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Eur J Cancer. 2017 Oct;84:149-158. doi: 10.1016/j.ejca.2017.07.021. Epub 2017 Sep 4.

Orphan Drug Regulation: A missed opportunity for children and adolescents with cancer.

Author information

Department of Clinical Research, Gustave Roussy, Paris-Sud University, Paris, France; Innovative Therapy for Children with Cancer, Villejuif, France. Electronic address:
Innovative Therapy for Children with Cancer, Villejuif, France; Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK.
Imagine for Margo, 9 Avenue Eric Tabarly, 78112 Fourqueux, France.
Zoé4life, 1036 Sullens, Switzerland.
KickCancer, 24 Rue de L'Aurore, 1000 Bruxelles, Belgium.
Innovative Therapy for Children with Cancer, Villejuif, France; Paediatric Drug Development, Children and Young People's Unit, The Royal Marsden NHS Foundation Trust, Sutton, SM2 5PT, UK; Division of Clinical Studies and Cancer Therapeutics, The Institute of Cancer Research, Sutton, SM2 5NG, UK.



Oncology represents a major sector in the field of orphan drug development in Europe. The objective was to evaluate whether children and adolescents with cancer benefited from the Orphan Drug Regulation.


Data on orphan drug designations (ODDs) and registered orphan drugs from 8th August 2000 to 10th September 2016 were collected from the Community Register of medicinal products for human use. Assessment history, product information and existence of paediatric investigation plans were searched and retrieved from the European Medicine Agency website.


Over 16 years, 272 of 657 oncology ODDs (41%) concerned a malignant condition occurring both in adults and children. The five most common were acute myeloid leukaemia, high-grade glioma, acute lymphoblastic leukaemia, graft-versus-host disease and soft-tissue sarcomas. 74% of 31 marketing authorisations (MAs) for an indication both in adults and children (26 medicines) had no information for paediatric use in their Summary of Product Characteristics (SmPC) at the time of the first MA. Furthermore, 68% still have no paediatric information in their most recently updated SmPC, at a median of 7 years after. Only 15 ODDs (2%) pertained to a malignancy occurring specifically in children and only two drugs received an MA: Unituxin for high-risk neuroblastoma and Votubia for sub-ependymal giant-cell astrocytoma.


The Orphan Drug Regulation failed to promote the development of innovative therapies for malignancies occurring in children. Major delays and waivers occurred through the application of the Paediatric Medicines Regulation. The European regulatory environment needs to be improved to accelerate innovation for children and adolescents dying of cancer.


Childhood cancer; New drugs; Orphan

[Indexed for MEDLINE]

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