Translating cancer research and innovation into policy and care: a panel discussion.

so good morning everyone a very warm welcome to the University of Leeds both for those of you who are here with us in person and also our live streaming audience um from across the UK we're delighted to have people from as far an authors Edinburgh and South and Southampton joining us for this webinar we're also delighted to to welcome Dr Owen Jackson who's director of policy at cancer research UK who's returning to Leeds and I'll let him explain what that means in a minute and I'm also joined by three Leeds academics so um I'd just like to start by saying this is a joint initiative put together through the Leeds Cancer Research Center working closely with policy leads and to thank Juliet jobson and Danielle battle for organizing this event as well as it taking place live we're also recording it so it could be shared with our academic community at a later date so we're really looking forward to this and having lots of time for discussion and interaction that before we get going I'd like to invite the panelists to introduce themselves thanks David um hopefully you can hear me um it's it's great to be back in Leeds as you say David I I was um uh leader to my Alum I am a leads alumni uh I was here uh between 2003 and 2010 um as a undergraduate and then a postgrad uh study in microbiology um and then uh left to join the civil service uh if you want to know more about that you can come along to my talk on careers in policy making this afternoon if you're interested um but uh the reason I'm here today is as you've said I'm now director of policy at cancer research UK who as many of you will know are the biggest independent funder of cancer research in the world um a real UK success story and an organization funding one and a half billion pounds of research over the next five years um so I'll I'll stop there but I know um you'll want me to say more about the co uk policy robot I'll stop laughing and hustle into others um Katie thanks David thank you Aaron um so I'm Katie Spencer I'm a clinical academic oncologist um and I'm based in the academic unit of Health economics from an academic point of view my research really focuses on improving cancer outcomes by combining the use of routine cancer data with strong understanding of Health economics and the constraints that we face within the Health Service to deliver the best outcomes we can I'm also particularly interested in the use of radiotherapy in advanced cancer and that interest in radiotherapy also focuses in my role as NHS England's clinical lead for radiotherapy data I think is good to go all right everyone I'm David Meese I'm professor of Health economics at University of Leeds um most of my role involves trying to establish the value for money of new treatments and interventions for use in the NHS I work on mainly on oncology and hematology studies and and some surgery projects as well I sit on a nice technology appraisal committee which means I have some understanding of the approval process for new medicines uh in England and Wales thank you David thank you so I'm David Cairns I'm a professor of clinical trials research and I trained as a statistician I'm a leads graduate in fact mathematics in the year 2000 it's my misspenter youth I'm also deputy director of the Leeds cancer research UK CTU at the Leeds Institutes of clinical trials research and I designed setup coordinate and analyze academic clinical trials many funded by cancer research UK or other Charities and bodies funded by the Department of Health in some cases in partnership with industry an academic clinical trials in my opinion at least my bread and butter they're always conceived to change practice so thank you very much I think we've got a fantastic panel very well equipped to talk about the topic today and just to remind you that's around translating cancer research and Innovation into policy and care um but before we actually get into that in too much detail um I'd like to perhaps ask going to to set the scene a little bit about cancer research UK policy and some of the exciting activities are taking place um there's a Manifesto that is due to come out soon and I think all of our audience will be very keen to get your overall perspectives of it thanks David yeah so uh as I mentioned cancer research UK are a real UK success story we're very unique I think um as an organization that that focuses all the way through from basic research all the way through to better outcomes for patients and historically I think in policy terms cancer research UK was seen as an organization that knew a lot about Cancer Care so whenever government had a cancer problem cancer research UK and you know a few others often are the big charities are on the top of that call sheet when when ministers and and government officials are thinking God how do we deal with this big cancer problem we've got now the chef the Charities trying to make at the moment is frankly we're a research organization that happens to focus on cancer so as I mentioned we we fund one and a half billion pounds of research over five years on all aspects of of cancer research all the way from very very basic understanding of Cell Biology all the way through to new treatments in the clinic and we're fairly unique in that we we we are we have knowledge and experience at every every stage of that so as well as being a funder and running institutes like um or participating initiatives like The Institute in Leeds um we also uh are involved directly involved in the translation of this Research into clinical practice and into commercial uh commercial science so through something called cancer research Horizons which is a a a an innovation engine that the charity owns we use that to really drive all of that new innovation that our research is funding into new clinical practice and into new commercial science so we've got a fairly broad set of experience and one of the messages we're trying to get across to government and to others is actually if you're interested in doing good research in the UK you know one of the challenges that researchers face we've got direct experience of that we you know we support 4 000 clinicians scientists across the whole of the UK and every all four of the UK Nations and we can help so so my job within the charity is to be that interface between the charity and our objectives uh ultimately driven by wanting to be cancer sooner and all uh all aspects of policy making so everything from national uh and international policy I have a responsibility for our small but perfectly formed Global policy team that's our for instance in the next few months going to the tobacco the tobacco cop so much like there's a cop on climate there's a cop on Tobacco Control it's happening in Panama this year to try and drive Global change about how we deal with the scourge of tobacco and tobacco smoking um all the way through National politics so I have a big team that folks in Westminster all the way through subnational politics so I've got teams in or other