Ethical and clinical safety considerations in the design of an effectiveness trial

A comparison of buprenorphine versus naltrexone treatment for opioid dependence

Edward V. Nunes, Joshua Lee, Dominic Sisti, Andrea Segal, Arthur Caplan, Marc Fishman, Genie Bailey, Gregory Brigham, Patricia Novo, Sarah Farkas, John Rotrosen

Research output: Contribution to journalArticle

Abstract

We examine ethical challenges encountered in the design of an effectiveness trial (CTN-0051; X:BOT), comparing sublingual buprenorphine-naloxone (BUP-NX), an established treatment for opioid dependence, to the newer extended-release injectable naltrexone (XR-NTX). Ethical issues surrounded: 1) known poor effectiveness of one possible, commonly used treatment as usual control condition—detoxification followed by counseling without medication; 2) the role of patients' preferences for treatments, given that treatments were clinically approved and available to the population; 3) differences between the optimal “usual treatment” clinical settings for different treatments making it challenging to design a fair comparison; 4) vested interest groups favoring different treatments exerting potential influence on the design process; 5) potentially vulnerable populations of substance users and prisoners; 6) potential therapeutic misconception in the implementation of safety procedures; and 7) high cost of a large trial limiting questions that could be addressed. We examine how the design features underlying these ethical issues are characteristic of effectiveness trials, which are often large trials that compare treatments with varying degrees of existing effectiveness data and familiarity to patients and clinicians, in community-based treatment settings, with minimal exclusion criteria that could involve vulnerable populations. Hence, investigators designing effectiveness trials may wish to remain alert to the possibility of similar ethical issues.

Original languageEnglish (US)
Pages (from-to)34-43
Number of pages10
JournalContemporary Clinical Trials
Volume51
DOIs
StatePublished - Nov 1 2016

Fingerprint

Buprenorphine
Naltrexone
Opioid Analgesics
Safety
Ethics
Therapeutics
Vulnerable Populations
Therapeutic Misconception
Prisoners
Public Opinion
Patient Preference
Counseling
Research Personnel
Costs and Cost Analysis
Injections

Keywords

  • Buprenorphine
  • Clinical trial
  • Effectiveness
  • Ethics
  • Naltrexone
  • Opioid dependence

ASJC Scopus subject areas

  • Medicine(all)
  • Pharmacology (medical)

Cite this

Ethical and clinical safety considerations in the design of an effectiveness trial : A comparison of buprenorphine versus naltrexone treatment for opioid dependence. / Nunes, Edward V.; Lee, Joshua; Sisti, Dominic; Segal, Andrea; Caplan, Arthur; Fishman, Marc; Bailey, Genie; Brigham, Gregory; Novo, Patricia; Farkas, Sarah; Rotrosen, John.

In: Contemporary Clinical Trials, Vol. 51, 01.11.2016, p. 34-43.

Research output: Contribution to journalArticle

Nunes, Edward V. ; Lee, Joshua ; Sisti, Dominic ; Segal, Andrea ; Caplan, Arthur ; Fishman, Marc ; Bailey, Genie ; Brigham, Gregory ; Novo, Patricia ; Farkas, Sarah ; Rotrosen, John. / Ethical and clinical safety considerations in the design of an effectiveness trial : A comparison of buprenorphine versus naltrexone treatment for opioid dependence. In: Contemporary Clinical Trials. 2016 ; Vol. 51. pp. 34-43.
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