Orphan Drugs

  1. Research

    Determinants of orphan drugs prices in France: a regression analysis

    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 ...

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

    Orphanet Journal of Rare Diseases 2017 12:75

    Published on: 21 April 2017

  2. Research

    Off-label use of orphan medicinal products: a Belgian qualitative study

    Off-label use of (orphan) medicinal products for (rare) diseases is quite common but not underpinned by clinical studies to confirm efficacy and safety. No risk-analyses by regulatory agencies are carried out....

    Marc Dooms, David Cassiman and Steven Simoens

    Orphanet Journal of Rare Diseases 2016 11:144

    Published on: 28 October 2016

  3. Research

    State of rare disease management in Southeast Asia

    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...

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

    Orphanet Journal of Rare Diseases 2016 11:107

    Published on: 2 August 2016

  4. Research

    Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007–13) and prospective (2014–18) MIDAS sales data analysis

    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...

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

    Orphanet Journal of Rare Diseases 2016 11:68

    Published on: 21 May 2016

  5. Research

    The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network

    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 ...

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

    Orphanet Journal of Rare Diseases 2016 11:66

    Published on: 18 May 2016

  6. Research

    Impact of orphan drugs on Latvian budget

    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...

    Konstantins Logviss, Dainis Krievins and Santa Purvina

    Orphanet Journal of Rare Diseases 2016 11:59

    Published on: 11 May 2016

  7. Research

    Italian program for independent research on drugs: 10 year follow-up of funded studies in the area of rare diseases

    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...

    Giuseppe Traversa, Lucia Masiero, Luciano Sagliocca and Francesco Trotta

    Orphanet Journal of Rare Diseases 2016 11:36

    Published on: 12 April 2016

  8. Research

    Survey of healthcare experiences of Australian adults living with rare diseases

    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...

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

    Orphanet Journal of Rare Diseases 2016 11:30

    Published on: 24 March 2016

  9. Research

    Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs

    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...

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

    Orphanet Journal of Rare Diseases 2016 11:23

    Published on: 10 March 2016

  10. Research

    The availability and affordability of orphan drugs for rare diseases in China

    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...

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

    Orphanet Journal of Rare Diseases 2016 11:20

    Published on: 27 February 2016

  11. Position statement

    The context for the thematic grouping of rare diseases to facilitate the establishment of European Reference Networks

    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...

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

    Orphanet Journal of Rare Diseases 2016 11:17

    Published on: 24 February 2016

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