In recent months the focus on the need to develop new drugs and fund new treatments for rare diseases has intensified.
In the UK, NHS England is seeking to engage pharma to come up with some practical solutions by holding a consultation on investing in specialised services – including rare and very rare diseases.
“This should not be based on a blunt assessment of affordability but on the impact of treatment on the unmet medical need, burden of illness and impact on patients and carers,”
The biopharma industry trade body, the BioIndustry Association (BIA).
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