all four of the constituent nations of the UK I have teams in Belfast in in Edinburgh and in Cardiff doing a very exactly the same job um and then also you know as well as just doing the actual policy making interaction we need to develop we need to think about what we're going to say so I have a team that really turn our knowledge and understanding from the evidence often driven by funding research that we funded ourselves into what does that mean for policymakers what we want to what do we want to see change and as David mentioned a big thing that we're doing this year a very significant event for us this year will be the publication of what we are calling our Manifesto for cancer research and care so this is cruc's perspective on what's the long-term shifts that we need to see in the UK to really drive a change in cancer outcomes and you know the name is deliberately chosen cancer research and care we're going to talk as much about the challenges for research as we are about the challenges for care um and as of about you know we at the end of November this year we're going to put out our view of that what's the long-term changes that we need and the reason we're doing it now is obviously the Manifesto It's Manifesto season we're about to go into a general election year one of the few times in the political cycle where people are willing to think long term um so uh this is a big moment for us for it to influence and and we've spent the last nine months or so talking listening engaging with experts you know all the way from as I say from academics through clinicians patient patient Representatives um and a significant amount of our internal expertise as well to put together that full package um so we can talk and talk a bit more about that as we go if if that's interesting that's um really helpful and insightful and um I think it's great to hear that there is a Manifesto being led by cancer research UK um I wondered if you like comment on the lack of a of a Manifesto for cancer because at the moment um our audience may or may not be aware we don't have a current long-term cancer plan sure um one thing we know because we funded Research into it uh through the international accounts benchmarking partnership lcbp we know that countries that have long-term strategies backed up with political ambition backed up with funding are the countries that have seen a real change in cancer outcomes in the last few years so if you look at Denmark if you look at Australia two really good examples of countries that have had ambition a long-term plan and a clear way of funding that long-term plan and they've seen huge improvements in in cancer outcomes the UK has seen improvements in cancer outcomes across the board we are getting better and that's fantastic for patients you know it does cancer survival is increasing all the time and and that's fantastic challenges that are com you know our peers if you like are improving faster than we are so uh the lack of a long-term cancer strategy that multiple got three three governments in succession told us was going to be published uh um and and the most and the current government have have decided not to pursue and not to publish is a challenge so we don't have a long-term strategy currently we don't have a clear pathway to achieve the Ambitions the government has set and we don't have dedicated funding to deliver that strategy because the strategy doesn't exist so the manifesto is if you like see our UK's view of some of those things that would need to be taken forward as long-term policies if we're going to see that kind of improvement that that frankly are our peers have seen and that the cancer patients in the UK should expect to see great thank you very much that's a very very clear and important explanation for all of us so I think we'll we'll now move to talk a little bit more about the Translating research and Innovation into policy and care so I'm conscious that we have a a great diverse audience both in person and through the web streaming so Casey I might ask you just to very briefly give us a sort of an overview particularly for those who have limited cancer experience about what we're talking about here thank you David so I think the translational pathway really is about everything that goes from the point of basic scientific discovery and underst using the ideas the insights and the outputs of that basic scientific discovery to really move to delivering improvements in cancer outcomes so that's about not only getting those scientific discoveries into drug development it's about doing the trials for those drugs getting those drugs approved and reimbursed and then getting them to patients and getting to every patient that needs them um and actually there's been a lot of discussion previously about gaps in that translational pathway and there are two major gaps that have been identified and so the first translational Gap is talked about as being between that understanding of the basic scientific problem and getting a drug that solves it produced and through trials and so that's that's the first translational Gap the second translational Gap is at least as important but sometimes possibly a little more challenging to understand and that's where we go from having a successful clinical trial to actually delivering benefit to our patients across the country and that's about dissemination it's about reimbursement it's about inequalities in care and it's about implementation science and getting Technologies into into practice and this isn't just about novel pharmaceutical agents either we have to recognize the translational pathway has a role across the whole of a patient's cancer Journey so that might be about talking with patients and education and Behavioral Science to understand lifestyle factors and health seeking behaviors it may be about ensuring that the screening techniques that we have are optimized and deliver the outcomes we want that we do the targeted tests that are needed to drive targeted therapies and that we Implement technology whether that be Pharmaceuticals or radiotherapy artificial intelligence even to really start to drive improvements in care for all of our patients so you can see that that translational pathway affects patients all the way through and actually covers quite a massive range of researchers Owen alluded to with both research and Care being highly relevant thank you that's that's really clear and sets the scene very nicely and our three leads panelists all bring complementary and different expertise to actually go into this in a in a little bit more detail so David perhaps I could come to you and get you to talk a little bit from your own uh expertise and clinical trials thank you David and thank you to Katie for setting the scene so well so my interests are really um in clinical trials and how do we translate new drug treatments into the clinic so pardon me in a sense randomized controlled trials Remain the gold standard evidence for new drug treatments pharmaceutical companies set up their portfolio of registration studies aiming