Trick or treat? When new therapies stretch the price barrier

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that affects around 10,000 people globally. The red blood cells in PNH patients are weak and are destroyed more rapidly than normal. This causes the urine to turn red or dark during an episode (or paroxysm) of red cell destruction (or hemolysis).

Usually the urine is darker first thing in the morning (nocturnal) and clears through the day. Each episode of dark urine usually lasts for a few days and episodes may occur very occasionally or very often. During an attack of dark urine many patients have mild abdominal discomfort. However, some patients with PNH never have attacks of dark urine, so the condition can be erratic.

The standard treatment for PNH was blood transfusions every three months to stave off chronic tiredness, jaundice and difficulty swallowing. Alexion Pharmaceuticals has developed a new treatment for PNH called Soliris (eculizumab), an infusion therapy given every couple of weeks that reduces the need for dependence on blood transfusions. Eculizumab is a humanised monoclonal antibody that inhibits terminal complement activation.

The catch?

The treatment costs approx. $400K per year, something that has raised a lot of concern, as you can see from reports here, here and here.

It's hard to justify such a high price for a therapy when the top end cost for drugs that target around 10,000 patients globally is under $300K a year; even some UK PCT's thought it was not cost effective in the absence of NICE review, as reported recently on BBC News.