The NHS has just been given permission to buy patisiran, a new gene silencing drug which halts the progress and can reverse the progress of a disease called amyloidosis, a horrible nerve condition which starts with pins and needles in the feet and hands and progresses rapidly to paralysis, organ failure and death, usually within 3-15 years of the first symptoms being seen. It’s an hereditary disease, devastating entire families. It is also very rare, about 150 people are affected in the UK.
In the US the average cost of this drug is $345,000 per patient per year (not sure but I think once you are on it, you are on it for life if it works). It is administered by IV drip once every three weeks. In the US the manufacturers (not my company, sadly) are thought to be offering a ‘money back if it doesn’t work’ deal (which of course means they are sensationally confident). It has taken them 15 years to develop the drug to this stage, in a branch of science that many other companies have given up on.
NICE originally turned down the drug for us, on grounds that the £300,000 pa price was too much. Now they have been offered a ‘confidential discount’ and have given permission for its use.
I’d like to hear what people think of this, if anyone is interested. It’s a great example of the direction medicine is going - very effective treatments tailored specifically to certain patients and diseases. Of course the more targeted the medicine means relatively small numbers of patients will get it and the more expensive it will be. A real health care funding dilemma.
The Pharma industry deserves no sympathy at all, but it’s a big risk to take spending billions over many years on something with a high chance of failure with a very small ‘market’ even if it does work.
