Ethics framework for treatment use of investigational drugs.

BMC Medical Ethics
Jan Borysowski, Andrzej Górski

Abstract

Expanded access is the use of investigational drugs (IDs) outside of clinical trials. Generally it is performed in patients with serious and life-threatening diseases who cannot be treated satisfactorily with authorized drugs. Legal regulations of expanded access to IDs have been introduced among others in the USA, the European Union (EU), Canada and Australia. In addition, in the USA an alternative to expanded access is treatment under the Right-to-Try law. However, the treatment use of IDs is inherently associated with a number of ethically relevant problems. The objective of this article is to present a coherent framework made up of eight requirements which have to be met for any treatment use of an ID to be ethical. These include a justified need for the use of an ID, no threat to clinical development of the ID, adequate scientific evidence to support the treatment, patient's benefit as the primary goal of the use of an ID, informed decision of a patient, fair access of patients to IDs, independent review, as well as the dissemination of treatment results. While this framework is essentially consistent with the legal regulations of expanded access of the USA, the EU, Canada and Australia, it is substantially wider in scope ...Continue Reading

References

Feb 1, 2014·Ecancermedicalscience·Cecilia Nardini
Sep 25, 2014·Wiener medizinische Wochenschrift·Gerhard Hamilton, Barbara Rath
Oct 18, 2014·The American Journal of Bioethics : AJOB·Mary Jean WalkerVikki Entwistle
Dec 17, 2014·Personalized Medicine·Benjamin E BerkmanLisa Eckstein
Jan 15, 2015·The New England Journal of Medicine·Jonathan J DarrowAaron S Kesselheim
Feb 2, 2015·The Lancet Oncology·Paul LoriganAndrew M Hudson
Mar 12, 2015·The New England Journal of Medicine·Monique L AndersonRobert M Califf
May 23, 2015·Expert Opinion on Pharmacotherapy·Arthur L Caplan, Alison Bateman-House
Jan 14, 2016·Trials·Antonella IudicelloPaola Mosconi
Feb 13, 2016·JAMA : the Journal of the American Medical Association·Arthur L Caplan, Amrit Ray
Mar 10, 2016·Perspectives in Clinical Research·Sanjaykumar Patil
Aug 19, 2016·Viruses·Che C Colpitts, Thomas F Baumert
Sep 13, 2016·Journal of Infection and Public Health·Marianne FrieriAnthony Boutin
Dec 3, 2016·Intractable & Rare Diseases Research·Gayathri BalasubramanianBehsad Zomorodi
Dec 6, 2016·Therapeutic Innovation & Regulatory Science·Jonathan P JarowRichard Moscicki
Mar 30, 2017·Lancet·Kjell Asplund, Göran Hermerén
Jul 1, 2017·The Journal of Law, Medicine & Ethics : a Journal of the American Society of Law, Medicine & Ethics·Jeffrey P Spike
Jul 25, 2017·BMC Medicine·Jan BorysowskiAndrzej Górski
Oct 3, 2017·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Edward S KimRichard L Schilsky
Dec 23, 2017·Clinical Pharmacology and Therapeutics·Emily JungAaron S Kesselheim
Feb 6, 2018·The American Journal of Geriatric Psychiatry : Official Journal of the American Association for Geriatric Psychiatry·Elissa KolvaRebecca Saracino
May 4, 2018·Therapeutic Innovation & Regulatory Science·Kelly McBride Folkers, Alison Bateman-House
Aug 12, 2018·Journal of Nuclear Medicine : Official Publication, Society of Nuclear Medicine·Brandon BrownKarine Dubé
Feb 10, 2019·Orphanet Journal of Rare Diseases·Andrew E MulbergVivian Kessler
May 1, 2019·European Journal of Internal Medicine·Jan Borysowski, Andrzej Górski
May 18, 2019·American Society of Clinical Oncology Educational Book·Ryan D NippElectra D Paskett
Oct 10, 2019·JAMA Network Open·Victoria L BartlettJoseph S Ross
Oct 10, 2019·The New England Journal of Medicine·Jinkuk KimTimothy W Yu
Oct 16, 2019·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Holly Fernandez LynchPatricia J Zettler
Jun 9, 2020·Frontiers in Pharmacology·Ricardo Eccard da SilvaClaudia G S Osorio-de-Castro

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