to get their product to Regulators so in the US the FDA in Europe the ENA and this is a very carefully constructed program with a purpose of getting a license for their product in a particular indication and alongside this they have they often have a program of investigator-initiated studies so this is a space for academics to initiate research with the company where you might be provided with the drug provided with funds to do the drug to do the drug trial perhaps um and really this is the place where most of the trials that I've worked on um where they sit their investigator initiated studies potentially in in conjunction with Charities but really what is their purpose because they're not aiming to provide the um the company with a license they're there to provide um complementary evidence whether that be evidence in a particular country in particular combinations of drugs that the primary registration studies aren't focusing on or perhaps different modes of delivery may be instead of infusions fast infusions or subcut subcutaneous infusions they're there to support the package of evidence to help move the product to the to um to license and to reimbursement in different countries so I'm going to tell you about um a particular series of clinical trials that we've conducted in Leeds um the where we have something like a 30-year experience of doing these so these are academic trials in multiple myeloma multiple myeloma is a bone marrow cancer it's the second most common hematological malignancy there's around 5000 new cases each year um the UK in the UK and around the world in the last 30 years outcomes have been transformed just like Owen was saying um 20 years ago five-year survival was something like 20 now five year survival is something like 60 percent and there's new trials with results better than that likely to come out very soon um the studies the academic studies in the UK have unimaginatively been numbered they don't have names and I'll begin to talk about uh myeloma sevens this was a study conducted from Leeds around 20 years ago it was a study that showed that high-dose chemotherapy and stem cell support really improved overall survival so the first big clinical conference I ever went to in 2016 the American Society of hematology Congress I think it was in San Diego I went to an educational session on the first day and as a fairly young academic new to trials really it was so exciting to see when they were breaking down the building blocks of why they treat patients using the evidence from clinical trials that I knew about clinical trials that we'd run in the UK to help formulate the evidence required for the treatment pathway um myeloma nine that was a well it followed myeloma seven um but missed out eight so that showed the transformative use of bisphosphonates in improving bone health and improving outcomes as well myeloma 10 showed that Titus chemotherapy and Salvage stem cell support improved overall survival in relapsed patients there's a there's a real series of successes here which we're lucky to be involved in which have been a pleasure to be involved in you look at nice guideline number 35 on diagnosis and management of myeloma all of these studies are evidenced in constructing the UK and treatment pathway and it's not just the UK treatment pathway you look at the esmo guidelines the European Society of medical oncology the imwg the international myeloma working group all of these guidelines evidence these trials as the reasons why patients are treated today with these kinds of treatments however the study that I'd like to tell you about the most is one which is um has played a central role in my career it's really important to me it's the 11th myeloma trial myeloma 11. um it was a study funded by cruk in 2009 and it recruited from 2010 to 2016. it's the largest myeloma clinical trial conducted in the world ever so far it has nearly four and a half thousand patients in it um it recruited something like a fifth that newly diagnosed patients in the UK each year in its most um prolific years it was a trial that the community really built um bought into and really took took to their heart and put patience into it it had a complex adaptive design but that's irrelevant today um it tests the use of lenolidomide treatment and next generation's thalidamind in various stages of the treatment pathway for newly diagnosed patients so the size of the trial meant that it really attracted the close attention of the company manufacturing the drug we were working with this company they were providing us with discounted drug they were providing with with some funds to support the coordination of the study and we were all called to a meeting in London the chief investigator was at the icr um at the time so we went to the offices on the Fulham Road the icr the ICL kind of annex on the Fulham road we took the train to lead from Leeds to London the train was late um that's not completely unusual we took the uh underground across town to South Kensington were greeted by this very strange phenomenon of the chief investigator out in the rain looking for us so you can tell that something unusual was happening and he was quite agitated so we were all taken to this room this room which was too small for a meeting with the company and the academic trial group was outnumbered something like four to one by the company Representatives they understood the value of this data set right from the beginning um so we entered into negotiations about them potentially using this data set for submissions to Regulators so the FDA and the in the EMA in particular in particular and really it was a fascinating insight into how industry runs their trials how they consider academic clinical trials and how many questions that they had for us they almost audited everything we did to ensure that it would be acceptable um to them and fit the processes that they would normally expect in an industrial sorry an industry clinical trial um now this kind of request for use of data um it's not common from an academic clinical trial I want to talk to a colleague of yours in CI UK and they told me maybe once or twice a decade a cruk funded clinical trial would find its space find its way into this kind of discussion and there's many interesting things happened um this has a value of course and crook at the time at least delegated the responsibility for understanding the value of this data set to the university something that doesn't happen to the university every day um so it was so exciting there were so many face-to-face meetings over many many days with the company understanding everything thrashing things out um and in the end they decided that our timelines did not match their timelines the exclusivity of the drug the patent life that's really that really guides many decisions doesn't it so the company decided that they did not want to wait to use this data set to um include in there including their license rather they decided that they'd like to use the data set to help support Health technology appraisals like David has no doubt going