Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations

Author:

Vreman Rick A.,de Ruijter Angela S.,Zawada Anna,Tafuri Giovanni,Stoyanova-Beninska Violeta,O’Connor Daniel,Naumann-Winter Frauke,Wolter Franziska,Mantel-Teeuwisse Aukje K.,Leufkens Hubert G.M.,Sidiropoulos Iordanis,Larsson Kristina,Goettsch Wim G.

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology

Reference67 articles.

1. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products;Anon;Off. J. Eur. Union,1999

2. Inventory of Union and Member State incentives to support research into, and the development and availability of orphan medicinal products. State of Play 2015, European Commission;Anon,2016

3. Post-orphan Medicinal Product Designation Procedures. Guidance for Sponsors, EMA;EMA,2019

4. Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe;Fregonese;Drug Discov. Today,2018

5. European regulation on orphan medicinal products: 10 years of experience and future perspectives;Committee for Orphan Medicinal Products and the European Medicines;Nat. Rev. Drug Discov.,2011

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