This article is published in today’s issue of Scottish Pharmacist, which you can read on line at http://content.yudu.com/libraryHtml/A42dhg/ScottishPharmacistVo/reader.html?page=12

In early 2016, former GP and NHS Fife medical director, Dr Brian Montgomery, was asked by the Scottish Government to conduct an independent review of access to new medicines in Scotland.

The Government published his report in December, with the Health Secretary saying his recommendations will now be taken forward.

Dr Montgomery had set out to answer two core questions. The first was whether the new approach adopted by SMC following a review in 2013 had led to an increase in access to end-of life, orphan and ultra-orphan medicines. The second question was how might systems and processes be improved further?

John Macgill asked Dr Montgomery what he found when he was conducting his review?

BM: What I found was evidence that access had indeed increased. There are two different perspectives when measuring access for patients. We were seeing a mix of the impact of the new approach adopted by SMC and also the impact of IPTRs [Individual Patient Treatment Requests] processed by individual health boards.

I found that SMC did appear to be saying yes more frequently than previously and certainly if you looked at it broadly across the three groups of medicines, the acceptance rate by SMC had gone up. However, if you then dug into that in a little more detail and you teased apart the ultra-orphans, it did look as though they had benefited less in that, relatively speaking, fewer of these were being accepted by SMC.

In the report I talk about ‘true ultra-orphans’ which are the drugs that are used in very very small numbers of patients. So, where the definition of an ultra-orphan, in Scottish terms, is one hundred patients or fewer, true ultra-orphans can be a handful of patients. If you looked at the acceptance rates for these, they had not increased at all. There were seven drugs that fell into this category in the period and, of the seven, only one was accepted for use by SMC. However, this is where the IPTRs kicked in, and we saw the acceptance rate of IPTRs for these medicines had increased to something like 90%. So, despite SMC saying no, access was still being achieved through IPTRs.

JM: Did you feel that there was a point where the SMC’s methodology just couldn’t cope because, with such small patient, and thus small trial, populations, SMC’s approach just didn’t fit?

BM: I think there was a suggestion of that but that is perhaps more of an issue if one looks ahead to what is probably coming in the future. I think the challenge that we had was less to do with SMC’s methodology and more to do with a one-size-fits-all approach, particularly in terms of trying to assess cost effectiveness.

JM: So, there is no fundamental issue with SMC’s QALY-based methodology?

BM: Except that you will remember that one of the requirements of the new approach was that the QALY was no longer the major consideration that it had been previously, so whereas previously the QALY was seen as a fairly hard threshold, the requirement of the new approach was that the QALY be taken out of the equation in terms of the end of life, orphan and ultra-orphans medicines.

JM: You talk about ‘true ultra-orphans’ for very small patient populations. Is this very rare conditions only, or do you include the cancers with sub groups whose genotype brings the population down to a very small number?

BM: It doesn’t yet include these but that was one of my concerns. If one looks at those very highly targeted medicines in the future, we could find a significantly greater number of medicines being classed as true ultra-orphans and the case being made for treating them differently.

JM: So your recommendation going forward is for these very rare conditions to be dealt with through a Peer Approved Clinical System separate from SMC?

BM: I wasn’t as specific as that. What I recommended was that SMC should still be asked to comment on these drugs in terms of clinical and cost effectiveness but SMC should no longer be the final decision-maker in terms of access. Certainly, a peer system could be one of the options. My concern was that if one looks at the situation we currently have, far less what we might have in the future, effectively we have a situation where 90% of SMC decisions in relation to these drugs were being overturned, which I don’t think is particularly good for anyone.

JM: How do we get more out of the same pot of money when we are seeing a continual process of innovation and new medicines coming through?

BM: I wouldn’t just restrict it to medicines. I would say ‘new technologies’ because there are plenty of other non-medicine technologies that are making increasing demands, offering huge potential and opportunities around treatment, but nonetheless at a cost that the current financial model is not going to be able to afford.

JM: Is this then about our willingness as a society to pay for new medicines, or not to pay, and perhaps to stop spending money on other things to afford them?

BM: I do have major concerns about sustainability going forward, and affordability going forward and this shouldn’t be a discussion on a medicine by medicine basis. There needs to be a much wider discussion about the health service that we as a population want and what we are prepared to afford because we now have the situation were the options available to us potentially cost much more than the resources available to us.

JM: What do you see as the role of pharmacy in this?

BM: Something I would expect of my pharmacy colleagues, indeed of wider professional colleagues, is that we are collectively using the resource that we have to absolutely the best effect. One of the things I have been conscious of over my career is how poor we can be in making sure that we are using finite resources to the maximum impact. As professions, we need to think about the cost effectiveness, not just the clinical effectiveness, of the interventions we are using.

I would also be looking to my pharmacy colleagues to be very active participants in some of the mechanisms that I think will have to come in. I am thinking here particularly of managed access schemes, particularly when one looks at the prospect of drugs coming to market earlier than previously and without necessarily the evidence base that we traditionally have expected. They’ll have a big role in the ongoing assessment of new medicines should SMC exercise the option of saying yes subject to ongoing evaluation.

JM: Do you feel that pharmacists have a role in the ownership of data?

BM: They absolutely have to and, not only that, they have to be very actively involved in determining what data and datasets are collected and used to what purpose. You’ll have got a sense from the report that one of the limitations was not so much the quality but the consistency of the data, which makes it very difficult to compile data across the health boards and then to be able to draw whole system conclusions. I think also that much of the data that is collected is, quite frankly, for management and administrative purposes. One of the questions we were unable to answer as part of the review was whether we can demonstrate that people have benefited from increased access not just whether they have got increased access.

JM: What do you hope will be the legacy of your review?

BM: I hope it provokes the kind of discussion where we really think long and hard about what it is we want to do with the resource that is available to us so that we get away from it being about single medicines or individuals.