The capture-recapture method to estimate surveillance data for rare diseases

The capture-recapture method to estimate surveillance data for rare diseases
An article published by Smith MG, et al. in the Orphanet Journal of Rare Diseases compares two methods to estimate the number of cases of two rare conditions (fragile X syndrome (FXS) and muscular dystrophy (MD)): the basic surveillance through active medical record review and a linked administrative data file. © Inserm/Latron, Patrice Active [...]

European Medicines Agency releases framework and action plan for increased collaboration with academia

European Medicines Agency releases framework and action plan for increased collaboration with academia
In its endeavor to further interactions with the academic community, the European Medicines Agency (EMA) has recently presented a framework that formalizes increased collaboration as well as an action plan to do so. They have also introduced a web page for academia which provides up-to-date information on the EMA and academia collaboration. [...]

Tell OECD how health systems can improve sustainable access to innovative pharmaceutical therapies

Tell OECD how health systems can improve sustainable access to innovative pharmaceutical therapies
The OECD is undertaking an international stakeholder dialogue to improve patient access to innovative pharmaceutical treatments and ensure the sustainability of health spending as well as continued innovation that meets patient needs. In this capacity, the OECD is inviting submissions to identify issues with the current system, to understand [...]

Rare disease policy in 11 countries: an analysis

Rare disease policy in 11 countries: an analysis
The national rare disease policy in 11 countries have been compared and contrasted in an article published in the Orphanet Journal of Rare Diseases. The authors evaluated the rare disease policy landscape in Germany, France, the United Kingdom, Canada, Bulgaria, Turkey, Argentina, Mexico, Brazil, China, and Taiwan across five key dimensions. [...]

Risky business? Rare disease patients and caregivers willing to endure certain side-effects for better drug response

Risky business? Rare disease patients and caregivers willing to endure certain side-effects for better drug response
An article published in the Orphanet Journal of Rare Diseases discusses how rare disease patients and caregivers make benefit-risk assessments for therapeutic options that may be potentially available to them. The survey conducted by the authors show that when faced with a hypothetical treatment, treatments that have the highest chance of wor [...]

European Reference Networks: a new concept came to reality

European Reference Networks: a new concept came to reality
The 3rd official European Reference Network (ERN) conference took place in Vilnius, Lithuania, on the 9th of March. The following day was dedicated to the kick-off meetings for the 24 thematic European Reference Networks (ERNs). This was a major event, involving approximately 600 delegates, and was organised under the auspices of the Preside [...]