It’s less than a week since 2014’s Rare Disease Day, an annual event held to raise awareness for rare diseases and improve access to treatment and medical representation for individuals with rare diseases and their families. Linked to this issue is the announcement from UK’s NICE (National Institute for health and Care Excellence) that it is asking the manufacturer of just such a treatment to justify its massive price tag.
A rare/orphan disease is classified in the US as one that affects fewer than 200,000 Americans, whereas in the EU, it’s any disease that affects less than 5 per 10,000. According to the European Organization for Rare Diseases (EURORDIS), treatment for many rare diseases is insufficient, as are the social networks to support individuals with rare diseases and their families.
There are special rules to encourage manufacturers to develop treatments for such diseases. These include tax benefits, extended patent protection etc. After all, by definition, the market is going to be limited.
However, NICE has asked Alexion why, despite these benefits, it is still charging what NICE claims is up to £340,200 per year per patient for its drug, Soliris (eculizumab). This makes Soliris the most expensive drug currently on the market.
The drug is a highly effective treatment for aHUS – a chronic condition that affects around 200 people in the UK and causes severe inflammation of blood vessels plus the formation of blood clots in small blood vessels throughout the body. Sufferers of aHUS may develop significant kidney damage, heart failure, thrombosis (blood clots) and brain injury.
NICE usually weighs up the cost-effectiveness of drugs by assessing patient benefit etc – but this is the first time it has directly asked for justification of the price changed for a drug. Its official statement includes the question "Is the preferred 'total cost' reasonable in the context of R&D and manufacturing costs for this technology?"
Not only drug manufacturers but EURORDIS and the sufferers of orphan diseases it represents will be viewing developments with some interest. Does this signal the beginning of a clamp down on over charging? Will it drive developers away from working on orphan diseases? Is NICE simply trying to channel resources to where they will do the most good?
I’d love to know what you think.
