Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe-Reference-Cited by-同舟云学术

Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe

Published:2018-01 Issue:1 Volume:23 Page:90-100
ISSN:1359-6446
Container-title:Drug Discovery Today
language:en
Short-container-title:Drug Discovery Today

Author:

Fregonese Laura,Greene Lesley,Hofer Matthias,Magrelli Armando,Naumann-Winter Frauke,Larsson Kristina,Sheean Maria,Stoyanova-Beninska Violeta,Tsigkos Stelios,Westermark Kerstin,Sepodes Bruno

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology

Reference32 articles.

1. European Commission website: DG Health and Food Safety>Public health>Rare diseases>Policy. Available at: http://ec.europa.eu/health/rare_diseases/policy/index_en.htm.

2. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products. Available at: http://ec.europa.eu/health/files/eudralex/vol-1/reg_2000_141/reg_2000_141_en.pdf.

3. Post-orphan Medicinal Product Designation Procedures. Guidance for sponsors 24 January 2017. Available at: http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2015/11/WC500196994.pdf.

4. E-RARE Website: ERA-Net for Research Programmes on Rare Diseases. Available at: http://www.erare.eu/national-rd-funding. [Accessed 20 April 2017].

5. European Commission website: Research & Innovation>Health>Research>Areas>Rare disease. Available at: https://ec.europa.eu/research/health/index.cfm?pg=area&areaname=rare.

Cited by 16 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. A Double-Edged-Sword Approach to Fighting Pandemics: Patent Waivers and Incentives to Innovate;Review of European and Comparative Law;2023-09-30

2. Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases;BMJ;2023-05-09

3. Regulatory Considerations Toward Orphan Drug Designation and Orphan Drug Exclusivity in the United States and European Union: Structural Similarity, Clinical Superiority/Significant Benefit, and Case Studies;Therapeutic Innovation & Regulatory Science;2022-11-18

4. A Rational Approach to Drug Repositioning in β-thalassemia: Induction of Fetal Hemoglobin by Established Drugs;Wellcome Open Research;2022-08-17

5. 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;Frontiers in Pharmacology;2022-08-11