In late 2021, the health technology assessment of the French National Authority for Health was seized by the French Ministry of Solidarity and Health to address a specific challenge, the identification of solutions contributing to the development of the methodological expertise in new types of clinical studies for rare diseases. Experts from the rare diseases environment were gathered by OrphanDev, the French network of expertise dedicated to rare diseases. They allowed to identify some of the current issues in France concerning rare diseases, and then present different solutions, in particular related to the evaluation process of orphan drugs and the collection of data on rare diseases
Rare diseases, although individually rare, affect approximately 6–8% of the total population with mo...
According to the WHO a disease or disorder is defined as rare in Europe when it affects fewer than ...
This dissertation emphasizes the challenges raised by the management of rare diseases and is structu...
In late 2021, the health technology assessment of the French National Authority for Health was seize...
Rare diseases imply clinical and economic burden as well as a significant challenge for health syste...
Rare diseases are a heterogenic group of disorders with a little in common except of their rarity a...
A timely diagnosis is a critical step to ensure a proper access to expert clinical management for pa...
The interest to rare diseases has increased in the recent decades. Legislation seeks to facilitate p...
A rare disease is a health disorder that affects a small proportion (i.e., <1:2,000 in Europe) of th...
An estimated 400 million individuals suffer from rare diseases globally. Tackling rare diseases has ...
© 2020, Advanced Scientific Research. All rights reserved. Rare diseases are defined as diseases wit...
The interest to rare diseases has increased in the recent decades. Legislation seeks to facilitate p...
International audienceBACKGROUND:Although rare disease patients make up approximately 6-8% of all pa...
Rare diseases are a major health-care burden worldwide. Very little is known about the cause, behavi...
International audienceThe French-style organization in the field of rare diseases allows a close con...
Rare diseases, although individually rare, affect approximately 6–8% of the total population with mo...
According to the WHO a disease or disorder is defined as rare in Europe when it affects fewer than ...
This dissertation emphasizes the challenges raised by the management of rare diseases and is structu...
In late 2021, the health technology assessment of the French National Authority for Health was seize...
Rare diseases imply clinical and economic burden as well as a significant challenge for health syste...
Rare diseases are a heterogenic group of disorders with a little in common except of their rarity a...
A timely diagnosis is a critical step to ensure a proper access to expert clinical management for pa...
The interest to rare diseases has increased in the recent decades. Legislation seeks to facilitate p...
A rare disease is a health disorder that affects a small proportion (i.e., <1:2,000 in Europe) of th...
An estimated 400 million individuals suffer from rare diseases globally. Tackling rare diseases has ...
© 2020, Advanced Scientific Research. All rights reserved. Rare diseases are defined as diseases wit...
The interest to rare diseases has increased in the recent decades. Legislation seeks to facilitate p...
International audienceBACKGROUND:Although rare disease patients make up approximately 6-8% of all pa...
Rare diseases are a major health-care burden worldwide. Very little is known about the cause, behavi...
International audienceThe French-style organization in the field of rare diseases allows a close con...
Rare diseases, although individually rare, affect approximately 6–8% of the total population with mo...
According to the WHO a disease or disorder is defined as rare in Europe when it affects fewer than ...
This dissertation emphasizes the challenges raised by the management of rare diseases and is structu...
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