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Defining orphan conditions in the context of the European orphan regulation: challenges and evolution

Nature Reviews Drug Discovery volume 18, pages 479–480 (2019)Cite this article

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The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for Orphan Medicinal Products, we discuss how to define orphan conditions in the context of the European regulatory framework.

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References

  1. European Commission. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products (Official Journal of the European Communities, 1999).

  2. European Commission. Commission Notice on the Application of Articles 3,5 and 7 of Regulation (EC) No 141/2000 on Orphan Medicinal Products (EC, 2016).

  3. European Commission. Guideline on the Format and Content of Applications for Designation as Orphan Medicinal Products and on the Transfer of Designations from One Sponsor to Another, 27.03.2014 (EC, 2014).

  4. Ayme, S. et al. Rare diseases in ICD11: making rare diseases visible in health information systems through appropriate coding. Orphanet J. Rare Dis. 10, 35 (2015).

  5. Hunter, N. L., Rao, R. G. & Sherman, R. E. Flexibility in the FDA approach to orphan drug development. Nat. Rev. Drug Discov. 16, 737–738 (2017).

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Author notes

  1. These authors contributed equally: Daniel J. O’Connor, Maria E. Sheean, Matthias P. Hofer

Authors and Affiliations

  1. MHRA, London, UK

    Daniel J. O’Connor

  2. EMA, London, UK

    Maria E. Sheean, Matthias P. Hofer, Stelios Tsigkos, Segundo Mariz, Laura Fregonese & Kristina Larsson

  3. MDC, Berlin, Germany

    Maria E. Sheean

  4. EURORDIS-Rare Diseases, Europe, France

    Virginie Hivert

  5. Former MPA, Uppsala, Sweden

    Kerstin Westermark

  6. BfArM, Bonn, Germany

    Frauke Naumann-Winter

  7. MEB, Utrecht, Netherlands

    Violeta Stoyanova-Beninska

  8. Children’s University Hospital Zagreb, Zagreb, Croatia

    Ingeborg Barišić

  9. C. Poma Hospital, Mantova, Italy

    Giuseppe Capovilla

  10. Istituto Superiore di Sanità, Rome, Italy

    Armando Magrelli

  11. University of Lisbon, Lisbon, Portugal

    Bruno Sepodes

Authors
  1. Daniel J. O’Connor
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  2. Maria E. Sheean
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  3. Matthias P. Hofer
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  4. Stelios Tsigkos
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  5. Segundo Mariz
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  6. Laura Fregonese
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  7. Kristina Larsson
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  8. Virginie Hivert
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  9. Kerstin Westermark
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  10. Frauke Naumann-Winter
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  11. Violeta Stoyanova-Beninska
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  12. Ingeborg Barišić
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  13. Giuseppe Capovilla
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  14. Armando Magrelli
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  15. Bruno Sepodes
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Corresponding author

Correspondence to Daniel J. O’Connor.

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Competing interests

The authors declare no competing interests.

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Disclaimer

The views expressed in this article are the personal views of the author(s) and may not be understood or quoted as being made on behalf of or reflecting the position of the European Medicines Agency or one of its committees or working parties.

Supplementary information

Supplementary Figure

Flow chart for designation of an orphan condition in the European Union

Supplementary Table

Minutes of COMP discussions on accepted and unaccepted orphan conditions

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O’Connor, D.J., Sheean, M.E., Hofer, M.P. et al. Defining orphan conditions in the context of the European orphan regulation: challenges and evolution. Nat Rev Drug Discov 18, 479–480 (2019). https://doi.org/10.1038/nrd.2018.128

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