More Evidence Needed For Chiesi’s Ultra Orphan Lamzede, Says NICE
Chiesi still has work to do to convince NICE that its ultra rare disease treatment Lamzede is a cost-effective treatment.
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New approvals in the EU were again dominated by oncology drugs in 2018, although medicines for infectious diseases were a close second. The number of orphan drugs more than doubled over the previous year, to 17. There were also some star arrivals in multiple sclerosis, genetic disorders, metabolism and migraine.
Patient access schemes have helped the Scottish Medicines Consortium recommend rare disease products, including Regeneron/Sanofi’s Libtayo for skin cancer.