Scotland’s health technology assessment body has agreed to reimburse Chiesi’s Elfabrio for Fabry disease, mirroring the decision from several other European countries – however, France turned down the rare disease drug, while Germany questioned its benefit.
Scotland has become the seventeenth country in Europe to agree to reimburse Elfabrio (pegunigalsidase alfa), Chiesi’s treatment for Fabry disease that was rejected by France’s health technology assessment (HTA) body.
The Scottish Medicines Consortium announced its decision to fund the enzyme replacement therapy this month, meaning that the product will soon be available on a routine
BioMarin’s ultra-rare disease drug Brineura has breached cost-effectiveness thresholds for highly specialized treatments in England and looks set to be denied routine funding on the National Health Service.
Policy experts and pharma executives react to President Trump’s executive order on “equalizing” drug prices between the US and comparable nations at the ISPOR 2025 conference.
In an interview with the Pink Sheet, Fonadazione Telethon’s Stefano Benvenuti discusses how companies developing and marketing rare disease treatments can overcome the challenges presented by EU-joint clinical assessment evidence requirements.
England’s health technology assessment institute, NICE, must ensure that there is clarity for manufacturers on how its methods and processes might change following testing in its sandbox environment, the Association of the British Pharmaceutical Industry said.
Policy experts and pharma executives react to President Trump’s executive order on “equalizing” drug prices between the US and comparable nations at the ISPOR 2025 conference.
In an interview with the Pink Sheet, Fonadazione Telethon’s Stefano Benvenuti discusses how companies developing and marketing rare disease treatments can overcome the challenges presented by EU-joint clinical assessment evidence requirements.
New draft guidance on the third cycle of Medicare drug price negotiations goes against Merck and BMS expectations for their cancer drugs. The guidance also tackles Part B and renegotiated prices for the first time.
