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

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, pe...

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Autores: Spencer R, Rossi C, Lees M, Peebles D, Brocklehurst P, Martin J, Hansson SR, Hecher K, Marsal K, Figueras F, Gratacos E, David AL, EVERREST Consortium
Tipo de documento: artigo
Estado:Versão publicada
Data de publicação:2019
País:España
Recursos:Fundació Sant Joan de Déu
Repositório:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
OAI Identifier:oai:fsjd.fundanetsuite.com:p15488
Acesso em linha:https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=15488
Access Level:Acceso aberto
Palavra-chave:European Medicines Agency
fetal growth restriction
incidence
orphan disease
placental insufficiency
pregnancy
prevalence
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repository_id_str
spelling Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.Spencer RRossi CLees MPeebles DBrocklehurst PMartin JHansson SRHecher KMarsal KFigueras FGratacos EDavid ALEVERREST ConsortiumEuropean Medicines Agencyfetal growth restrictionincidenceorphan diseaseplacental insufficiencypregnancyprevalenceOBJECTIVE: 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 2.93-3.43%), 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 3.07-3.60 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. TWEETABLE ABSTRACT: Placental insufficiency meets the European Medicines Agency requirements for orphan disease designation.WILEY2019info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttps://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=15488BJOG-AN INTERNATIONAL JOURNAL OF OBSTETRICS AND GYNAECOLOGYISSN: 14700328ISSNe: 14710528reponame:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déuinstname:Fundació Sant Joan de DéuInglésinfo:eu-repo/semantics/openAccessoai:fsjd.fundanetsuite.com:p154882026-05-27T12:37:41Z
dc.title.none.fl_str_mv Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
title Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
spellingShingle Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
Spencer R
European Medicines Agency
fetal growth restriction
incidence
orphan disease
placental insufficiency
pregnancy
prevalence
title_short Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
title_full Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
title_fullStr Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
title_full_unstemmed Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
title_sort Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe.
dc.creator.none.fl_str_mv Spencer R
Rossi C
Lees M
Peebles D
Brocklehurst P
Martin J
Hansson SR
Hecher K
Marsal K
Figueras F
Gratacos E
David AL
EVERREST Consortium
author Spencer R
author_facet Spencer R
Rossi C
Lees M
Peebles D
Brocklehurst P
Martin J
Hansson SR
Hecher K
Marsal K
Figueras F
Gratacos E
David AL
EVERREST Consortium
author_role author
author2 Rossi C
Lees M
Peebles D
Brocklehurst P
Martin J
Hansson SR
Hecher K
Marsal K
Figueras F
Gratacos E
David AL
EVERREST Consortium
author2_role author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv European Medicines Agency
fetal growth restriction
incidence
orphan disease
placental insufficiency
pregnancy
prevalence
topic European Medicines Agency
fetal growth restriction
incidence
orphan disease
placental insufficiency
pregnancy
prevalence
description 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 2.93-3.43%), 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 3.07-3.60 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. TWEETABLE ABSTRACT: Placental insufficiency meets the European Medicines Agency requirements for orphan disease designation.
publishDate 2019
dc.date.none.fl_str_mv 2019
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=15488
url https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=15488
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv WILEY
publisher.none.fl_str_mv WILEY
dc.source.none.fl_str_mv BJOG-AN INTERNATIONAL JOURNAL OF OBSTETRICS AND GYNAECOLOGY
ISSN: 14700328
ISSNe: 14710528
reponame:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname:Fundació Sant Joan de Déu
instname_str Fundació Sant Joan de Déu
reponame_str r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
collection r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
repository.name.fl_str_mv
repository.mail.fl_str_mv
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