to talk about so they provided us with Resources with support to make sure that the data set was published in a timely manner that it was of a high quality and indeed in the nice technology appraisal that approved lenolidmate maintenance post stem cell transplant the myeloma 11 data set is one of the two primary sources of data it was discussed at Great length um so how we did that was it was interesting that it's important to keep a distance between Academia and Industry and a third-party Consultants who actually did some of the work in the middle and it was also a really fascinating way to work to show a level of Independence that was required is now available to patients after stem cell transplant in the UK and so I say the UK so that doesn't just mean nice David that means the Scottish medicines Consortium as well and the data was used there the same evidence was used around the world to provide evidence in health technology appraisals the ones that I remember for no particular reason are in Denmark and in New Zealand because I had some conversations about that um so now patients have access to this treatment that improves survival in countries all around the world um thanks to a academic trial funded by cruc and sponsored by the University of Leeds and so far it's the most exciting things are happening in my career and and I like to think of it as an example for other things that can happen in academic clinical trials so David thank you for that that fascinating uh story um oh and I don't know if there's any comments you want to make at this point briefly well just I I think um uh it shows something it reveals something about about um science that I think is poorly understood with the policy community and that is how science gets done uh you because you you identified a few things there I think that are really important that we that I as a as a former policy Maker Now a policy influencer maybe um and and as a science as a former scientist um kind of see which is science takes a long time um and science is an inherently good science Global you know World leading Sciences inherently global so you know the fact that you talked about the collaborations that you were doing the fact that you talked about um you know how this work has influenced the role you know practicing other countries and those are two really key things that for a policy audience CR UK talks about a lot you know um Richard Dole proved the link between smoking and cancer in 1950 and it took until 2002 I think to get uh to get smoking ban into the UK I was actually in the old bar the day after the smoking ban came in and I remember uh the smell as much something else um but uh so so that that pace and that time that good science takes combined with the time that policy making can sometimes take I think is just something is just something I've reflected thank you very much so David means we're going to come to you next because um David Cairns is very nicely um sort of set out an Exemplar and mentioned on a couple of occasions the the nice technology appraisal committee which you sit on so it'd be really great to have your perspectives as to how that committee works and actually how those decisions are made yeah sure um and and thanks for that uh story I wasn't aware of that uh David very very nice story um yeah so I sat on a nice uh technology appraisal committee there were four of these they meet monthly to appraise uh new treatments so any new treatment or medicine that's going to be used routinely in the NHS has to go through what's called a nice technology appraisal process and this is where the um the producer of the the medicine submits evidence to nice and independent external assessment groups and this evidence in the form of clinical trial results um economic health economic results budget impact systematic reviews evidence synthesis that kind of thing in a huge dossier maybe six seven hundred pages this is reviewed by nice and the external assessment groups they're usually uh academic centers that do this and then this evidence is provided to the uh the committee and it's discussed their committee meeting which includes obviously the the committee itself nice the company other stakeholders including patients clinicians and NHS England and the role of of that meeting really is to try and decide what the most plausible estimate is of cost Effectiveness so is this value for money which bits of data which assumptions do we accept and therefore what is an acceptable price for this medicine and um the key metric of this uh this analysis is cost per quality adjusted life here okay so how much does it take how much does it cost to produce an additional quality I'm using this new medicine compared to standard of care whatever that is and if it's below a certain threshold twenty thousand pounds then that medicine can be recommended for routine use on the NHS and now importantly the technology appraisal process comes with a funding mandate so if we recommend a treatment in those committees it has to be available for patients within three months of that the publication of that document because that's not the same for other HDA processes that nice have that cover Diagnostics and med tech for example so there's a really important funding mandate leverage with these these committee meetings it's obviously they're a big deal for companies because they don't just influence policy in in England and Wales these results are quite often taken up by other jurisdictions and and internationally as well and they can quite often be price setting for the company um so it's a big deal uh nice being around for over 20 years now and in that time they've conducted over 800 appraisals uh phenomenal number and it's increasing every year about half of those are in cancer and the approval rate is hovering around 80 probably just below 80 for for cancer treatments and when I say approval there are various decisions that can be made it's a straight yes uh or it's uh we think it could be cost effective but actually we want to collect more information more data on this so we uh think it could be eligible for the cancer drug Fund in which case NHS England will negotiate on price patients will have access access to the treatment in the normal way but there will be a requirement for the company to collect further Evidence which could be additional follow-up on the trials they've got going and it will be a review date set to look at the new evidence and decide whether actually should be moved into routine commissioning and a review uh time period is usually between two and five years uh yeah so I think that's that's probably all I need to say at a moment on that that's that's okay that's very very helpful what I think we'll come back to some of the points that that you made a bit later but come to Katie next and uh get you to talk a little bit from a slightly different perspective rather than drugs to talk about radiotherapy thank you David um thank you David that's a really nice outline of the pathway to reimbursement Pharmaceuticals and I think it's fair to say