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. 2016 Feb 15;4:18–26. doi: 10.1016/j.reth.2015.11.002

Table 2.

The EU's compassionate use mechanism for drugs.

Country (regulator) France (ANSM) France (ANSM) Germany (BfArM) UK (MHRA)
Category Nominative ATU Cohort ATU Compassionate Use Specials
Safety reporting regulation Articles R. 5121-150 et seq. of the French code of Public Health Articles R. 5121-150 et seq. of the French code of Public Health Ordinance on the placing on the market of unauthorized medicinal products for compassionate use.
  • •

    Regulation 170 of The Human Medicines Regulations 2012.

  • •

    The supply of unlicensed medicinal products (“specials”) MHRA Guidance Note 14.
Regulation
  • •

    DIRECTIVE 2001/83/EC Article 5

  • •

    Article L.5121-12 of the French Public Health Code
  • •

    REGULATION (EC) No 726/2004 Article 83

  • •

    Article L.5121-12 of the French Public Health Code
  • •

    REGULATION (EC) No 726/2004 Article 83

  • •

    The 14th amendment of the German Medicines Act (Updated by the 15th amendment in July 2009)
  • •

    DIRECTIVE 2001/83/EC Article 5

  • •

    Regulations 1994 (SI 1994/3144)

  • •

    Regulations 2005 (SI 2005/2789)

  • •

    Regulations 2012 (SI 2012/1916)
Criteria
  • 1)

    For the treatment of serious or rare diseases.

  • 2)

    In the absence of a suitable therapeutic alternative (with a Marketing Authorization) available in France.

  • 3)

    When there is presumed to be a positive benefit/risk.
  • 1)

    For the treatment of serious or rare diseases.

  • 2)

    In the absence of a suitable therapeutic alternative (with a Marketing Authorization) available in France.

  • 3)

    When there is presumed to be a positive benefit/risk.
  • 1)

    Not for single patient.

  • 2)

    Not licensed in member state of the EU/EEA.

  • 3)

    Serious debilitating or life-threatening disease.

  • 4)

    The patients are not satisfactorily treated by a medicinal product authorized in Germany.

  • 5)

    Application either at EMA or any other Member State of EU/EEA.
  • 1)

    In response to an unsolicited order.

  • 2)

    Manufactured and assembled in accordance with the specification of a person who is a doctor, dentist, nurse independent prescriber, pharmacist independent prescriber or supplementary prescriber.

  • 3)

    For use by a patient for whose treatment that person is directly responsible in order to fulfil the special needs of that patient; and meets the conditions specified in regulation 167(2)-(8).
Population Individual patient Group Group Individual patient
When can it be used Efficacy/safety ratio presumed to be favourable for these patients based on available data. Efficacy and safety are strongly presumed. Not described Not described
Prior regulator approval Required Required Required Required
Charge Country health care system or donation by company. Country health care system or donation by company. Free of charge Country (the National Health Service in Part VIIIB of Drug Tariff).
Examples of products MYLOTARG (gemtuzumab ozogamicin) LENVIMA (Lenvatinib) Zanamivir i.v (Zanamavir) Modafinil (as an example of “Specials”)

The EU: The European Union, EEA: The European Economic Area, EMA: European Medicines Agency, ANSM: French National Agency for Medicines and Health Products Safety, BfArM: The Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte), UK: United Kingdom, MHRA: The Medicines and Healthcare Products Regulatory Agency, ATU: Authorisation Temporaire d' Utilisation (Temporary Authorizations for Use).

France source: Notice to applicants for Temporary Authorisation for Use (ATU). ANSM 2015. http://ansm.sante.fr/var/ansm_site/storage/original/application/cadfbcf9594614d59c8915670853a28b.pdf.

Temporary Authorisations for Use (ATU): http://agence-tst.ansm.sante.fr/html/pdf/5/atu_eng.pdf.