- NICE has now approved Alexion's Soliris for patients with atypical Hemolytic Uremic Syndrome (aHUS). The agency rejected the drug in January 2013, requesting additional information about manufacturing and R&D costs. Alexion has since provided that information and received a qualified approval.
- Atypical HUS (aHUS) is a serious and rare orphan disease that currently affects around 200 people in England. The number of people affected by aHUS is expected to grow by 20 people per year.
- Soliris has been dubbed the most expensive drug in the world and is estimated to cost approximately $543,000 per year per patient.
At a cost of more than half a million dollars per year to treat just one patient, Soliris is exorbitantly pricey. However, with such a small treatment population, NICE was able to estimate its long-term costs up front. According to British government estimates, treating aHUS patients with Soliris will cost the National Health Service (NHS) $95.5 million during the first year after approval. As the patient population slowly rises, the cost will rise to $135 million.
Though the additional information provided to NICE by Alexion was sufficient to gain approval, the approval is a qualified one. In order to receive Soliris, patients must be treated at specialty treatment centers. The agency also stipulates that systems must be put into place to monitor how many people are diagnosed with aHUS, how many of them receive Soliris, at which doses, and for how long. The rationale is that careful oversight will help reduce, or at least control, potentially explosive long-term costs.