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Worldwide collaboration for orphan drug designation

Nature Reviews Drug Discovery volume 15, pages 440–441 (2016)Cite this article

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A Corrigendum to this article was published on 15 July 2016

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The growing awareness of the need for pharmaceutical products to treat rare diseases led to the enactment of rare diseases legislation in the United States (Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat. Rev. Drug Discov. 9, 510–522 (2010))1, the European Union2 and Japan3 (see also Further information). The European Medicines Agency (EMA) has been engaged in collaborations with the US Food and Drug Administration (FDA)'s Office of Orphan Products Development (OOPD) since 2000 and with the Japanese Ministry of Health, Labour and Welfare (MHLW) and Pharmaceuticals and Medical Devices Agency (PMDA) Orphan Drug Designation Service since 2010. Identifying areas of similarity among these programmes has led to activities aimed at reducing redundancies and the administrative load for sponsors interested in submitting applications for orphan drug designation in each region, allowing for transparency and an increased sponsor understanding of each agency's processes and incentives (Table 1). It is hoped that there will be an increase in awareness of the benefits of parallel submission both at the orphan designation stage and post-designation, where input through protocol assistance (scientific advice) and paediatric input are subject to incentives. The reduction in regulatory consultation times and improved efficiencies in drug development due to these collaborative efforts should translate into accelerated timelines for the approval of successful orphan drugs.

Table 1 Comparison of incentives for products that are granted orphan drug designation
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In 2007, a joint application form for orphan drug submissions in the United States and the European Union was introduced, reducing the sponsor's administrative burden and encouraging parallel submissions to both agencies. The agencies have worked together to understand areas of similarity at the time of submission for an orphan drug designation, enhancing the understanding of key assessment criteria, which are similar in both systems. The OOPD Drug Designation Program carries out assessments for the FDA, whereas in Europe this is done by the Committee for Orphan Medicinal Products (COMP).

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Figure 1: Information in applications for orphan drug designation in the European Union from 2000 to 2014.

Change history

  • 15 July 2016

    The affiliations for Kerstin Westermark and Jordi Llinares-Garcia were listed incorrectly. These have been corrected in the online versions of the article.

References

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Acknowledgements

The views expressed in this article are the personal views of the authors 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), the US Food and Drug Administration or the Japanese Ministry of Health Labour and Welfare and the Pharmaceuticals and Medical Devices Agency of Japan.

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Authors and Affiliations

  1. European Medicines Agency, 30 Churchill Place, London, E14 5EU, Canary Wharf, UK

    Segundo Mariz, Lesley Greene & Bruno Sepodes

  2. Office of Orphan Products Development, US Food and Drug Administration, Silver Spring, 20993, Maryland, USA

    James H. Reese & Jordi Llinares-Garcia

  3. Medical Products Agency, Box 26, Uppsala, SE-75103, Sweden

    Kerstin Westermark

  4. European Organisation for Rare Diseases (Eurordis), 96 rue Didot, Paris, 75014, France

    Lesley Greene

  5. Evaluation and Licensing Division, Pharmaceutical and Food Safety Bureau (PFSB), Ministry of Health, Labour and Welfare (MHLW), 1-2-2 Kasumigaseki Chiyoda-ku, Tokyo, 100–8916, Japan

    Takahiro Goto & Tatsuro Hoshino

  6. Universidade de Lisboa, Faculdade de Farmácia, Lisboa, 1649–003, Portugal

    Bruno Sepodes

Authors
  1. Segundo Mariz
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  2. James H. Reese
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  3. Kerstin Westermark
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  7. Jordi Llinares-Garcia
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  8. Bruno Sepodes
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Correspondence to Bruno Sepodes.

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Mariz, S., Reese, J., Westermark, K. et al. Worldwide collaboration for orphan drug designation. Nat Rev Drug Discov 15, 440–441 (2016). https://doi.org/10.1038/nrd.2016.80

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