The Office for Health Economics (OHE) has released a report in which it examines the ethics behind the way medicines for ultra-orphan diseases are appraised.
The report emphasises the relatively high spend per patient in developing orphan and ultra-orphan medicines, which often leads to high incremental cost-effectiveness ratios (ICERs), far in excess of the conventional threshold used by the National Institute for Health and Care Excellence (NICE) of £20,000 – £30,000 per quality-adjusted life year (QALY).
To account for this, NICE established the highly-specialised technology (HST) assessment route for ultra-orphan products, in which the threshold is a sliding scale between £100,000 and £300,000/QALY. However, The Office of Health Economics (OHE) reports that the criteria for entry to the HST programme are stringent, and yet have also been criticised as ambiguous or vague.
The OHE report considers the ethics behind this effective different valuation of QALYs for rare diseases, and factors-in various ethical and economical principles in its discussion. A wide range of evidence is cited by OHE, including MAP’s own report Access to Orphan Medicines: A Case for Change.
Read the report here.