Value demonstration

Assessing drugs for ultra-rare conditions in the UK
Assessing drugs for ultra-rare conditions in the UK Alexion’s Soliris has become the first drug to pass through NICE’s new highly specialised technology process
No price ceiling for rare disease drugs, say MPs
No price ceiling for rare disease drugs, say MPs BIA survey comes ahead of consultation on evaluation framework for ultra-orphans
NICE and ‘ultra orphans’
NICE and ‘ultra orphans’ NICE and its processes for ‘highly specialised therapies’
Define the patient burden in rare diseases
Define the patient burden in rare diseases Putting patients and carers at the heart of the value demonstration equation in orphan drugs is key
EMA to expand orphan drug HTA collaboration
EMA to expand orphan drug HTA collaboration Will further develop the concept of parallel scientific advice in 2014
Supportive strategies for demonstrating value
Supportive strategies for demonstrating value Improved planning will support better data generation and improve understanding of rare diseases and the orphan drugs that treat them
Orphan indication? No easy access
Orphan indication? No easy access Health technology assessment for orphan drugs continues to present challenges for pharmaceutical companies
Rare disease strategy for the UK launched
Rare disease strategy for the UK launched Aims to improve understanding and boost research
NICE demands more data from Roche for MabThera in rare autoimmune disease
NICE demands more data from Roche for MabThera in rare autoimmune disease Unable to recommend expanding drug’s indication to include vasculitis
Revlimid faces NICE rejection for use in rare blood cancer
Revlimid faces NICE rejection for use in rare blood cancer Watchdog's draft guidance does not recommend Celgene's drug for NHS use in England and Wales
Payer focus in the personalised world of oncology and orphan diseases
Payer focus in the personalised world of oncology and orphan diseases Personalised medicine is transforming disease understanding and many traditional diagnoses may be fundamentally revised based on the new scientific understanding
Novartis’ Jakavi turned down by NICE for blood cancer
Novartis’ Jakavi turned down by NICE for blood cancer UK cost-effectiveness watchdog concerned about 'uncertainties' in data in draft guidance
Orphan drugs economically viable for pharma
Drugs for rare diseases can generate as much lifetime revenue as medicines for common conditions

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