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Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe

Spencer, R; Rossi, C; Lees, M; Peebles, D; Brocklehurst, P; Martin, J; Hansson, SR; ... EVERREST Consortium, .; + view all (2019) Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe. BJOG: An International Journal of Obstetrics and Gynaecology , 126 (9) pp. 1157-1167. 10.1111/1471-0528.15590. Green open access

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Abstract

OBJECTIVE: To determine whether a novel therapy for placental insufficiency could achieve orphan drug status by estimating the annual incidence of placental insufficiency, defined as an estimated fetal weight below the 10th centile in the presence of abnormal umbilical artery Doppler velocimetry, per 10,000 European Union (EU) population as part of an application for European Medicines Agency (EMA) orphan designation. DESIGN: Incidence estimation based on literature review and published national and EU statistics. SETTING AND POPULATION: European Union. METHODS: Data were drawn from published literature, including national and international guidelines, international consensus statements, cohort studies and randomised controlled trials, and published national and EU statistics, including birth rates and stillbirth rates. Rare disease databases were also searched. RESULTS: The proportion of affected pregnancies was estimated as 3.17% (95% CI 2.93% to 3.43%), using a weighted average of the results from two cohort studies. Using birth rates from 2012 and adjusting for a pregnancy loss rate of 1/100 gave an estimated annual incidence of 3.33 per 10,000 EU population (95% CI 3.07 to 3.60 per 10,000 EU population). This fell below the EMA threshold of 5 per 10,000 EU population. CONCLUSIONS: Maternal vascular endothelial growth factor gene therapy for placental insufficiency was granted EMA orphan status in 2015 after we demonstrated that it is a rare, life-threatening or chronically debilitating and currently untreatable disease. Developers of other potential obstetric therapies should consider applying for orphan designation, which provides financial and regulatory benefits. This article is protected by copyright. All rights reserved.

Type: Article
Title: Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/1471-0528.15590
Publisher version: https://doi.org/10.1111/1471-0528.15590
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: European Medicines Agency, Orphan disease, fetal growth restriction, incidence, placental insufficiency, pregnancy, prevalence
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Experimental and Translational Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL EGA Institute for Womens Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL EGA Institute for Womens Health > Maternal and Fetal Medicine
URI: https://discovery.ucl.ac.uk/id/eprint/10064799
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