# The current revision of the orphan medicines regulation in the EU: what is at stake for gene and cell therapy?

Aurélie Mahalatchimy, Éloïse Gennet

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Aurélie Mahalatchimy, Éloïse Gennet. The current revision of the orphan medicines regulation in the EU: what is at stake for gene and cell therapy?. European Society for Gene & Cell Therapy Congress 2022, Oct 2022, Edinburgh, United Kingdom. . halshs-03901429

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Submitted on 15 Dec 2022

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# The current revision of the orphan medicines regulation in the EU: what is at stake for gene and cell therapy?

Aurélie Mahalatchimy<fnref n="1" /> and Éloïse Gennet²

**Acknowledgement:** This work has been supported by ANR-funded I-BioLex project (ANR-20-CE26-0007-01, coord. A. Mahalatchimy).

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## REFERENCES

* Regulation 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products, OJ L 18, 22.1.2000, p. 1–5.
* Regulation 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation No 726/2004, OJ L 324, 10.12.2007, p. 121–137
* European Commission, Joint evaluation of Regulation (EC) No 1901/2006 and

* Regulation No 141/2000, Brussels, 11.8.2020, SWD(2020) 163 final
* European Parliament resolution of 2 March 2017 on EU options for improving access to medicines (2016/2057(INI))

* European Commission, "Rare diseases. A major unmet medical need", 2017.
* Communication from the Commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions

* European Parliament resolution of 24 November 2021 on a pharmaceutical strategy for Europe (2021/2013(INI))
* European Commission, Revision of the Union legislation on medicines for children and rare diseases - Public consultation factual summary report, 4 November 2021.

* European Commission, Revision of the EU general pharmaceutical legislation - Public consultation factual summary report, 10 February 2022.

* Pharmaceutical strategy for Europe, COM/2020/761 final

<footnote n="1">¹Permanent Researcher in law at the French National Centre for Scientific Research (CNRS, CR), Law Faculty, UMR 7318 DICE CERIC, CNRS, Aix Marseille University, Toulon University, Aix-en-Provence, France; ²Post-doctoral Researcher in Law, Law Faculty, UMR 7318 International, Comparative and European laws (DICE) CERIC, CNRS, Aix- Marseille University, Toulon University, Aix-en-Provence, France.</footnote>