Literature DB >> 21532564

European regulation on orphan medicinal products: 10 years of experience and future perspectives.

Kerstin Westermark, Birthe Byskov Holm, Mirjam Söderholm, Jordi Llinares-Garcia, Frida Rivière, Stiina Aarum, Florence Butlen-Ducuing, Stelios Tsigkos, Agnieszka Wilk-Kachlicka, Cinzia N'Diamoi, János Borvendég, David Lyons, Bruno Sepodes, Brigitte Bloechl-Daum, André Lhoir, Mariana Todorova, Ioannis Kkolos, Kateřina Kubáčková, Heidrun Bosch-Traberg, Vallo Tillmann, Veijo Saano, Emmanuel Héron, Rembert Elbers, Miranda Siouti, Judit Eggenhofer, Patrick Salmon, Maurizio Clementi, Dainis Krieviņš, Aušra Matulevičiene, Henri Metz, Albert Cilia Vincenti, Albertha Voordouw, Bożenna Dembowska-Bagińska, Ana Corrêa Nunes, Flavia Mirela Saleh, Tatiana Foltánová, Martin Možina, Josep Torrent i Farnell, Björn Beerman, Segundo Mariz, Marie Pauline Evers, Lesley Greene, Sigurdur Thorsteinsson, Lars Gramstad, Maria Mavris, Fabrizia Bignami, Annie Lorence, Chantal Belorgey.   

Abstract

In 2000, regulation on orphan medicinal products was adopted in the European Union with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 orphan drug designations have been granted by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products (COMP), and more than 60 orphan drugs have received marketing authorization in Europe. Here, stimulated by the tenth anniversary of the COMP, we reflect on the outcomes and experience gained in the past decade, and contemplate issues for the future, such as catalysing drug development for the large number of rare diseases that still lack effective treatments.

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Year:  2011        PMID: 21532564     DOI: 10.1038/nrd3445

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


  12 in total

Review 1.  Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.

Authors:  M Miles Braun; Sheiren Farag-El-Massah; Kui Xu; Timothy R Coté
Journal:  Nat Rev Drug Discov       Date:  2010-06-07       Impact factor: 84.694

2.  Rare essentials: drugs for rare diseases as essential medicines.

Authors:  Pieter Stolk; Marjolein J C Willemen; Hubert G M Leufkens
Journal:  Bull World Health Organ       Date:  2006-09       Impact factor: 9.408

3.  Expediting patients' access to medicines by improving the predictability of drug development and the regulatory approval process.

Authors:  L Liberti; A Breckenridge; H G Eichler; R Peterson; N McAuslane; S Walker
Journal:  Clin Pharmacol Ther       Date:  2010-01       Impact factor: 6.875

Review 4.  Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma.

Authors:  Hans-Georg Eichler; Francesco Pignatti; Bruno Flamion; Hubert Leufkens; Alasdair Breckenridge
Journal:  Nat Rev Drug Discov       Date:  2008-09-12       Impact factor: 84.694

5.  Does orphan drug legislation really answer the needs of patients?

Authors:  Marlene E Haffner; Josep Torrent-Farnell; Paul D Maher
Journal:  Lancet       Date:  2008-06-14       Impact factor: 79.321

6.  Clinical trials of orphan medicines.

Authors:  Brendan M Buckley
Journal:  Lancet       Date:  2008-06-14       Impact factor: 79.321

7.  Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency.

Authors:  Jan Regnstrom; Franz Koenig; Bo Aronsson; Tatiana Reimer; Kristian Svendsen; Stelios Tsigkos; Bruno Flamion; Hans-Georg Eichler; Spiros Vamvakas
Journal:  Eur J Clin Pharmacol       Date:  2009-11-20       Impact factor: 2.953

Review 8.  Challenges with advanced therapy medicinal products and how to meet them.