that the pathway to implementation of radiotherapy is much less linear um and often less well-defined um and I think we can we can think about it in a number of different ways um and to a degree it is actually about it's effectiven us underpinning and we will often have small changes in radiotherapy techniques and technologies that maybe are not actually desperately costly to implement it's a slight tweak to an existing linear accelerator added bit of kit that actually changes the way that we deliver treatment improves the accuracy of treatment for example could increase the dose of the tumor and reduce the dose to organs at risk and surrounding tissues and if that's a small change it may be that actually it works its way into practice in a sort of Fairly informal way over a period of time we then have changes that are maybe a little bit bigger maybe it's a novel indication for radiotherapy um and actually in that circumstance the bar for the evidence is set a little bit higher but sometimes that can be quite challenging so if we think about stereotactic radiotherapy for oligom metastases that's been implemented in the NHS but actually one of the challenges is that you've got to implement the technology before you can test it so unlike a pharmaceutical where you just take a novel drug into the clinic in radiotherapy you've got to get the systems and the processes up and running safely to deliver these treatments to then be able to deliver the clinical trial but once you've got that up and running the desire to deliver the clinical trial is somewhat diminished and so we have a really complex situation where we really have to work out how we're going to ensure that we get the evidence we need to support these treatments and so for something like stereotactic radiotherapy what happened was there was an acknowledgment that early data suggested this was beneficial but actually we needed more evidence and so it was implemented but with ongoing evaluation an ongoing collection of evidence to support that and its future expansion across the country and so that's really really important in terms of getting some of our photon-based treatments into practice I think when we move upper gear again we move into really big changes and that's where we're talking about proton beam therapy and the Mr linac where actually the cost the incremental cost of delivering these treatments is is huge compared to these changes in photon based treatments and the evidential requirements therefore go up as well and actually now the UK is delivering globally leading Trials of proton beam therapy with randomized studies aiming to properly Define well we think based on everything we know about physics and the way radiation behaves in the human body we think these treatments will be beneficial but let's demonstrate it let's show it and prove that the increased cost of these treatments just is justified by the benefits we see for our patients so it's fair to say that it's it's not as simple and I think the other thing I would add is that even once we've got all of that evidence even in photon beam treatments these are geographically constrained so there are 50 centers providing radiotherapy in England alone so patients have to travel to receive radiotherapy if we're talking about something like stereotactic radiotherapy maybe you start with 10 centers delivering that treatment and with protons with two centers delivering it across the UK so there's really important questions to be asked there about the equity of access that patients have and ensuring that all patients are able to access these treatments as soon as we are confident that they are offering good value for us and that they are appropriate for delivery in the NHS perfect so um we'll come back to some of those challenges so we've heard from the two Davids and Katie about the perspectives of both drugs and radiotherapy and how they ultimately come into clinical guidelines which are nice which we'll talk about a bit more but there's another side to policy decision which is in government and Owen you've got a lot of experience of having worked in in that space so I wondered if you could give us some insights in how the wheels and government turn with respect to to these side of things sure uh yes uh let's have a go at that then um I think um both Katie and David's explanation there is a very good segue actually because I think what we heard there are the different sides of of changing clinical policy and practice which is sometimes it's very clear what the Methodist and what the approach needed is to change from point A to point B you know you need to go through regulatory steps and there and there is a clear outcome and and then the other side of that which is a far Messier uh picture of changing practice involving changing Minds changing a bit of policy getting lots of people involved um and uh uh when I try and think about policy making and my experience in government and kind of thinking about you know what what we what we're talking about today you know turning uh uh policy and practices into better accounts care and cancer research um it's about complexity and um uh I was struggling to kind of conceptualize this last night in fact last night I was thinking how am I going to explain this and I found a really good article by a guy called Dr Paul Kenny who's a academic at University of Sterling which you can find online it's um it was the it's the first time I've seen written down a good summary of of how my experience was like in government which is uh there is no simple policy cycle there is no there is anyone who tries to tell you there is they can explain the policy making process to you is lying to you it's snake oil because what we're talking about here almost always when we're talking about national government policy making we're not talking about simple stuff we're talking about stuff that hasn't been resolved up until this point so it's inherently going to be complex and it's complex because it's involving people so it's emergently complex you know it's it's kind of it's fractal every you know every every additional person's perspective that gets involved it gets inherently more complicated and what Paul's article does I think is is it describes this in quite a nice way and and his article says let's have some images of what policy making looks like and and one of those images is a rocket launch and I think you know to your point Katie it's that point about we can't launch the rocket until all the 58 or probably hundreds of systems that feed into that have got to a point where they're comfortable so that's a bit like policy making frankly um uh you will have a hundred different considerations that are all pulling in slightly different directions that you're trying as you know as the civil servant advising the minister you're trying to kind of trade off it's not always as you know frankly your brain is not able to deal with all of that so you do end up collapsing complexity in order to make decisions um and the other one I liked is is punctured Continuum and I kind of