The Friday Blog: NICE, Knees and QALYs
We need to start with an apology and a thank you. The apology is to you all for not being able to publish what, for me at least, was an eagerly anticipated and very timely guest blog last week. We thought we had it all lined up but some late, unexpected logistical issues prevented it from going out. Fear not though, it is in the can and when it does arrive it will be every bit as relevant as it would have been. Now I have said that, you will know that the thank you is to our old friend Mark Chapman whose piece we did manage to get to you a fortnight ago. Mark is very upbeat about the prospects for our sector and tells me he believes that NICE is now finally taking HealthTech seriously. I would actually frame it slightly differently, and say NICE is finally taking HealthTech seriously again. It has long baffled me that there is such a pharma focus on appraisals and it was not always thus.
Some of you may be aware that Mark and I were colleagues at Medtronic for the first decade of this century, and I vividly remember being at a meeting together when we were challenged to estimate the proportion of the company’s UK revenue that came from products subject to full Technology Appraisal. Admittedly, it was a back of the envelope job, or, to be precise, a corner of the flipchart job, but we pretty quickly got to 80%. That sounds a bit bonkers today, but it was actually the case. My old friend and colleague David Hollingworth (who had Glenn Tilbrook play at his wedding) dined out for several years, quite literally all over the world, on the back of being responsible for the industry submission for Implantable Cardioverter Defibrillators. It was a significant piece of work which had considerable utility beyond the appraisal itself. Despite nervousness among manufacturers, NICE was able to understand that a technology that appeared to be costly, was also highly cost-effective. I was a serious student of health economics at the time and had been intrigued by the concept of number needed to treat (NNT), a statistic that gave you a sense of how much effort was required to deliver a tangible benefit. NNT is calculated by using the reciprocal of the absolute risk reduction for any given outcome from the data, and tells you how many people you need to treat to prevent one event. My last job in Pharma was selling statins, and I was doing so in the aftermath of the landmark 4S study. This was the publication that ended any debate about the value of lowering serum cholesterol and produced a sea change in clinical practice. It was the moment that statins achieved “put it in the water” status. I, for one, am glad they did, because I have been swallowing high doses of the stuff for 30 years without which, given my family history and heroic LDL reading in 1995, you may well not have the Friday blog. I spent a week at the time driving one of the study’s PIs around the North West of England delivering lectures to excited audiences of healthcare professionals. The doctor described how, when the results were presented at the European Society of Cardiology annual meeting, there was spontaneous applause and cheering, something that had not ever happened at the congress before, and probably not since. The NNT from that, practice changing study was in excess of 30. In comparison the NNT from the MADIT series of trials that provided the evidence base for ICDs was a small handful. I always regarded programmable active implantable devices as representing personalised medicine long before it was talked about in pharmaceuticals, it is a way that therapies can improve their cost effectiveness and something we will continue to see more of with the advent of omics and advances in companion diagnostics.
The submission also came in helpful a couple of years later after New Labour’s 2001 Shifting The Balance of Power paper heralded the introduction of a national tariff, designed to support other strands of reform such as choice and plurality of providers. As an industry we had a number of issues to address as a matter of urgency. The bespoke UK HRG system and associated reference costs were never designed as a payment system but that was how it was to be used with the inevitable glitches that followed. NHS cost collection was notoriously variable, and when, for example, I looked at the reference cost submissions for cardiac pacemaker implantation they ranged from £29 - £30,000. That is to say that of two hospitals using the same costing guidance, one said that it cost £29 and the other said it cost £30,000. To be fair to proponents of the system, the data were always trimmed and the argument that the average across all providers would probably be about right was not an unreasonable one. To use this as a payment system might be ok, as long as the underlying assumption that upwards of 70% of costs are fixed holds. If, however, you are implanting a device that costs tens of thousands of pounds in a procedure that, in skilled hands, takes less than 30 minutes, you need to think again. I heard the CEO of the now defunct Nuffield Orthopaedic Hospital in Oxford (more of this later) at a conference point out that he was doing more complex and more costly work than anyone else was either capable of or willing to do, and he was never going to get anywhere near tariff, so could somebody help him please. This is something policy makers had not considered, and it came down to industry to suggest a solution. And so high-cost device exclusions came into being. Even at this distance when I look at existing, current policy, I recognise the syntax, it largely having been written by Adrian Griffin of J&J, Stephen Hull who was with AdvaMed at the time and myself. The fact that we had all the costings laid out in the NICE submission helped us make the arguments, and, of course, the technology also went on to benefit from the guidance and associated funding mandate.
I had my own David Hollingworth moment when in 2000 Medtronic acquired Minimed (what goes around comes around). Within hours of the deal closing, I was talking to my new colleagues about what NICE did and how we needed to prepare for the upcoming appraisal of continuous insulin infusion therapy (pumps). It was a fun project, the work largely happening in cyberspace somewhere between California and Watford. That particular appraisal illustrated the difficulties in adoption of devices, even with constitutional implications of a positive appraisal determination. It was to be a number of years before utilisation rates got anywhere near those implicit in the guidance when it was published in 2003. As well as those technologies we had coronary artery stents, drug eluting stents, dual chamber pacemakers, cardiac resynchronisation therapy and spinal cord stimulators, so it is easy to see how we got to the 80% figure.
