Compassionate use of unauthorized drugs: Legal regulations and ethical challenges

  • Jan Borysowski
    Corresponding author.
    Centre for Studies on Research Integrity, Institute of Law Studies, Polish Academy of Sciences, Nowy Świat 72, 00-330 Warsaw, Poland

    Department of Clinical Immunology, Medical University of Warsaw, Nowogrodzka Str. 59, 02-006 Warsaw, Poland
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  • Andrzej Górski
    Department of Clinical Immunology, Medical University of Warsaw, Nowogrodzka Str. 59, 02-006 Warsaw, Poland

    Laboratory of Bacteriophages, Ludwik Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Rudolfa Weigla Str. 12, 53-114 Wrocław, Poland
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Published:April 26, 2019DOI:


      • 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.


      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.


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