punctured Continuum model which I've described in the past as slow slow fast so policy making like the Richard doll example you know we knew in 1950 that smoking was likely to be causing cancer and it took us until 2002 to get legislation through and in fact I'm sure if you looked at that time scale 1950 to 1995 was probably nothing or very very slow progress in terms of building up the evidence you know informing people getting people on side about understanding the risks the actual process of the policy change was very fast it would have probably been a year it will have been very intense for everybody involved um and that was my experience of policy making an example in a totally different sphere is I was working in the Northern Ireland office um during uh what we were calling then the storm and house talks so this was um big talks in Northern Ireland about in about 2014 I think to resolve the then current impasse around big issues around dealing with the trouble at the Legacy of the troubles and some issues around Community cohesion and uh in about two weeks we had to take the work of years and years of academics and civil society and and politicians and policy makers and turn that into a talks process that ended up with an agreement thankfully the day before Christmas Eve um and that was my experience it's it's and I think getting getting getting that understanding is you can have all the evidence as to why change is needed and it can still take a long time a lot longer than you would expect to get that change delivered and this links to my final point which is that um uh policy making is a bit like committing a crime you need means you need motive and you need opportunity and the one everyone forgets about is opportunity because you can have all the means of motive you like you can have all the evidence you can have all the kind of incentives in place but until there is a political opportunity to get something big changed it won't happen as a civil servant I would be constantly being approached by organizations and people saying you here's this great idea and I'd be like yep that is a race idea I'll put it on the pilot rate ideas um that we're not going to do um because government never has the bandwidth to do everything and so it's all about choosing what you're going to do and the opportunity when it arises is often something you can't control we try you know see our UK one of my one of my roles really is to try and influence the the discourse to the point where we do have an opportunity but you can't control that um a case in point I think was you know covid was the biggest opportunity you like when it comes to the voice and the role of science and research in policy making no one predicted it but it changed the dynamic so you can't plan for those opportunities you just need to be ready for them when they occur and when they do occur you've got to be able to kind of run it in Full Steam um so yes I think I'd say it's it's quite complicated it's you know anyone who tries to sell you it's simple is lying to you it's slow slow and then it's fast and don't forget about opportunity in the in the crime that is changing policy making brilliant thank you very much so um going back to you David Cairns um picking up Owen's point of slow slow fast you were talking about time scales for the trials which um have been very influential but may have more of a slow than a fast so how do we change to fast fast fast well that's a very good question I think um opportunity has really resonated with me actually because you you need whether there's coincidences or whether the timing's just right opportunity is so important so in the example that I just gave the Leonard I don't you thought I would have learned to have said the lenolidomide example um the patent expired uh last year so now you can get generic lenolidomide so the company had to be convinced that nice appraisal in 2021 that they were going to um they were going to I'm going to simplify it and say make enough money to make it worth their while to go through that process so if they hadn't done that you said David I think the companies often lead this process of um of um getting the drug into the indication um and looking for reimbursement if the company hadn't pushed that forward um then it wouldn't it wouldn't have happened and this drug that probably costs 20 times less than it did when the company and put the nice appraisal through if that hadn't happened it would have been much more difficult now for patients to access it even though there's a strong body of evidence that it's um that it extends life so opportunity was really necessary there and I can think of other um kind of lost opportunities the final analysis of this study is coming up um it's an overall survival analysis and it's for a agent made by another company that company decided five or six years ago that um that they weren't going to support that agent getting a license in the particular space we're testing so all the TR although the trial is going on there's a good chance it'll show that it extends life again there's very little chance of that changing practice in the UK or anywhere else now I looked at the technology appraisal guidance um last night preparing for this as well and it does say that Healthcare professionals anybody can put um can put proposals forward for technology appraisal but I think that's outside of the skill set there isn't necessarily the support for people to do that without a pharmaceutical company there to provide the opportunity so David um you um through the nice appraisal process I think many people internationally regard nice um in a very positive light and being very rigorous in its approaches um I was taken by a recent study that was published a few months ago through led by the lse I think and they actually looked at um the how countries with similar but not necessarily the same appraisal processes evaluated drugs and marginal benefit and the findings were quite striking so they were funded in Germany in 83 percent of cases Japan in 67 France and 50 Italy and 39 and the UK and Australia in 17 of cases this was in very marginal benefit approaches I suppose the question is how how standardized is the decision-making in other countries is this marginal example um not typical of how decision making is taking place across the world do you think nice has got that balance right through his experience over the last 20 years I I mean I'm an independent uh Committee Member so you know I don't work for nice um uh but I I do do feel that the framework that I have in place uh adds value and I think is important uh it's a really important um negotiating strategy with companies but obviously you've got to get straight and right balance in terms of providing the incentives for for companies you know saying yes enough but also making sure there's value for the patients because every time we we find a treatment that is not cost effective that in theory reduces the overall health of the population I think the the framework that nice have is being exported uh globally nice habit Department um that works internationally with with lots of different countries showing them how this framework um should work and and I can see that that process is is probably just going to expand to be honest um and and even in in the U.S uh we were

