TY - JOUR
T1 - Clinical Trial Design Challenges and Opportunities for Emerging Treatments for Opioid Use Disorder
T2 - A Review
AU - Kiluk, Brian D.
AU - Kleykamp, Bethea A.
AU - Comer, Sandra D.
AU - Griffiths, Roland R.
AU - Huhn, Andrew S.
AU - Johnson, Matthew W.
AU - Kampman, Kyle M.
AU - Pravetoni, Marco
AU - Preston, Kenzie L.
AU - Vandrey, Ryan
AU - Bergeria, Cecilia L.
AU - Bogenschutz, Michael P.
AU - Brown, Randall T.
AU - Dunn, Kelly E.
AU - Dworkin, Robert H.
AU - Finan, Patrick Hamilton
AU - Hendricks, Peter S.
AU - Houtsmuller, Elisabeth J.
AU - Kosten, Thomas R.
AU - Lee, Dustin C.
AU - Levin, Frances R.
AU - Mcrae-Clark, Aimee
AU - Raison, Charles L.
AU - Rasmussen, Kurt
AU - Turk, Dennis C.
AU - Weiss, Roger D.
AU - Strain, Eric C.
N1 - Funding Information:
Funding/Support: Financial support for this article was provided by the Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities, and Networks (ACTTION) public-private partnership with the US Food and Drug Administration (grant U01-FD005936).
Publisher Copyright:
© 2023 American Medical Association. All rights reserved.
PY - 2023/1/4
Y1 - 2023/1/4
N2 - Importance: Novel treatments for opioid use disorder (OUD) are needed to address both the ongoing opioid epidemic and long-standing barriers to existing OUD treatments that target the endogenous μ-opioid receptor (MOR) system. The goal of this review is to highlight unique clinical trial design considerations for the study of emerging treatments for OUD that address targets beyond the MOR system. In November 2019, the Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities, and Networks (ACTTION) public-private partnership with the US Food and Drug Administration sponsored a meeting to discuss the current evidence regarding potential treatments for OUD, including cannabinoids, psychedelics, sedative-hypnotics, and immunotherapeutics, such as vaccines. Observations: Consensus recommendations are presented regarding the most critical elements of trial design for the evaluation of novel OUD treatments, such as: (1) stage of treatment that will be targeted (eg, seeking treatment, early abstinence/detoxification, long-term recovery); (2) role of treatment (adjunctive with or independent of existing OUD treatments); (3) primary outcomes informed by patient preferences that assess opioid use (including changes in patterns of use), treatment retention, and/or global functioning and quality of life; and (4) adverse events, including the potential for opioid-related relapse or overdose, especially if the patient is not simultaneously taking maintenance MOR agonist or antagonist medications. Conclusions and Relevance: Applying the recommendations provided here as well as considering input from people with lived experience in the design phase will accelerate the development, translation, and uptake of effective and safe therapeutics for individuals struggling with OUD.
AB - Importance: Novel treatments for opioid use disorder (OUD) are needed to address both the ongoing opioid epidemic and long-standing barriers to existing OUD treatments that target the endogenous μ-opioid receptor (MOR) system. The goal of this review is to highlight unique clinical trial design considerations for the study of emerging treatments for OUD that address targets beyond the MOR system. In November 2019, the Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities, and Networks (ACTTION) public-private partnership with the US Food and Drug Administration sponsored a meeting to discuss the current evidence regarding potential treatments for OUD, including cannabinoids, psychedelics, sedative-hypnotics, and immunotherapeutics, such as vaccines. Observations: Consensus recommendations are presented regarding the most critical elements of trial design for the evaluation of novel OUD treatments, such as: (1) stage of treatment that will be targeted (eg, seeking treatment, early abstinence/detoxification, long-term recovery); (2) role of treatment (adjunctive with or independent of existing OUD treatments); (3) primary outcomes informed by patient preferences that assess opioid use (including changes in patterns of use), treatment retention, and/or global functioning and quality of life; and (4) adverse events, including the potential for opioid-related relapse or overdose, especially if the patient is not simultaneously taking maintenance MOR agonist or antagonist medications. Conclusions and Relevance: Applying the recommendations provided here as well as considering input from people with lived experience in the design phase will accelerate the development, translation, and uptake of effective and safe therapeutics for individuals struggling with OUD.
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U2 - 10.1001/jamapsychiatry.2022.4020
DO - 10.1001/jamapsychiatry.2022.4020
M3 - Review article
C2 - 36449315
AN - SCOPUS:85145641637
SN - 2168-622X
VL - 80
SP - 84
EP - 92
JO - JAMA psychiatry
JF - JAMA psychiatry
IS - 1
ER -