Uncertainty over how long patients benefit makes it hard to know whether the gene therapy is worth the money.
Normal clotting factor injections cost between £150,000 and £200,000 per patient per year for life.
But the gene therapy – branded Hemgenix – has an official list price of £2.6m.
Elliott says it’s “wild to think that’s how much it costs” and now he had multi-million pound blood he should “probably get life insurance”.
The treatment is being made available through a deal between the company CSL Behring, NHS England and the National Institute of Health and Care Excellence, the body that rules on cost-effectiveness.
I have pressed all the parties involved for details of the deal, but it is bound up in confidentiality agreements.
As I understand it, the NHS will track how much patients benefit for more than a decade and that will dictate how much CSL Behring are paid. If factor IX levels dip earlier than expected, then the NHS will pay less. It is essentially performance-related pay for drugs and it is the first such deal the NHS has agreed.
The therapy will be available immediately at eight centres – Oxford, Manchester, Leeds, Bristol, Birmingham, Cambridge and two in London.
Around 2,000 people in the UK have haemophilia B and the NHS anticipates treating 250 of them.
Not everyone will be suitable for the gene therapy as some are too young or have other health conditions that rule them out.
Prof Sir Stephen Powis, the NHS national medical director, said the “transformative” gene therapy could be “truly life-changing” and had been secured “at an affordable price”.