Authors:  Christian K Schneider; Paula Salmikangas; Bernd Jilma; Bruno Flamion; Lyubina Racheva Todorova; Anna Paphitou; Ivana Haunerova; Toivo Maimets; Jean-Hugues Trouvin; Egbert Flory; Asterios Tsiftsoglou; Balázs Sarkadi; Kolbeinn Gudmundsson; Maura O'Donovan; Giovanni Migliaccio; Jānis Ancāns; Romaldas Maciulaitis; Jean-Louis Robert; Anthony Samuel; Johannes H Ovelgönne; Marit Hystad; Andrzej Mariusz Fal; Beatriz Silva Lima; Anca Stela Moraru; Peter Turcáni; Robert Zorec; Sol Ruiz; Lennart Akerblom; Gopalan Narayanan; Alastair Kent; Fabrizia Bignami; J George Dickson; Dietger Niederwieser; María-Angeles Figuerola-Santos; Ilona G Reischl; Claire Beuneu; Rosen Georgiev; Maria Vassiliou; Alena Pychova; Mette Clausen; Taina Methuen; Sophie Lucas; Martina Schüssler-Lenz; Vasilios Kokkas; Zsuzsanna Buzás; Niall MacAleenan; Maria Cristina Galli; Aija Linē; Jolanta Gulbinovic; Guy Berchem; Mariusz Fraczek; Margarida Menezes-Ferreira; Nela Vilceanu; Mikulás Hrubisko; Petra Marinko; Marcos Timón; Wing Cheng; George Andrew Crosbie; Nick Meade; Michelino Lipucci di Paola; Thierry VandenDriessche; Per Ljungman; Lucia D'Apote; Olga Oliver-Diaz; Isabel Büttel; Patrick Celis
Journal:  Nat Rev Drug Discov       Date:  2010-03       Impact factor: 84.694

9.  Orphan drugs. EU regulations.

Authors:  Segundo Mariz; Jordi Llinares; Kerstin Westermark
Journal:  BMJ       Date:  2011-01-11

10.  Predictors of orphan drug approval in the European Union.

Authors:  Harald E Heemstra; Remco L de Vrueh; Sonja van Weely; Hans A Büller; Hubert G M Leufkens
Journal:  Eur J Clin Pharmacol       Date:  2008-01-22       Impact factor: 2.953

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  33 in total

1.  Accelerating access to treatments for rare diseases.

Authors:  Marc Dunoyer
Journal:  Nat Rev Drug Discov       Date:  2011-06-24       Impact factor: 84.694

2.  Time to revisit the orphan drug law.

Authors:  Silvio Garattini
Journal:  Eur J Clin Pharmacol       Date:  2011-08-13       Impact factor: 2.953

3.  Alkaptonuria: treasure your exceptions.

Authors:  Timothy M Cox
Journal:  J Inherit Metab Dis       Date:  2011-09-22       Impact factor: 4.982

4.  Alkaptonuria: leading to the treasure in exceptions.

Authors:  Timothy M Cox
Journal:  JIMD Rep       Date:  2011-12-06

Review 5.  Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives.

Authors:  Peipei Song; Jianjun Gao; Yoshinori Inagaki; Norihiro Kokudo; Wei Tang
Journal:  Intractable Rare Dis Res       Date:  2012-02

6.  High constitutive Akt2 activity in U937 promonocytes: effective reduction of Akt2 phosphorylation by the histamine H2-receptor and the β2-adrenergic receptor.

Authors:  Kristin Werner; Detlef Neumann; Roland Seifert
Journal:  Naunyn Schmiedebergs Arch Pharmacol       Date:  2015-10-16       Impact factor: 3.000

7.  Worldwide collaboration for orphan drug designation.

Authors:  Segundo Mariz; James H Reese; Kerstin Westermark; Lesley Greene; Takahiro Goto; Tatsuro Hoshino; Jordi Llinares-Garcia; Bruno Sepodes
Journal:  Nat Rev Drug Discov       Date:  2016-06-01       Impact factor: 84.694

8.  Involvement of patient organisations in research and development of orphan drugs for rare diseases in europe.

Authors:  M Mavris; Y Le Cam
Journal:  Mol Syndromol       Date:  2012-09-13

Review 9.  Animal models for metabolic, neuromuscular and ophthalmological rare diseases.

Authors:  Guillaume Vaquer; Frida Rivière; Maria Mavris; Fabrizia Bignami; Jordi Llinares-Garcia; Kerstin Westermark; Bruno Sepodes
Journal:  Nat Rev Drug Discov       Date:  2013-03-15       Impact factor: 84.694

10.  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

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