MEDICINES REVIEW


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 per cent. 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 for 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 per cent 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 where 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 professionals, 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


BACKGROUND In late 2013, in response to public concern that too few medicines for end of life and rare conditions were being accepted for use in Scotland, the then Cabinet Secretary for Health and Wellbeing asked SMC to review its processes. As a result a package of changes was introduced by SMC to the way it assessed these drugs.


New definitions were introduced for end of life and ultra-orphan medicines to sit alongside the existing European ‘orphan’ designation. A Patient and Clinician


RECOMMENDATIONS Dr Brian Montgomery made a series of 28 recommenda- tions in his report, published by the Scottish Government on 14 December (www.gov.scot/Publica- tions/2016/12/9192). These included several around agreeing national approaches and definitions on formularies, data, chemotherapy and outcomes shar- ing. He asked SMC to look again at how and when it engages with patients and industry, and proposed that SMC might also be able to give a new medicine a conditional ‘yes’


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.


John Macgill is a director of Ettrickburn, a communications and government relations consultancy specialising in Scottish healthcare and lifesciences. www.ettrickburn.com •


Engagement (PACE) meeting was added as an option to the consideration of these medicines, with an overriding presumption that SMC would say ‘yes’ more often.


Meanwhile, for medicines where SMC had said ‘no’, or were yet to be considered by SMC, the Scottish Government said it expected the Individual Patient Treatment Request (IPTR) system to become more flexible and to stop considering ‘exceptionality’ when reaching decisions. Proposals were also put forward for IPTRs to be considered using a Peer Approved Clinical System (PACS).


to allow patients to receive it while more information is gathered ahead of a further reassessment. Acknowledging what he called ‘the affordability challenge’, Dr Mont- gomery recommended a review of how managed access schemes are agreed, and a greater role for NHS National Procurement in negotia- tions on the cost of new medicines. Dr Montgomery suggested that Scotland needs to have a wider discussion around how best ‘to take advantage of the opportunities af- forded by anticipated developments in the way that new medicines will be introduced in the future’.


SCOTTISH PHARMACIST - 13


Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64