Cargando…

Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network

BACKGROUND: The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this...

Descripción completa

Detalles Bibliográficos
Autores principales:	Heard, Jean-Michel, Vrinten, Charlotte, Schlander, Michael, Bellettato, Cinzia Maria, van Lingen, Corine, Scarpa, Maurizio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2020
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6945588/ https://www.ncbi.nlm.nih.gov/pubmed/31907071 http://dx.doi.org/10.1186/s13023-019-1280-5

Ejemplares similares

Research activity and capability in the European reference network MetabERN
por: Heard, Jean-Michel, et al.
Publicado: (2019)

Social and medical needs of rare metabolic patients: results from a MetabERN survey
por: Sestini, Sylvia, et al.
Publicado: (2021)

The impact of COVID-19 on rare metabolic patients and healthcare providers: results from two MetabERN surveys
por: Lampe, C., et al.
Publicado: (2020)

One year of COVID-19: infection rates and symptoms in patients with inherited metabolic diseases followed by MetabERN
por: Paneghetti, Laura, et al.
Publicado: (2022)

The European Medicines Agency's approval of new medicines for type 2 diabetes
por: Blind, Eberhard, et al.
Publicado: (2018)

Biomarker and Companion Diagnostics—A Review of Medicinal Products Approved by the European Medicines Agency
por: Orellana García, Laura Patricia, et al.
Publicado: (2021)

No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products - a pilot study
por: Stolk, Pieter, et al.
Publicado: (2009)

Extension of the European Medicines Agency (EMA) approval of trifluridine/tipiracil for gastric cancer
por: Alsina, Maria, et al.
Publicado: (2019)

Predictors of orphan drug approval
por: Heemstra, Harald
Publicado: (2010)

Post‐Marketing Requirements for Cancer Drugs Approved by the European Medicines Agency, 2004–2014
por: Cherla, Avi, et al.
Publicado: (2022)

Patient-Reported Outcome Measures in Oncology Drugs Approved by the European Medicines Agency, 2017-2021
por: Ciani, Oriana, et al.
Publicado: (2023)

Possible strategies to cross the blood–brain barrier
por: Bellettato, Cinzia M., et al.
Publicado: (2018)

HTA Agencies Need Evidence-Informed Deliberative Processes Comment on "Use of Evidence-Informed Deliberative Processes by Health Technology Assessment Agencies Around the Globe"
por: Schlander, Michael
Publicado: (2020)

Barriers towards the publication of academic drug trials. Follow-up of trials approved by the Danish Medicines Agency
por: Berendt, Louise, et al.
Publicado: (2017)

European Medicines Agency approval summary: Zaltrap for the treatment of patients with oxaliplatin-resistant metastatic colorectal cancer
por: Stanel, Stefan Cristian, et al.
Publicado: (2017)

Regulators' Advice Can Make a Difference: European Medicines Agency Approval of Zynteglo for Beta Thalassemia
por: Schuessler‐Lenz, Martina, et al.
Publicado: (2019)

Patient-reported outcome measures in drugs for neurological conditions approved by European Medicines Agency 2017–2022
por: Ciani, Oriana, et al.
Publicado: (2023)

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)

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

Investigating the landscape of US orphan product approvals
por: Miller, Kathleen L., et al.
Publicado: (2018)

Lifecycle management of orphan drugs approved in Japan
por: Seki, Kiyoshi, et al.
Publicado: (2022)

Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study
por: Boyle, Jemma M., et al.
Publicado: (2021)

Availability and Affordability of Drugs With a Conditional Approval by the European Medicines Agency; Comparison of Korea With Other Countries and the Implications
por: Kwon, Hye-Young, et al.
Publicado: (2018)

Comparison of COVID-19 Vaccine Approvals at the US Food and Drug Administration, European Medicines Agency, and Health Canada
por: Lythgoe, Mark P., et al.
Publicado: (2021)

Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment
por: Malinowski, Krzysztof Piotr, et al.
Publicado: (2018)

Pharmacovigilance and the Italian Medicines Agency
por: Pimpinella, Giuseppe, et al.
Publicado: (2013)

Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13
por: Davis, Courtney, et al.
Publicado: (2017)

Hereditary Angioedema: The Economics of Treatment of an Orphan Disease
por: Lumry, William Raymond
Publicado: (2018)

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

Comparing access to orphan medicinal products in Europe
por: Zamora, Bernarda, et al.
Publicado: (2019)

Precision Medicines’ Impact on Orphan Drug Designation
por: Mueller, Christine M., et al.
Publicado: (2019)

An analysis of orphan medicine expenditure in Europe: is it sustainable?
por: Mestre-Ferrandiz, Jorge, et al.
Publicado: (2019)

The evidence base for psychotropic drugs approved by the European Medicines Agency: a meta-assessment of all European Public Assessment Reports
por: Erhel, Florian, et al.
Publicado: (2020)

Licensing of Orphan Medicinal Products—Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021
por: Naumann-Winter, Frauke, et al.
Publicado: (2022)

Leveraging existing opportunities for improved Orphan Drug approval in the EU
por: Edfjäll, Catarina
Publicado: (2012)

Introduction to Endo-ERN–scope and mission
por: Pereira, Alberto M., et al.
Publicado: (2021)

75(th) Meeting of the Ulster Society of Internal Medicine, Friday 19(th) May 2006: Erne Hospital, Enniskillen
Publicado: (2006)

Public spending on orphan medicines: a review of the literature
por: Gombocz, Margit, et al.
Publicado: (2020)

Regulatory Standards in Orphan Medicinal Product Designation in the EU
por: Tsigkos, Stelios, et al.
Publicado: (2021)

Psychopharmacology at the era of EMEA (European Medicines Agency)
por: Pestana, Luis Camara, et al.
Publicado: (2010)

Cannot write session to /tmp/vufind_sessions/sess_30ckgkmdept6poorpcq1120akk