Literature DB >> 27245390

Regulatory watch: The orphan drug pipeline in Europe.

Thomas Morel1, André Lhoir2, Eline Picavet1, Segundo Mariz3, Bruno Sepodes4, Jordi Llinares3, David Cassiman5.   

Abstract

Mesh：

Year:  2016        PMID： 27245390     DOI： 10.1038/nrd.2016.96

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN： 1474-1776            Impact factor:   84.694


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  10 in total

1.  Funding orphan medicinal products beyond price: sustaining an ecosystem.

Authors:  Oriol de Sola-Morales
Journal:  Eur J Health Econ       Date:  2019-12

Review 2.  From promising molecules to orphan drugs: Early clinical drug development.

Authors:  Marc Dooms
Journal:  Intractable Rare Dis Res       Date:  2017-02

Review 3.  Current Drug Repurposing Strategies for Rare Neurodegenerative Disorders.

Authors:  Sweta Shah; Marc Marie Dooms; Sofia Amaral-Garcia; Mariana Igoillo-Esteve
Journal:  Front Pharmacol       Date:  2021-12-21       Impact factor: 5.810

4.  Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases.

Authors:  Kathleen L Miller; Lewis J Fermaglich; Janet Maynard
Journal:  Orphanet J Rare Dis       Date:  2021-06-09       Impact factor: 4.123

Review 5.  Future of Rare Diseases Research 2017-2027: An IRDiRC Perspective.

Authors:  Christopher P Austin; Christine M Cutillo; Lilian P L Lau; Anneliene H Jonker; Ana Rath; Daria Julkowska; David Thomson; Sharon F Terry; Béatrice de Montleau; Diego Ardigò; Virginie Hivert; Kym M Boycott; Gareth Baynam; Petra Kaufmann; Domenica Taruscio; Hanns Lochmüller; Makoto Suematsu; Carlo Incerti; Ruxandra Draghia-Akli; Irene Norstedt; Lu Wang; Hugh J S Dawkins
Journal:  Clin Transl Sci       Date:  2017-10-23       Impact factor: 4.689

6.  Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012.

Authors:  Kim Pauwels; Isabelle Huys; Minne Casteels; Kristina Larsson; Caroline Voltz; Karri Penttila; Thomas Morel; Steven Simoens
Journal:  Orphanet J Rare Dis       Date:  2017-02-16       Impact factor: 4.123

Review 7.  Measuring what matters to rare disease patients - reflections on the work by the IRDiRC taskforce on patient-centered outcome measures.

Authors:  Thomas Morel; Stefan J Cano
Journal:  Orphanet J Rare Dis       Date:  2017-11-02       Impact factor: 4.123

8.  Global Development of Anticancer Therapies for Rare Cancers, Pediatric Cancers, and Molecular Subtypes of Common Cancers.

Authors:  H Kim Lyerly; Jun Ren; Renzo Canetta; Gi Hyun Kim; Sumimasa Nagai; Tomohiro Yamaguchi; Ken Hatogai; Hiroshi Katayama; Silvy Da Rocha Dias; Daniel McManus; Kathy Soltys; Zhimin Yang; Olufumilayo Olopade; Nancy Goodman; Greg Reaman; Thomas Gross
Journal:  J Glob Oncol       Date:  2018-12

9.  Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs.

Authors:  Viviana Giannuzzi; Annalisa Landi; Enrico Bosone; Floriana Giannuzzi; Stefano Nicotri; Josep Torrent-Farnell; Fedele Bonifazi; Mariagrazia Felisi; Donato Bonifazi; Adriana Ceci
Journal:  BMJ Open       Date:  2017-09-11       Impact factor: 2.692

10.  Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe.

Authors:  Andreas M Farkas; Segundo Mariz; Violeta Stoyanova-Beninska; Patrick Celis; Spiros Vamvakas; Kristina Larsson; Bruno Sepodes
Journal:  Front Med (Lausanne)       Date:  2017-05-16
  10 in total

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