At the same time, I was also seeing things from the other side of the fence, serving for nine years from 2003 as a member of the NICE Technology Appraisal Advisory Committee. I was conflicted out of all those technologies, but there were others, notably CPAP machines for sleep apnoea. It was the only occasion I can remember that an expert witness caused the committee to do a complete 180. None of us really understood the condition and had interpreted the papers as suggesting that this was really a problem for middle-aged men who needed to cut down on their pork life and get some exercise as someone once said. The committee meeting, therefore, and in contrast to highly dramatic theatre it oftentimes was, turned into a bit of a damp squib. Towards the end, and really only out of politeness to the patient witness who had sat quietly through two hours of presentations and discussion without being called on, the Chair turned and asked if we could see an actual device. The gentleman opened a box and proceeded to place a mask over his head. Committee, even the more hard to impress, hawkish members, looked on incredulously. “You go to bed wearing that?” Suddenly we all got it. If someone was prepared to sleep in a contraption that resembled what you might imagine was used by the early pioneers of deep-sea diving, then this was a condition that needed to be taken seriously and patients given appropriate access to the technology.
But, for reasons I am still not sure about, the number of devices and diagnostics gradually dried up. Maybe all the big-ticket items with the sort of evidence base that NICE was capable of understanding back then were covered, but by the time I stood down all we seemed to look at were cancer drugs. This not only became a bit tiresome, but for a lay person also became really quite depressing. We were looking at people suffering from horrible conditions, usually through no fault of their own, with really bad prognoses and the treatments offering little in the way of increasing the quantity of life but having a marked impact in the opposite direction on its quality. One of the wiser economists on Committee once suggested that in such circumstances we should just tell people that we are sorry, but there is no good news here and just give them £30K (the incremental cost effectiveness ratio threshold) and tell them to enjoy what time they had left. How many QALYs, he wondered, would that buy you?
My sincere thanks to all of those who have enquired as to the welfare of The Doctor, who is now just over two weeks post total knee replacement. We were blessed to have been referred to the wonderful Royal Orthopaedic Hospital which has the benefit of being one of those cold elective centres that we need more of (see above) so were not worried about queues of ambulances outside filling up surgical beds with medical patients. Operationally things went very smoothly, although I began to get a real sense of what multi-disciplinary teams are all about. The surgeon was very happy with the results on X-ray but felt that was where his work as a technician stopped. Our journey, however, was only just starting and we were far more interested in pain management and mobility than what the imaging was showing. The real work will now be done by physios and the ROH’s community outreach people. It will be a long road.
Who would be a sports fan eh? Especially at the moment if your sport is rugby union football and your team is England. For months now assorted pundits have been telling us that England has the world’s greatest rugby team, and the current Six Nations Championship will just represent more serene progress towards it lifting the World Cup next year. But things are not going to plan. To be honest I do not really know what is going on. First up, England hammered Wales, France hammered Ireland and Italy beat a hapless Scotland. Next, Scotland hammered England, France hammered Wales and Ireland squeezed past Italy in a game that really should have ended in a draw. Last time out Ireland hammered England, France hammered Italy and Wales would have beaten Scotland but for one of the worst schoolboy howlers I have ever seen in a televised game, let alone an international. If you did not see it, do not follow these things, or just want to offer some advice to the young ones, in a game of rugby when you know your opponents are about to kick the ball back to you, what you should not do, ever, in any circumstances is turn your back on them. Even, or perhaps especially, if you are the grandson of one of the greatest sportsmen Britain has ever produced. Obviously the kick had to be accurate enough to exploit Welsh doziness, and Scotland’s mercurial fly half, Finn Russell, landed it on the proverbial sixpence such that Darcy Graham gathered without breaking stride and scored. What will be most galling for Welsh coaches and supporters alike is that this should not have been unexpected. It is a well-rehearsed tactic Russell has executed more than once before and on the telly. The Welsh players, including the twice removed Botham, would have been shown the footage and put on alert. It was a moment in which Welsh heads did not just drop, they went completely. It was hard not to feel for the Welsh, for much of the game it looked like they would pull off an unexpected victory. Still, Wales did look a bit more like Wales again, and we may have finally seen some green shoots, although in the final two games they will face a considerably more physical challenge than was posed by the Scots. Scotland, despite back-to-back successes will be aware that their greatest tests are yet to come. And as for England? Well, a week off to lick wounds, regroup and regather will not be unwelcome before embarking on what looks like a mini-Grand Tour. First stop is Rome. Italy has never beaten England, so sooner or later it will happen for the first time. I can tell you now that an awful lot of English fans have one of those horrible feelings that it will be sooner. After that it is Paris, where I think the best option will be to just sit back and enjoy the majesty of the French, who, at the moment, might actually be the world’s greatest rugby team.
More English self-loathing and ultimate disappointment is available at the Men’s T20 Cricket World Cup. England has, by common consent, been deeply unconvincing, but, and in what is a good habit to get into, has somehow managed to win five of the six games played so far, and, by dint of some rogue Sri Lankan weather, became the first team to qualify for the semi-finals. It is the hope that kills you. Always the hope. The hope here is in the old adage that in any tournament, in any sport, you want to produce your best at the end, not the start, and also that the short format is a great leveller. On any given day, any of the remaining teams could beat any of the others, and taking it to the last over with only one or two wickets remaining can only add to the experience.
I did manage some rather more stress-free recreation at the weekend by way of an outing with an old pal from my Pharma days, the Mancunian exile, Raj Patel. Raj and I have a couple of things in common that united in perfect harmony. We both enjoy a late, long, lazy lunch, and Raj’s partner Annabel being a pescatarian, brought into play Adam Stokes’s wonderful Oyster Club in Birmingham city centre. We were probably ridiculously ambitious on the food and beverage front, especially on a Sunday, and the quantity and quality of both were immense. To revisit our earlier theme, the amount of QALYs we generated trended towards the infinite. The other thing we share is what some people might regard, rather unkindly, as a questionable taste in music, and when the last of the pink Provence had been poured, we alighted to an evening of unashamed, joyous nostalgia at the Alexander Theatre, provided by Leicester’s finest. And you know what? At the moment, they might just be the world’s greatest Rock n’ Roll band.
