NICE's Soliris request – a watershed for ultra-orphan drugs or more of the same?
Published in FirstWord 6 March 2014 by Shrinivas Rao Mukku and Simon King
Forbes' healthcare editor Matthew Herper noted back in January that when "Alexion's performance stops beating like clockwork, we'll know that pricing pressure has come to medicine."
Herper was referencing more specifically the performance of Alexion Pharmaceuticals' ultra-orphan therapy Soliris, which is indicated for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uremic syndrome (aHUS), and accounts for all of the company's revenue ($1.6 billion in 2013).
Highlighting the particularities of the ultra-orphan drug model, Soliris is regularly cited for its status as being the world's most expensive pharmaceutical product, costing around $400,000 per patient per year in the US market.
That price has yet to be challenged in the US, but in Europe the ultra-orphan model appears to be under increased pressure. Does a decision by the UK's National Institute for Health and Care Excellence to ask Alexion to explain the price of Soliris suggest that this trend may be intensifying?
Biosimilars: Friend or foe to healthcare?
Our recent article in February’s Pharma Market Europe and our latest Free Thinking white paper looks at the opportunities for biosimilars and the threats to innovator biologics.
Biologics, whose active substance is derived from a living organism, have enabled healthcare to make significant therapeutic advances across a whole raft of therapy areas since their introduction in the 1970s. However, these medicines come at a price, reflecting the complexities involved in developing and manufacturing products derived from living cells. Annual treatment costs in the US can range from US$25,000 to US$200,000. Biosimilars medicines, the follow-on “generics” to biologics which have come off-patent, present a significant opportunity to introduce cutting-edge therapies to the treatment landscape for many diseases, while also addressing the cost-effectiveness demands now being made on global healthcare systems.