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Orphanet Journal of Rare Diseases

Orphan Drugs

  1. Content type: Research

    The introduction of the orphan drug legislation led to the increase in the number of available orphan drugs, but the access to them is often limited due to the high price. Social preferences regarding funding ...

    Authors: Daria Korchagina, Aurelie Millier, Anne-Lise Vataire, Samuel Aballea, Bruno Falissard and Mondher Toumi

    Citation: Orphanet Journal of Rare Diseases 2017 12:75

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  2. Content type: Research

    Rare diseases, also referred to as orphan diseases, are characterised by their low prevalence with majority of them are chronically debilitating and life threatening. Given the low prevalence and the widely di...

    Authors: Asrul Akmal Shafie, Nathorn Chaiyakunapruk, Azuwana Supian, Jeremy Lim, Matt Zafra and Mohamed Azmi Ahmad Hassali

    Citation: Orphanet Journal of Rare Diseases 2016 11:107

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  3. Content type: Position statement

    Most statistical design and analysis methods for clinical trials have been developed and evaluated where at least several hundreds of patients could be recruited. These methods may not be suitable to evaluate ...

    Authors: Ralf-Dieter Hilgers, Kit Roes and Nigel Stallard

    Citation: Orphanet Journal of Rare Diseases 2016 11:78

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  4. Content type: Review

    In case of orphan drugs applicability of the standard health technology assessment (HTA) process is limited due to scarcity of good clinical and health economic evidence. Financing these premium priced drugs i...

    Authors: Tamás Zelei, Mária J. Molnár, Márta Szegedi and Zoltán Kaló

    Citation: Orphanet Journal of Rare Diseases 2016 11:72

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  5. Content type: Research

    Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value...

    Authors: T. Morel, S. Aymé, D. Cassiman, S. Simoens, M. Morgan and M. Vandebroek

    Citation: Orphanet Journal of Rare Diseases 2016 11:70

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  6. Content type: Research

    Health Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons). An estimated 9 % of Canadians...

    Authors: Victoria Divino, Mitch DeKoven, Michael Kleinrock, Rolin L. Wade, Tony Kim and Satyin Kaura

    Citation: Orphanet Journal of Rare Diseases 2016 11:68

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  7. Content type: Research

    Among the unique features of the Rare Diseases Clinical Research Network (RDCRN) Program is the requirement for each Consortium to include patient advocacy groups (PAGs) as research partners. This development ...

    Authors: Peter A. Merkel, Michele Manion, Rashmi Gopal-Srivastava, Stephen Groft, H. A. Jinnah, David Robertson and Jeffrey P. Krischer

    Citation: Orphanet Journal of Rare Diseases 2016 11:66

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  8. Content type: Research

    Number of orphan medicinal products on the market and number of rare disease patients, taking these usually expensive products, are increasing. As a result, budget impact of orphan drugs is growing. This facto...

    Authors: Konstantins Logviss, Dainis Krievins and Santa Purvina

    Citation: Orphanet Journal of Rare Diseases 2016 11:59

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  9. Content type: Research

    In 2005 the Italian Medicines Agency (AIFA) started a program on independent research on drugs, with the aim to promote clinical research in areas of limited commercial interest. For 3 years (2005–2007) an are...

    Authors: Giuseppe Traversa, Lucia Masiero, Luciano Sagliocca and Francesco Trotta

    Citation: Orphanet Journal of Rare Diseases 2016 11:36

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  10. Content type: Research

    Few studies have examined whether the healthcare needs of people living with rare diseases are being met. This study explores the experiences of Australian adults living with rare diseases in relation to diagn...

    Authors: Caron Molster, Debra Urwin, Louisa Di Pietro, Megan Fookes, Dianne Petrie, Sharon van der Laan and Hugh Dawkins

    Citation: Orphanet Journal of Rare Diseases 2016 11:30

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  11. Content type: Research

    The objective of this study was to assess the potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement (P&R) process with regard to orphan dr...

    Authors: Katarzyna Kolasa, Krzysztof M. Zwolinski, Zoltan Kalo and Tomasz Hermanowski

    Citation: Orphanet Journal of Rare Diseases 2016 11:23

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  12. Content type: Research

    Orphan drugs are intended to treat, prevent or diagnose rare diseases. In recent years, China healthcare policy makers and patients have become increasingly concerned about orphan drug issues. However, very fe...

    Authors: Shiwei Gong, Yingxiao Wang, Xiaoyun Pan, Liang Zhang, Rui Huang, Xin Chen, Juanjuan Hu, Yi Xu and Si Jin

    Citation: Orphanet Journal of Rare Diseases 2016 11:20

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  13. Content type: Position statement

    In the past few years there has been a political imperative driving the creation of European Reference Networks as these are considered a promising way to achieve equity in access to the most up to date medica...

    Authors: Teresinha Evangelista, Victoria Hedley, Antonio Atalaia, Matt Johnson, Stephen Lynn, Yann Le Cam and Kate Bushby

    Citation: Orphanet Journal of Rare Diseases 2016 11:17

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