Cargando…

The influence of the European paediatric regulation on marketing authorisation of orphan drugs for children

BACKGROUND: Drug development for rare diseases is challenging, especially when these orphan drugs (OD) are intended for children. In 2007 the EU Paediatric Drug Regulation was enacted to improve the development of high quality and ethically researched medicines for children through the establishment...

Descripción completa

Detalles Bibliográficos
Autores principales:	Kreeftmeijer-Vegter, Annemarie Rosan, de Boer, Anthonius, van der Vlugt-Meijer, Roselinda H, de Vries, Peter J
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2014
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4237943/ https://www.ncbi.nlm.nih.gov/pubmed/25091201 http://dx.doi.org/10.1186/s13023-014-0120-x

Ejemplares similares

Roll out of intraveneous artesunate under named patient programmes in the Netherlands, Belgium and France
por: Kreeftmeijer-Vegter, Annemarie Rosan, et al.
Publicado: (2013)

New functionalities in Orphanet for orphan drugs, R&D and marketing authorisations to better serve the rare diseases community
por: Hivert, Virginie, et al.
Publicado: (2010)

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs
por: Giannuzzi, Viviana, et al.
Publicado: (2017)

Mobility endpoints in marketing authorisation of drugs: what gets the European medicines agency moving?
por: Jaeger, Simon U, et al.
Publicado: (2022)

Extension of Indication for Authorised Oncology Products in the European Union: A Joint Effort of Multiple Stakeholders
por: Mulder, Jorn, et al.
Publicado: (2021)

The European Medicines Agency's EU conditional marketing authorisations for COVID-19 vaccines
por: Cavaleri, Marco, et al.
Publicado: (2021)

Orphans in the Market: The History of Orphan Drug Policy
por: Mikami, Koichi
Publicado: (2019)

Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
por: Iglesias-Lopez, Carolina, et al.
Publicado: (2021)

Adaptive designs in clinical trials: from scientific advice to marketing authorisation to the European Medicine Agency
por: Collignon, Olivier, et al.
Publicado: (2018)

Manual blood exchange transfusion does not significantly contribute to parasite clearance in artesunate-treated individuals with imported severe Plasmodium falciparum malaria
por: Kreeftmeijer-Vegter, Annemarie R, et al.
Publicado: (2013)

Registries for orphan drugs: generating evidence or marketing tools?
por: Hollak, Carla E. M., et al.
Publicado: (2020)

Marketing authorisation and pricing of FDA-approved cancer drugs in Brazil: a retrospective analysis
por: Ivama-Brummell, Adriana M., et al.
Publicado: (2023)

Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation
por: Mulder, Jorn, et al.
Publicado: (2021)

Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties
por: Pontes, Caridad, et al.
Publicado: (2018)

Impact of Amending the Acetylcysteine Marketing Authorisation on Treatment of Paracetamol Overdose
por: Thompson, G., et al.
Publicado: (2013)

An analysis of marketing authorisation applications via the mutual recognition and decentralised procedures in Europe
por: Ebbers, Hans C., et al.
Publicado: (2015)

Treatment outcome of intravenous artesunate in patients with severe malaria in the Netherlands and Belgium
por: Kreeftmeijer-Vegter, Annemarie R, et al.
Publicado: (2012)

Predictors of orphan drug approval in the European Union
por: Heemstra, Harald E., et al.
Publicado: (2008)

Experiences and Perspectives of Marketing Authorisation Holders towards Medication Safety Monitoring during Pregnancy: A Pan-European Qualitative Analysis
por: Sillis, Laure, et al.
Publicado: (2022)

Disentangling the Cost of Orphan Drugs Marketed in the United States
por: Althobaiti, Hana, et al.
Publicado: (2023)

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation
por: Detela, Giulia, et al.
Publicado: (2019)

GANIL authorised
Publicado: (1975)

Description of the Risk Management of Medication Errors for Centrally Authorised Products in the European Union
por: Hoeve, Christina E., et al.
Publicado: (2019)

Prices of Orphan Drugs in Four Western European Countries Before and After Market Exclusivity Expiry: A Cross-Country Comparison of List Prices and Purchase Prices
por: Dane, Aniek, et al.
Publicado: (2023)

Market access of orphan drugs and the role of multi-criteria decision making
por: Simoens, Steven
Publicado: (2012)

Additional Risk Minimisation Measures for Medicinal Products in the European Union: A Review of the Implementation and Effectiveness of Measures in the United Kingdom by One Marketing Authorisation Holder
por: Agyemang, Elaine, et al.
Publicado: (2017)

The pre‐ and post‐authorisation data published by the European medicines agency on the use of biologics during pregnancy and lactation
por: Ghalandari, N., et al.
Publicado: (2020)

Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries
por: Stawowczyk, Ewa, et al.
Publicado: (2019)

Authorisation for work with electrical equipment
por: Balle, Christoph
Publicado: (2013)

National Association of Authorised Officers
por: Weston, W. H. A.
Publicado: (1952)

Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia: rationale and description
por: Brandi, Maria Luisa, et al.
Publicado: (2022)

Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval
por: Harada, Kenji, et al.
Publicado: (2019)

Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments
por: Winstone, Julie, et al.
Publicado: (2015)

Guidance on the renewal of the authorisation of feed additives
por: Bampidis, Vasileios, et al.
Publicado: (2021)

The European challenges of funding orphan medicinal products
por: Szegedi, Márta, et al.
Publicado: (2018)

Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?
por: Brabers, Anne EM, et al.
Publicado: (2011)

Orphan devices: yesterday is history; tomorrow is mystery: towards a European orphan device directive?
por: Dooms, Marc M
Publicado: (2016)

The work of the European Union Reference Laboratory for Food Additives (EURL) and its support for the authorisation process of feed additives in the European Union: a review
por: von Holst, Christoph, et al.
Publicado: (2016)

Clinical Trial Authorisation: A Final Look Back to Better Appraise the New European Regulation
por: Vignot, Stéphane, et al.
Publicado: (2023)

Orphan Crops Browser: a bridge between model and orphan crops
por: Kamei, Claire Lessa Alvim, et al.
Publicado: (2016)

Cannot write session to /tmp/vufind_sessions/sess_uo3vukcf77can2k4pbofe435g5