title: Measures of outcome for stimulant trials: ACTTION recommendations and research agenda.
abstract: BACKGROUND
  The development and approval of an efficacious pharmacotherapy for stimulant use disorders has been limited by the lack of a meaningful indicator of treatment success, other than sustained abstinence.
  
  
  METHODS
  In March, 2015, a meeting sponsored by Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities, and Networks (ACTTION) was convened to discuss the current state of the evidence regarding meaningful outcome measures in clinical trials for stimulant use disorders. Attendees included members of academia, funding and regulatory agencies, pharmaceutical companies, and healthcare organizations. The goal was to establish a research agenda for the development of a meaningful outcome measure that may be used as an endpoint in clinical trials for stimulant use disorders.
  
  
  CONCLUSIONS
  Based on guidelines for the selection of clinical trial endpoints, the lessons learned from prior addiction clinical trials, and the process that led to identification of a meaningful indicator of treatment success for alcohol use disorders, several recommendations for future research were generated. These include a focus on the validation of patient reported outcome measures of functioning, the exploration of patterns of stimulant abstinence that may be associated with physical and/or psychosocial benefits, the role of urine testing for validating self-reported measures of stimulant abstinence, and the operational definitions for reduction-based measures in terms of frequency rather than quantity of stimulant use. These recommendations may be useful for secondary analyses of clinical trial data, and in the design of future clinical trials that may help establish a meaningful indicator of treatment success.
authors.affiliation: Yale University School of Medicine, West Haven, CT 06516, USA. Electronic address: brian.kiluk@yale.edu.
authors.forename: Brian D
authors.initials: BD
authors.lastname: Kiluk
authors.affiliation: Yale University School of Medicine, West Haven, CT 06516, USA.
authors.forename: Kathleen M
authors.initials: KM
authors.lastname: Carroll
authors.affiliation: Xcenda, Palm Harbor, FL, USA.
authors.forename: Amy
authors.initials: A
authors.lastname: Duhig
authors.affiliation: National Institute on Alcohol Abuse and Alcoholism (NIAAA), Bethesda, MD, USA.
authors.forename: Daniel E
authors.initials: DE
authors.lastname: Falk
authors.affiliation: Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Kyle
authors.initials: K
authors.lastname: Kampman
authors.affiliation: Johns Hopkins School of Medicine, Baltimore, MD, USA.
authors.forename: Shengan
authors.initials: S
authors.lastname: Lai
authors.affiliation: National Institute on Alcohol Abuse and Alcoholism (NIAAA), Bethesda, MD, USA.
authors.forename: Raye Z
authors.initials: RZ
authors.lastname: Litten
authors.affiliation: National Institute on Drug Abuse (NIDA), Bethesda, MD, USA.
authors.forename: David J
authors.initials: DJ
authors.lastname: McCann
authors.affiliation: National Institute on Drug Abuse (NIDA), Bethesda, MD, USA.
authors.forename: Ivan D
authors.initials: ID
authors.lastname: Montoya
authors.affiliation: National Institute on Drug Abuse (NIDA), Bethesda, MD, USA.
authors.forename: Kenzie L
authors.initials: KL
authors.lastname: Preston
authors.affiliation: National Institute on Drug Abuse (NIDA), Bethesda, MD, USA.
authors.forename: Phil
authors.initials: P
authors.lastname: Skolnick
authors.affiliation: Division of Research, Kaiser Permanente, University of California-San Francisco, San Francisco, CA, USA.
authors.forename: Constance
authors.initials: C
authors.lastname: Weisner
authors.affiliation: University of Pennsylvania and Treatment Research Institute, Philadelphia, PA, USA.
authors.forename: George
authors.initials: G
authors.lastname: Woody
authors.affiliation: National Institute on Drug Abuse (NIDA), Bethesda, MD, USA.
authors.forename: Redonna
authors.initials: R
authors.lastname: Chandler
authors.affiliation: Embera Neurotherapeutics, Indiana University School of Medicine, Carmel, IN, USA.
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Detke
authors.affiliation: Johns Hopkins School of Medicine, Baltimore, MD, USA.
authors.forename: Kelly
authors.initials: K
authors.lastname: Dunn
authors.affiliation: University of Rochester School of Medicine and Dentistry, Rochester, NY, USA.
authors.forename: Robert H
authors.initials: RH
authors.lastname: Dworkin
authors.affiliation: National Institute on Alcohol Abuse and Alcoholism (NIAAA), Bethesda, MD, USA.
authors.forename: Joanne
authors.initials: J
authors.lastname: Fertig
authors.affiliation: University of Rochester School of Medicine and Dentistry, Rochester, NY, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Gewandter
authors.affiliation: Virginia Commonwealth University, Richmond, VA, USA.
authors.forename: F Gerard
authors.initials: FG
authors.lastname: Moeller
authors.affiliation: National Institute on Drug Abuse (NIDA), Bethesda, MD, USA.
authors.forename: Tatiana
authors.initials: T
authors.lastname: Ramey
authors.affiliation: National Institute on Alcohol Abuse and Alcoholism (NIAAA), Bethesda, MD, USA.
authors.forename: Megan
authors.initials: M
authors.lastname: Ryan
authors.affiliation: Johns Hopkins School of Medicine, Baltimore, MD, USA.
authors.forename: Kenneth
authors.initials: K
authors.lastname: Silverman
authors.affiliation: Johns Hopkins School of Medicine, Baltimore, MD, USA.
authors.forename: Eric C
authors.initials: EC
authors.lastname: Strain
mesh_terms: D019969:Amphetamine-Related Disorders / Q000175:diagnosis / Q000628:therapy; D000697:Central Nervous System Stimulants / Q000009:adverse effects*; D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards*; D003226:Congresses as Topic; D006801:Humans; D017410:Practice Guidelines as Topic / Q000592:standards*; D019966:Substance-Related Disorders / Q000175:diagnosis / Q000628:therapy*; D016896:Treatment Outcome
chemical_list: D000697:Central Nervous System Stimulants
keywords: Clinical trials; Outcome measures; Stimulant use disorders