Outcome Measures in Medication Trials for Substance Use Disorders

摘要: There are multiple therapeutic options to treat tobacco (e.g., nicotine replacement therapies, varenicline) and alcohol naltrexone, acamprosate) use disorders. In contrast, there currently no FDA-approved pharmacotherapies stimulant cocaine, methamphetamine) Based on a commentary published by FDA staff, period of sustained abstinence appears required for regulatory approval first-in-class medication Certainly, achieving remains the goal treatment in both real world medical practice clinical trials. However, if can help patients significantly reduce (short abstinence) while attempting quit, such reductions could confer meaningful benefit. The has adopted “percentage subjects with heavy drinking days” as an endpoint pharmacotherapy trials disorders; this suggests may be potential path forward developing analogous, non-abstinence endpoints disorder drug per se must have prognostic value order considered acceptable basis approval. Thus, even provide use, challenge ahead is demonstrate that “success” accompanied benefits accrue dimensions readily understood their families, sufficient reimbursed third party payers. Emerging data sets discussed paper indicate other than ultimately validated outcome measures