John Murray of the Specialised Healthcare Alliance raises a number of spectres around treatments for rare diseases, without discussing the underlying principles of prioritisation, writes Jonathan Howell
A number of orphan drugs cost£250,000 per patient per year, which could mean several million pounds over the lifetime of a patient. This can leave you with a cost per quality adjusted life year of£500,000, which compares poorly with the National Institute for Health and Clinical Excellence's upper threshold of£30,000.
For£250,000, I could put 10 patients on renal dialysis. I would know exactly what I was getting for them, whereas many of the orphan drugs have a limited research base and the long-term outcomes are uncertain. In a sense, this means 10 lives against one potential life.
I have every sympathy for patients with rare diseases, but this is precisely the same sympathy as I have for any patient with a serious disease. My job is to help make sure that the NHS uses its resources in the most fruitful way possible and to the best value.
We need to be more open and seek more publicity about how and why we make the value-based decisions we do. We need a debate to ensure NHS priorities and decisions are reasonable and not distorted.
Dr Jonathan Howell, consultant in public health with specialised services commissioning, Stafford