Literature DB >> 20824461

National plans and strategies on rare diseases in Europe.

Domenica Taruscio1, Luciano Vittozzi, Rumen Stefanov.   

Abstract

This analysis of national plans and strategies on RD in Europe shows that a few countries have already set up national plans. Existing national plans show a good consistency, but also a quite different stage of progress, depending on start date as well as on resource allocation. Several other EU countries have launched actions on RD, often with a considerable strategic effort; however, such initiatives are yet not integrated in a consistent national strategy taking into account the EC recommendations. The project EUROPLAN represents a major initiative to support the development of a shared strategy on RD at EU and Member State level; critical steps include the comparative evaluation of existing plans and actions, identification of gaps and achievements, the development of consensus indicators, as well as the integration of successful national achievements within the EU strategy.

Mesh:

Year:  2010        PMID: 20824461     DOI: 10.1007/978-90-481-9485-8_26

Source DB:  PubMed          Journal:  Adv Exp Med Biol        ISSN: 0065-2598            Impact factor:   2.622


  8 in total

Review 1.  The Italian National Centre for Rare Diseases: where research and public health translate into action.

Authors:  Domenica Taruscio; Linda Agresta; Annalisa Amato; Giuseppe Bernardo; Luana Bernardo; Francesca Braguti; Pietro Carbone; Claudio Carta; Marina Ceccarini; Federica Censi; Simona Coppola; Patrizia Crialese; Marta De Santis; Stefano Diemoz; Carlo Donati; Sabina Gainotti; Gianluca Ferrari; Giovanna Floridia; Claudio Frank; Rosa Giuseppa Frazzica; Amalia E Gentile; Orietta Granata; Yllka Kodra; Manuela Latrofa; Paola Laricchiuta; Armando Magrelli; Cristina Morciano; Agata Polizzi; Stefania Razeto; Marco Salvatore; Antonella Sanseverino; Daniele Savini; Paola Torreri; Fabrizio Tosto; Flavia Villani; Giorgio Vincenti; Luciano Vittozzi
Journal:  Blood Transfus       Date:  2014-04       Impact factor: 3.443

2.  The need for a next-generation public health response to rare diseases.

Authors:  Rodolfo Valdez; Scott D Grosse; Muin J Khoury
Journal:  Genet Med       Date:  2016-10-27       Impact factor: 8.822

3.  The health and economic burden of haemophilia in Belgium: a rare, expensive and challenging disease.

Authors:  Séverine Henrard; Brecht Devleesschauwer; Philippe Beutels; Michael Callens; Frank De Smet; Cedric Hermans; Niko Speybroeck
Journal:  Orphanet J Rare Dis       Date:  2014-03-21       Impact factor: 4.123

4.  Diagnostic Efficacy and Therapeutic Decision-making Capacity of an Artificial Intelligence Platform for Childhood Cataracts in Eye Clinics: A Multicentre Randomized Controlled Trial.

Authors:  Haotian Lin; Ruiyang Li; Zhenzhen Liu; Jingjing Chen; Yahan Yang; Hui Chen; Zhuoling Lin; Weiyi Lai; Erping Long; Xiaohang Wu; Duoru Lin; Yi Zhu; Chuan Chen; Dongxuan Wu; Tongyong Yu; Qianzhong Cao; Xiaoyan Li; Jing Li; Wangting Li; Jinghui Wang; Mingmin Yang; Huiling Hu; Li Zhang; Yang Yu; Xuelan Chen; Jianmin Hu; Ke Zhu; Shuhong Jiang; Yalin Huang; Gang Tan; Jialing Huang; Xiaoming Lin; Xinyu Zhang; Lixia Luo; Yuhua Liu; Xialin Liu; Bing Cheng; Danying Zheng; Mingxing Wu; Weirong Chen; Yizhi Liu
Journal:  EClinicalMedicine       Date:  2019-03-17

5.  Red urine and a red herring - diagnosing rare diseases in the light of the COVID-19 pandemic.

Authors:  Philipp Jud; Gerald Hackl; Alexander Christian Reisinger; Angela Horvath; Philipp Eller; Vanessa Stadlbauer
Journal:  Z Gastroenterol       Date:  2021-11-12       Impact factor: 1.769

6.  Key outcomes from stakeholder workshops at a symposium to inform the development of an Australian national plan for rare diseases.

Authors:  Caron Molster; Leanne Youngs; Emma Hammond; Hugh Dawkins
Journal:  Orphanet J Rare Dis       Date:  2012-08-10       Impact factor: 4.123

7.  Rare diseases: Canada's "research orphans".

Authors:  Samir Gupta
Journal:  Open Med       Date:  2012-02-28

8.  Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation.

Authors:  Monika Wagner; Hanane Khoury; Jacob Willet; Donna Rindress; Mireille Goetghebeur
Journal:  Pharmacoeconomics       Date:  2016-03       Impact factor: 4.981

  8 in total

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