Clinical Trial Design Challenges and Opportunities for Emerging Treatments for Opioid Use Disorder: A Review.
JAMA psychiatry – January 01, 2023
Source: PubMed
Summary
Emerging treatments for opioid use disorder (OUD) are essential, as traditional methods often face barriers. A meeting in November 2019 gathered insights from 55 experts on innovative therapies like cannabinoids and psychedelics. Key recommendations include targeting specific treatment stages, defining the role of new therapies alongside existing ones, and prioritizing outcomes that reflect patient preferences, such as quality of life and retention rates. Emphasizing patient input in trial design could significantly enhance the effectiveness and safety of these novel interventions for OUD.
Abstract
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. 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. 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.