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Literature DB >> 19552743

Orphan drug development is not taking off.

Roberta Joppi, Vittorio Bertele, Silvio Garattini.

Abstract

Mesh：

Substances：

Year: 2009 PMID： 19552743 PMCID： PMC2686065 DOI： 10.1111/j.1365-2125.2009.03369.x

Source DB: PubMed Journal: Br J Clin Pharmacol ISSN： 0306-5251 Impact factor: 4.335

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6 in total

1. Rare diseases and orphan drugs.

Authors: J K Aronson
Journal: Br J Clin Pharmacol Date: 2006-03 Impact factor: 4.335

2. Orphan drug development is progressing too slowly.

Authors: Roberta Joppi; Vittorio Bertele; Silvio Garattini
Journal: Br J Clin Pharmacol Date: 2006-03 Impact factor: 4.335

3. Rare diseases: what's next?

Authors: Giuseppe Remuzzi; Silvio Garattini
Journal: Lancet Date: 2008-06-14 Impact factor: 79.321

Review 4. Why rare diseases are an important medical and social issue.

Authors: Arrigo Schieppati; Jan-Inge Henter; Erica Daina; Anita Aperia
Journal: Lancet Date: 2008-06-14 Impact factor: 79.321

5. Clinical trials of orphan medicines.

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

6. 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

6 in total

18 in total

Review 1. [Off-label therapy: current problems from the perspective of the Pharmaceutical Commission of the German Medical Profession].

Authors: R W C Janzen; W D Ludwig
Journal: Z Rheumatol Date: 2012-02 Impact factor: 1.372

Review 2. Rare diseases, orphan drugs and their regulation: questions and misconceptions.

Authors: Erik Tambuyzer
Journal: Nat Rev Drug Discov Date: 2010-11-09 Impact factor: 84.694

3. Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia.

Authors: Carl Rudolf Blankart; Tom Stargardt; Jonas Schreyögg
Journal: Pharmacoeconomics Date: 2011-01 Impact factor: 4.981

4. Safety-related regulatory actions for orphan drugs in the US and EU: a cohort study.

Authors: Harald E Heemstra; Thijs J Giezen; Aukje K Mantel-Teeuwisse; Remco L A de Vrueh; Hubert G M Leufkens
Journal: Drug Saf Date: 2010-02-01 Impact factor: 5.606

5. Time to revisit the orphan drug law.

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

6. Access to orphan drugs despite poor quality of clinical evidence.

Authors: Alain G Dupont; Philippe B Van Wilder
Journal: Br J Clin Pharmacol Date: 2011-04 Impact factor: 4.335

Review 7. An empirical review of major legislation affecting drug development: past experiences, effects, and unintended consequences.

Authors: Aaron S Kesselheim
Journal: Milbank Q Date: 2011-09 Impact factor: 4.911