talking earlier about this there is no formal um in law acknowledgment of you know the cost per quality uh those kind of considerations aren't made but there is an organization called Isa which does independent reviews of Economic and clinical evidence and they produce reports which are used um occasionally by decision makers in the the negotiation process so um I think that that process not going to go away um and budgets are constrained choices need to be made and the quality threshold that you you mentioned the twenty thousand pounds is that fairly static has that changed over the years or or not no it's it's it the evidence the only evidence underpinning it is pretty well there isn't really any um but what it's supposed to indicate is how efficient the healthcare system is at generating a full year of health basically there have been some attempts to quantify that and and those attempts have showed actually it's probably somewhere below twenty thousand pounds per quality but it hasn't moved it's unlikely to despite inflation um what what should happen in theory is that the more uh efficient treatments that you fund then the more efficient the healthcare system is at producing quality so if anything you can argue that that threshold should come down but that's uh you know politically I think that that's that's just not not an option right thank you very much we'll come to Katie um I'd just like to mention to the live stream audience we're going to come to q a in in a minute so if you have any questions for the panel please put them into the chat um and then they will be put to us by Danny or Juliet um Katie you mentioned about proton beam therapy very topical sitting here today because many of you may not be aware that William Bragg was a Cavendish professor of physics here in Leeds and there's a blue plaque on the Parkinson building commemorating that and not only did it discover X-ray crystallography and won the Nobel Prize with his son for it he also was determining the future which is now being tested so perhaps you could tell our audience very briefly what proton beam radiotherapy is and why clinical trials are so important thank you David so Photon beam radiotherapy is delivered with electromagnetic radiation and photons um and if you've seen an extra ever be seen an x-ray and you can see that photons pass through the human body to some extent um and that's a good thing in many ways it means that radiotherapy with photons can treat organs that are deep within the body and increasingly we're able to really really Target that however because they pass all the way through inevitably some of the energy that the photon carries and that treats the cancer gets spread around in the tissues around the cancer and that's damaging to those tissues and is the reason we see late effects following radiotherapy and what protons do is a little bit different these are bigger particles and actually when they interact with matter instead of passing all the way through depending on their energy they will stop and they will deposit their energy where they stop and so if we can work out where they're going to stop and make sure that aligns with where the tumor is we can try and make sure that the dose delivered to the tumor is higher and that the dose delivered to the surrounding organs is is reduced so clearly really beneficial both for tumor control and late effects so that's all the theory which is great um and and and well evidence this is physics however we don't really know that those benefits that we sort of see dosymmetrically and that we can we can model it with a computer are actually seen in real life and so that's where Trials come in for radiotherapy proton beam therapy and there are multiple trials now running um the the trial looking at the use of proton beam therapy for head and neck cancers for oropharyngeal Cancers and that's I think nearly nearly finished accrual now um there's also trials looking at the use of proton beam therapy for brain tumors and breast cancer and there's an increasing portfolio under development now demonstrating the evidence base to to see whether we actually we can start rolling out the proton treatments more widely because at the moment the routine indications are in the children's pediatric setting where we know that those late effects are so so important and actually the ex there's an Acceptance in the clinical community and the policy community that actually the benefit the lifelong benefit we see for kids in that context justifies the increased expensive protons in the adult setting we now need to demonstrate whether that benefit is justifying the cost there too so that's a very nice explanation and I think um it's a sort of parallel example to nice as to how the UK have taken the evaluation of new technology very seriously when it's substantially more expensive so the two proton beam facilities in Manchester and at UCL serving the NHS and actually through the business case that was developed for protons there was capacity and excess treatment costs built into that to actually generate the clinical trial evidence to determine whether proton beams should be more widely used in the UK that's in contrast to a situation where in the U.S many hospitals set up their own private facilities many of those are now closing um and actually one of the key manufacturers has stopped manufacturing proton beam radiotherapy machines until the evidence comes and that will be predominantly from northern Europe such as UK and Denmark so I think we've had a very very nice and varied discussion um and I can come back to the panelists for some more comments but I'd just like to ask of anyone in the audience would like to ask a question and to check whether we have any um any questions from our web audience so firstly is anyone in the audience like to ask or comment uh to loan your microphone David so interesting thank you I have lots of questions so but I'll do that later don't worry I guess just following on from the from the proton discussion that you just had um and it we've established that we need the the clinical trials um research to build that evidence base but coming back to what we said at the beginning that part of the reason for policy is to try to increase access to treatments for patients and we know with protons that at the moment there's only two centers in the country and through those trials we're trying to address some of the access issues but I'm just wondering in the in the pathway of clinical trials research where do you think that access questions come in if we think if you're thinking ahead to policy and how we we provide access to a wider audience should I take that please thanks Sarah I mean I think that's a really really important question and clearly there is a there is a policy priority to try and ensure