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Orphan drugs and rare diseases – value demonstration

The third in a series of supplements to look at orphan drugs and rare disease, this issue covers the role of value demonstration.

It features expert opinion on the European health technology assessment (HTA) landscape for orphan drugs, strategies for demonstrating value and the important of putting patients and carers at the heart of the equation.

There’s also a look the changes to how ‘ultra orphans’ are assessed in the UK by NICE.



View: Orphan drugs and rare diseases: Value Demonstration


Further online orphan drug and rare diseases resources are available from PMLiVE in its dedicated online section for the area.

This showcases our orphan drug and rare disease content, splitting it up for easy navigation into sections on CROs and trial design, early access and regulation, strategic communications and value demonstration.

Orphan drugs and rare diseases 

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