Not so NICE...

Earlier today I caught this bit on the news $-$ that's the story of the latest NICE deliberation on ataluren, a treatment for Duchenne muscular dystrophy. That's a rare and horrible condition $-$ no doubt about it. The story is mainly that NICE has preliminary decided not to recommend ataluren for reimbursement (the full Evaluation consultation document is here).

I thought the report in the news was not great and, crucially (it seems to me), it missed the point. The single case of any individual affected is heart-breaking, but the media are not doing a great service to the public in picturing (more or less) NICE, or as they say "the drug watchdog", as having rejected the new drug because it's too expensive.

ITV reporting quotes the father of one of the children affected as saying:

How do we tell Archie he is not allowed a drug that will keep him walking and living for longer because NHS England and drug companies cannot agree on a price?

That's the real problem, I think $-$ the presumption (which most of the media do nothing to explore or explicitly state) is that the drug will keep the affected children walking and living longer. Trouble is that this is not a certainty at all $-$ on the basis of the evidence currently available. The Evaluation consultation document says (page 34)

There were no statistically significant differences in quality of life betweenthe ataluren and placebo groups. The company stated there was apositive trend towards improved quality of life with ataluren 40 mg/kg dailyin the physical functioning subscale. The company submission alsodescribed a positive effect on school functioning and a negative trend inemotional and social subscales.

So the point, I think, is that if the treatment was associated with much more definitive evidence, then the discussion would be totally different. What has not been mentioned, at least not that I have seen, is that the estimated total cost per person per year of
treatment with ataluren of £220,256 is affected by the uncertainty in the evidence and assumptions enconded in the model presented for assessment. And it is this uncertainty that needs to be assessed and carefully considered...