Finding the Key...
Finding the Key...
NICE’s arm had to be twisted recently by the Court of Appeal in order to release a working economic model used in the Institute’s re-appraisal of Alzheimer’s disease drugs. Pf takes a closer look at the ruling and its implications.
Readers will know that the NHS this month celebrated its 60th birthday, commemorated by Lord Darzi’s Next Stage Review. Another birthday on the horizon, but one less likely to be celebrated in the same way, is the 10th anniversary of the National Institute of Health and Clinical Excellence in spring 2009. At its annual conference in December this year, Chairman Professor Sir Michael Rawlins will start to say his goodbyes as he is stepping down. So, as we move towards the end of the Institute’s first decade, is it the end of an era and, perhaps, the beginning of a new one?
New paradigms
Of course, the industry has never liked NICE much, ever since its inception. For many drugs, NICE has added an extra step to market, and it has always been hoped quietly that it would just go away. Recently there was comment in the industry press that if advice from the SMC and NICE was essentially the same, then why were there two such bodies and wouldn’t it be better just to have the SMC – faster, cheaper and with a better appeals system? But this is actually missing the point – these are two very different health technology assessment (HTA) bodies responding to the needs of different jurisdictions. The SMC provides rapid health technology assessment advice, at arms length from the devolved Scottish Government; NICE provides in-depth appraisal guidance as well as more rapid guidance. Assessment and appraisal are not the same thing. There are many differences between the two kinds of ‘products’, with assessment essentially being the ‘science’ and appraisal being a ‘value judgment’.
Following the House of Commons Health Select Committee’s second inquiry into NICE, some things may change, but NICE is not going to go away. It is not going to be canned, and companies therefore need to adjust their views accordingly – it is no longer an option for the industry to default to the usual ‘stick to the knitting’ thinking. So it is worth recalling that in the very early days, NICE stressed that, on occasion, it might have to advise that certain new treatments, although offering limited clinical benefits, would not be cost effective – that is, that the NHS could achieve much greater health benefits by using its resources in other ways. It also said that there was no absolute right for new medicines to get onto the market and they had to compete with all the other forms of investment that the NHS has to make. Professor Sir Michael Rawlins said at the same time, “I understand that it would be galling, after investing large sums of money in the development of new treatments, to find that the Institute does not endorse their use in the NHS. Nevertheless, no healthcare system in the world can afford to use technologies that fail the tests of clinical and costeffectiveness, and nor should they.”
This is, then, the new paradigm that companies need to acclimatise better to. Certainly, the impact of NICE on UK pharma in these last nearly 10 years has been profound, as this relates to market access, and companies have often appealed against NICE decisions. But until fairly recently, NICE decisions had never been challenged in the courts. Then a judicial review was lodged by one company over NICE’s decision on Alzheimer’s disease drugs.
The Alzheimer’s disease drugs story
The first appraisal of these drugs was published in 2001 and was positive. But all drugs are reappraised after a while and in its re-appraisal in 2006, NICE proposed a complete U-turn by recommending that these drugs should no longer be funded by the NHS. The decision created a terrible stink and uproar in the industry, not to mention patient groups. NICE then modified its decision to allow the use of the drugs in moderate disease only, still insisting that the drugs were not cost-effective in early stages of the disease. The company concerned then applied for a judicial review on the grounds that the process by which NICE had developed its guidance was unfair, which was heard in June 2007. The Alzheimer’s Society was also listed as an interested party.
In August 2007, the judge ruled in favour of NICE on five out of the six grounds brought to court and, in September, NICE published an amended version to fully comply with the judge’s ruling – about providing specific advice regarding people with learning disabilities and making the guidance more appropriate for people for whom English was not their first language. But essentially the guidance still stood. And then in the same month, leave to appeal against the judge’s ruling was applied for on one point of the original ruling: that NICE had not breached principles of procedural fairness by providing a ‘read only’ version of the economic model. The appeal was heard this April and the outcome was announced at the beginning of last month.
Judicial Review outcome
Most interestingly, the Court of Appeal found in favour of the company, ruling that NICE should have been more transparent in the way it made decisions over the Alzheimer’s drugs. It found that NICE had indeed breached principles of procedural fairness by providing a ‘locked’ version of the economic model and that, by doing so, it was withholding information that placed the company at “a significant disadvantage” should it have wanted to challenge the NICE ruling. The Court also said that “without the fully executable model, a consultee cannot check whether there are variables to which the model is particularly sensitive and make informed representations accordingly”. NICE will now have to provide the company with an executable version of the cost-benefit analysis model used in the appraisal, so that they can comment on it. NICE will then have to consider these comments and this could mean that NICE has to review its decision.
“I understand that it would be galling, after investing large sums of money in the development of new treatments, to find that the Institute does not endorse their use in the NHS. Nevertheless, no healthcare system in the world can afford to use technologies that fail the tests of clinical and cost-effectiveness, and nor should they” Professor Sir Michael Rawlins
The Institute has stressed that it has not yet been asked to amend or withdraw the current guidance and that the drugs continue to be recommended only for people at the moderate stage of the disease. In a press statement, NICE Chief Executive Andrew Dillon said, “We will be considering very carefully the findings and the implications for the time it takes us to provide advice to patients and the NHS on the use of new treatments. The ruling will increase the complexity of our drug appraisals in some cases and they may take longer as a result.” But the ABPI welcomed the Court of Appeal’s ruling, saying that it makes NICE’s method of arriving at its recommendations on a medicine’s availability more open and subject to greater scrutiny. ABPI Director General Richard Barker said it provides “further momentum behind the drive to make NICE processes more transparent”. Whilst this is indeed the case, note that NICE has had to pay 60% of costs resulting from the original Judicial Review (taxpayers’ money); and NICE will now have to pay the costs of the latest appeal.
Behind the scenes
Well, as always in cases like this, the situation would seem to be much more complex than presented. Certainly, submitting ‘confidential’ data to the Institute has been an issue for the industry for some time now and some companies remain unhappy about doing this. But the arrival of NICE has also been a boon to the various academic units around the UK who actually do the assessments, with this work providing considerable income for them. So it is a strong likelihood that the economic model used in this particular NICE appraisal was produced by an academic group who insisted on ‘commercial in confidence’ to give it ‘copyright’, and that was the reason why the model was locked. So if the ABPI is insisting on more transparency from NICE, then NICE justifiably has the right to insist on more transparency from companies in the data they are willing to release – clearly companies cannot have it both ways.
Other issues raised
There is another huge issue here regarding the evidence base in submissions to NICE. Clearly, the evidence set on drugs coming to the end of their patent life is largely based on registration data, and the data file provided for early NICE appraisals may just no longer be appropriate for the more sophisticated methods that NICE now uses. Reimbursement may not be recommended – sometimes for lack of good economic data, but also for other reasons. These reasons will likely include the use of drug outcome measures of questionable clinical significance, limitations in the quality of supporting clinical trials, lack of comparative trials with best available alternatives, and questionable clinical significance of effect size. In other words, evidence for licence is no longer enough for payers and, for pipeline drugs now in phase III trial planning, these areas will have to be addressed urgently.
Conclusions
So where are we now? Well, the bottom line is that the 2006 guidance on the Alzheimer’s disease drugs still stands and this particular story is most definitely set to continue. But the genie is now very much out of the bottle. NICE will have to expect more judicial reviews, not just from companies, but also possibly from patient groups. And it won’t just be NICE. Expect PCTs too to be challenged in the courts in future over decisions related to funding of drug provision at a local level, as PCTs will increasingly have to refuse to make (expensive) decisions over so-called ‘exceptional’ cases – they simply won’t have the money.