Landmark legislation in Italy: Provisions for the treatment of rare diseases and for the support of research and production of orphan drugs
FDA approves first drug to improve growth in children with most common form of Dwarfism
NICE creates new menu of treatment options for those suffering from depression
We examine how real-world evidence strategies help address uncertainties in rare disease treatments ...
Achieving HTA approval for orphan drugs requires strategic planning, early engagement, strong eviden...
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