I have explained before that the UK's National Institute of Health and Clinical Excellence (NICE) is not responsible for evaluating the clinical efficacy and subsequent use of a particular drug, but rather evaluates the economic benefits, or not. of recommending whether such a drug or procedure should be provided on our National Health Service. Woe-be-tide the NHS trust that doesn't follow a positive guideline. However, even though clinicians are allowed to use a drug that has been clinically approved but not NICE approved, the NHS Trust management teams are not happy to do this as they haven't budgeted for it.
There have been various cases recently in the news where people with terminal illnesses have taken their local NHS trusts to court to receive clinician-recommended but not NICE-recommended medications – it seems the patients have won as the NICE guidelines are non-statutory.
Dr Crippen pointed me to Lake Cocytus' post about a palliative drug in the relief of Alzheimer's Disease called donepezil produced by the BigPharma Eisai Ltd. Lake Cocytus then pointed me to the official transcript of the appeal of appeal courts.
It seems every fuckin' newspaper reported the judgement differently ... it seems the media interpreted the judgement as about this particular treatment. Some said it was a victory for Alzheimer's patients, some said it was a victory for NICE.
It wasn't about the drug. It wasn't about Alzheimer's. It was about NICE.
Before I go into the details of this particular case, I should explain a little of what I have learned of NICE's evaluation process, of its guidelines for the use of drugs and treatments in the NHS.
In the original appeal of this case it was established that “NICE generally seeks to ascertain the incremental cost per quality adjusted life year (or 'cost per QALY') of using a particular health technology”. What the definition of a QALY is I don't know ... hmm I'll have to look this up on NICE's website.
“The general threshold for an acceptable cost per QALY is approximateley £20,000 per QALY ... Above approximateley £30,000 per QALY, technologies are not normally recommended ... Between £20,000 and £30,000 per QALY, NICE will consider whether there are special considerations which justify recommending the technology, such as its innovative nature or the particular needs of the relevant patient group.”
To model the cost-effectiveness of a drug NICE uses a piece of software developed in the USA known as the AHEAD model, then adapted by the Southampton Health Technology Assessment Centre (SHTAC) to which SHTAC owns intellectual property rights.
During the lengthy process of revisions and appeals NICE will only provide the manufacturer or other interested parties with a read-only version of their economic model whilst an appellant must provide a fully executable version of any alternative economic model they have themselves used.
This particular case brought by the manufacturer, Eisai Ltd, involved a revision of NICE guidelines of Alzheimer Disease drug donepezil restricting its use to a narrower group of Alzhimer patients. NICE, using its own economic model, did manage to bring down the cost per QALY significantly but nowhere enough to meet its own guidelines. So Eisai went to the courts.
Eisai was not happy with applying the normal QALY model to Alzeimer's. And I do not have the clinical knowledge to argue that. However, that was not the basis of their appeal.
Eisai basically contended that NICE was guilty of unfair “trading” practices by only providing appellants with a read-only copy of the model in which the reliability of the model could not be tested by running sensitivity analyses, whilst appellants had to provide NICE with fully executable versions of their own alternative models.
The case went to a lower appeal which Eisai lost and then a higher appeal which Eisai has won.
It wasn't about Alzeimer's. It wasn't about donepezil. To the extent the drug still may well not be approved even when NICE produce the appellants with an executable economic model ... and I guess it will have to now renegotiate its agreement with SHTAC.
It is about the openness of NICE evaluation procedures. It is about copyright and industry confidentiality. NICE arguments centered on the contractural confidentiality agreements forced on it by SHTAC. A NICE witness even suggested they could reverse-engineer the software !
However, intellectual property rights and copyright were hardly mentioned, as the case was brought by BigPharma who are interested in their own intellectual property and copyright rights
Bloody hell ... are the Times, Daily Telegraph and Guardian legal and medical correspondents such fuckwits about medico-legal judgements? Did they irresponsibly scan this in 5 minutes and write a report when I took four hours to produce these shallow comments ?
Update: The original SHTAC study can be found here.