments
With no FDA drug approvals to date for stimulant use disorder, the FDA has published draft guidance outlining current recommendations for development programs and trial designs of moderate-to-severe cocaine use, methamphetamine use and prescription stimulant use disorder.
The 14-page draft guidance offers the latest agency thinking on early phase development, efficacy trial considerations, endpoints, benefit-risk considerations, labeling, and the use of expedited programs for stimulant use disorder treatments, though ever-changing evidence and continued FDA research in this area means the recommendations aren’t likely to remain static.
“This guidance reflects current recommendations based on a number of uncertainties about the best approaches for treating stimulant use disorder and … evaluating responses to treatment. This guidance also incorporates lessons learned about approaches for evaluating responses to treatment that are unlikely to be successful,” the agency said. “As the evidence supporting the development of drugs for stimulant use disorder treatment evolves, the recommendations in this guidance and any recommendations given to sponsors at milestone meetings may change.”
Importantly, the FDA cautions sponsors about prospective challenges when it comes to designing trials to evaluate the safety and effectiveness of stimulant use disorder drugs, including selecting trial populations and choosing the most appropriate clinical endpoints.
In addressing these hurdles, “there are opportunities to improve clinical trial design and develop trials that are more person-centered,” the agency notes, “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 guidance also notes the strong heterogeneity of patients meeting DSM-5 criteria for stimulant use disorder and the resulting challenge of identifying drugs that are effective for the whole subset of patients diagnosed with cocaine/methamphetamine use disorder and patients meeting the broader criteria for stimulant use disorder. It also calls out the differing drug mechanisms between cocaine, methamphetamine and other stimulants that can contribute to differences in clinical presentation and treatment responses among patients as another challenge.
The FDA is open to discussing various approaches to stimulant use disorder drug development programs, the guidance emphasizes, and sponsors are encouraged to get in touch with the agency early on in the process.
Read the full guidance here.