A call for consensus in defining efficacy in clinical trials for opioid addiction: combined results from a systematic review and qualitative study in patients receiving pharmacological assisted therapy for opioid use disorder

Trials. 2020 Jan 6;21(1):30. doi: 10.1186/s13063-019-3995-y.


Background: Given the complex nature of opioid addiction treatment and the rising number of available opioid substitution and antagonist therapies (OSAT), there is no 'gold standard' measure of treatment effectiveness, and each successive trial measures a different set of outcomes which reflect success in arbitrary or opportune terms. We sought to describe the variation in current outcomes employed across clinical trials for opioid addiction, as well as determine whether a discrepancy exists between the treatment targets that patients consider important and how treatment effectiveness is measured in the literature.

Methods: We searched nine commonly used databases (e.g., EMBASE, MEDLINE) from inception to August 1, 2015. Outcomes used across trials were extracted and categorized according to previously established domains. To evaluate patient-reported goals of treatment, semi-structured interviews were conducted with 18 adults undergoing methadone treatment.

Results: We identified 60 trials eligible for inclusion. Once outcomes were categorized into eight broad domains (e.g., abstinence/substance abuse), we identified 21 specific outcomes with furthermore 53 subdomains and 118 measurements. Continued opioid use and treatment retention were the most commonly reported measures (46%, n = 28). The majority of patients agreed that abstinence from opioids was a primary goal in their treatment, although they also stressed goals under-reported in clinical trials.

Conclusions: There is inconsistency in the measures used to evaluate the effectiveness of OSATs. Individual and population level decision making is being guided by a standard of effect considered useful to researchers yet in direct conflict with what patients deem important.

Trial registration: PROSPERO, CRD42013006507.

Keywords: Clinical trials; Efficacy; Methodology; Opioid addiction; Patient important outcomes; Treatment effectiveness.

Publication types

  • Systematic Review

MeSH terms

  • Clinical Trials as Topic / standards*
  • Consensus*
  • Humans
  • Opiate Substitution Treatment / methods*
  • Opioid-Related Disorders / rehabilitation*
  • Qualitative Research
  • Research Design / standards*
  • Treatment Outcome