that access to trials is Equitable across the country I think that's hugely important but then at the same time actually when we're talking about something where there's an uncertain benefit and there is a cost to patients for traveling actually we can't expect patients necessarily all to want to undergo that however I think it's absolutely crucial that we use those trials to understand patients decision making and their willingness to travel for treatment because whilst in the uncertain setting of a clinical trial with uncertain benefits it might be justified to say well okay that's a decision that we've made under uncertainty if we know that there is a benefit we need to be able to address inequalities in care and only by understanding those inequalities and the reasons why some patients don't get the same levels of access can we really start to address it so I think that for me is the key thing with with accessing trials just to reinforce that point it's it's an area that um at click we're we're increasingly thinking about so um we've published a report uh during the summer on inequalities and cancer outcomes in Wales and you know that was on the back of a lot of work we did and interviews we did with the communities in Southwest sapphires in particular and that you know travel travel time to treatment is a big barrier and I think a lack of understanding in service design around things that prevent people from taking up appointments was a big problem so how do we build services around user needs um I think is is certainly something that from the Charities perspective we're keen to understand more about um you know not to get too simplistic about it but things like you know people having to take a day off work in order to attend an appointment for people who uh frankly risk a day of work is not an option well what's your choice so I think it's a really really good point and it's as much about service designers as well as it is about kind of physical and kind of practical barriers I think so I think there's a question from behind yeah thank you thanks um uh Josh from the Department of Health I work in the international section there and just uh going back to one of the earlier comments about kind of long-term policy or possibly the absence of it and I wondered if there are any kind of Reflections on it appears as a kind of in the absence of a long-term cancer policy shift towards and in the context of multi-morbidity possibly a kind of collective strategy looking at some of those major conditions I wonder if you had any Reflections on the both the kind of potential opportunities for improving cancer outcomes in that context and the potential drawbacks of cancer being a group together in that kind of major conditions context thanks we'd like to take that I mean um thanks Josh it's a great question and um uh the government's decision to pursue major condition strategy earlier in the year given that successive governments had said they would publish a cancer strategy a long-term cancer strategy for cancer is a significant step back I think it's fair to say and from the Charities perspective you know we uh we said at the time you know we will work with the government on the major condition strategy it's very you know there are clearly some aspects that are that are going to be improved by taking a kind of more holistic view of um comorbidities for example you know smoking drives lots of diseases including cancer but also hypertension um um also I mean um COPD et cetera so there are definitely some benefits to taking a more patient-centered view of of ill health that said there are some specific things around cancer that we've talked about a bit about today you know the length of time it takes to do a high impact research um the uh the complexity of the disease you know we talk about site-specific cancers we talk about lung cancer we should talk about lung cancers they are very different um so so from a lung cancer but from a sorry from a cancer perspective I think on the balance of problem on the balance of of choices the major condition strategy is not as effective and it is not as long term it's not setting out to be as long term as long-term cancer strategy would be so um so yeah it's uh for for certainly for for disease areas that didn't have any strategy you know hugely welcome I think for diseases like cancer where you know one in two of the popular one out of every in the rooms day every other one of us will be directly affected by cancer in our lifetimes that can't be said of other diseases so the disease burden for cancer is different um through some of those other diseases so you know it's a mixed bag uh we are you know we are part of the of the MCS and working with Department of Health on it at the moment um we said at the time we continue to say I can't strategy is needed so that's very nicely putting um the actual um reach that cancer will have and its importance is it's very clear um it how much evidence exists though in about the influence of a cancer policy in terms of um outcome so I mentioned the ICP the international accounts benchmarking pathway partnership and the evidence is pretty good actually so icpp looked at I think eight or nine jurisdictions around the world um of that are that are you know broadly comparable to the UK and and measured the uh the impacts of different policy choices and as I said there's a there's a strong correlation between countries that pursue long-term strategies and better outcomes for cancer um uh uh and and it's not you know as I said it's not just having a long-term strategy there is a need for political commitment and a need for frankly taking some of some of the bipartisan stuff out of it um because sustained cancer strategies normally mean sustained Beyond parliamentary terms and there is a need for sustained funding and I think both of those things in recent years have been missing in the UK but I'd check whether there's any um no okay please just pass the microphone that'd be great thank you um for a really interesting discussion I was uh interested by David Cain's reflection that um nice technology approvals are often or usually driven by industry um and that it's outside the skill set of academics often to take things too nice um is that a problem do we think that's a question to all of the panel and if it is a problem then is it a problem for academics for policy makers or for funders or all of them so I'll I'll begin to say that I I think it is a problem I know of other examples where there's almost an excellent clinical trial result which is orphaned because there's not a pathway to um a technology appraisal so I think it is a problem I think academics probably require support in understanding how to make this transfer to policy and when potentially this is something that cruc could help us with at some point but anyone else well the other barrier of course is uh is that it's a cost to go through the the appraisal process I can't remember exactly what is it's but it's o

2023-09-30 11:11

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