Highlights
- •Compassionate use is use of unauthorized drugs outside of clinical trials.
- •Many countries have introduced legal regulations concerning compassionate use.
- •Compassionate use is associated with a number of ethical challenges.
- •Ethical review of compassionate use is quite controversial.
- •Some ethics codes contain guidelines regarding compassionate use.
Abstract
Compassionate use (also referred to as expanded access) is therapeutic use of unauthorized
drugs outside of clinical trials. The objective of this review is to discuss practical
aspects of the current legal regulations concerning compassionate use that have been
introduced in the European Union, the USA (both the Food and Drug Administration regulations
and Right-to-try laws), Canada and Australia. We also present main ethical challenges
associated with use of unauthorized drugs such as possible difficulties with obtaining
informed consent and fair patient selection. Moreover, we discuss guidelines, especially
those contained in the Declaration of Helsinki, which may aid doctors in the ethical
conduct of compassionate treatments.
Keywords
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Article info
Publication history
Published online: April 26, 2019
Accepted:
April 19,
2019
Received in revised form:
April 10,
2019
Received:
March 15,
2019
Identification
Copyright
© 2019 European Federation of Internal Medicine. Published by Elsevier B.V. All rights reserved.
