NICE topic selection, methods and processes review explained virtual event series: Methods focus
Welcome everyone to this first webinar to support our methods, processes and topic selection consultation for health technology evaluation at NICE. We're here today gathered with two three panellists to discuss some of the proposals we're making to support your understanding of the consultation. This webinar will be recorded and we will be having a Q&A at the end of the presentation. NICE plays a critical role in the healthcare system in the United Kingdom, next slide please. Our work is vital in ensuring rapid access to clinically and cost-effective health technologies to benefit patients, the NHS and the life sciences. Evolving and improving our methods and processes ensures that we adapt to emerging changes in healthcare and in health technologies.
Broadly there's five reasons to change for the change that we're making today in how we assess and appraise health technologies, but of course our overarching goal is to improve the health of people using the NHS and social care systems and in doing so to support equitable access for people using the NHS. My name is Meindert Boysen, I'm the director for health technology evaluation at NICE and I'll be chairing this session. There's five overarching reasons are as health technologies rapidly advance so too do the methods and processes of evaluation. The second is that reviewing our methods and processes allows us to remain agile and responsive, to support rapid access to clinically and cost-effective technologies. Thirdly, our methods and processes underpin a robust and consistent and transparent decision-making by our committees that need to evolve as societal preferences change.
Fourth, the greater need for alignment between the work of regulators assessors and payers in the United Kingdom to create an ever more receptive environment for emerging health technologies. And fifthly, COVID has put a spotlight on the importance of medical technologies such as diagnostics, devices and digital tech and these methods and processes allow us to level up with how we deal with these exciting technologies. You will find that through all our proposals there's really clear benefits for patients such as prompt access to innovative health technologies and an easier way to contribute to our evaluations. For the life scientists they will find a responsive flexible and predictable way of working with NICE and they of course will also see flexibility when the evidence we find is difficult to interpret and and to analyse.
And of course we also provide a much broader, through these proposals, a much broader support for a comprehensive evidence base including real world evidence. And finally for the NHS it will ensure our proposals will ensure a fair equitable and evidence-based access to innovations and of course in the end as is important for our role, technologies that are clinically effective and value for money will be supported through our proposals. I'm confident that these methods process and topic selection changes will support the ambition laid out by the prime minister in his life sciences vision to make the UK the best place to discover, develop, test, trial and launch and in the end adopt new technologies. This webinar is focusing on methods, there will be three other webinars where we will be talking about the highly specialised selection criteria, our processes and the modifiers used in in methods, so if you're interested in any of those three we'll give you later some more details about how you can participate in in those webinars. Methods are about the sort of evidence that we require and how we take them into account when making decisions about the value of new technologies. Richard Diaz a senior technical advisor and Ian Watson, another senior technical advisor at NICE will be talking you through the proposals we're making in our methods update and after their presentation I will be coming back with our third guest Mark Rasburn and we'll be having a Q&A with all panellists. Over to Richard first.
Thank you Meindert, as many of you know this has been a very large undertaking and for good reason as the methods underpin everything that we do in evaluating new technologies, next slide please. As a result we divided the work into two main steps first what um so what is needed to change and then second how how should we make those changes so at the end of the first stage of the consultation uh the first stage we consulted on on the types of changes and whether there was a good um case for for making those changes. So that consultation ran from November to December 2020 and the responses that we got from that consultation fed into this stage which is the how and the responses were used to formulate answers to that question so how should we do make make the changes that were proposed in the first stage so throughout the whole process we've engaged with a wide variety of stakeholders including patients, patient groups, industry, we included pharmaceutical companies companies specialising in medical technologies and those that focus on diagnostics we also included members from across all of academia as well as the system partners in the government, the the Department of Health and the NHS and we anticipate that this participation will continue throughout this consultation and during the implementation period. Next slide. Thank you, so these are the five areas from the first consultation into which each of the main cases for change fall into. So there's valuing the benefits of technologies, understanding and improving the evidence the structured decision-making framework that's to be used by committees and then how to deal with challenging uh the challenges of particular types of technologies and last but hardly least is the merging of the manual across the technology appraisals program, the med tech program, the highly specialised program and diagnostics, so four programs merged into one.
Next slide. So during the first consultation we received a large number of responses as you can see we received 197 responses from organisations and individuals from the breakdown you can see that unsurprisingly the largest number of comments that we received were from the from industry so from pharma, device industries, diagnostics, consultancy, industry bodies that sort of thing but we also had a substantial number of responses, over half from patient organisations, NHS, NICE, academia, individuals and other organisations which when included with the patient organisation as I said represented more than half of responses. So um there was strong or general support for the majority of the proposals which is not really surprising as they were developed in conjunction with external participation so you could understand why many of the proposals are supported uh and that said there were some some proposals for which there were mixed views but that was mainly because those topics um still had the how to be developed and so without the how being defined people were uncertain whether they could um give the full support to a change without knowing what that change would be so that's where this consultation comes into play into play because now the how has been established and people can provide their comments on on um those those cases for change. Um as I mentioned before the consultation comments were essential in helping us to formulate the how and and so your responses for this consultation will be as important in helping us implement the changes so and now Ian will provide us some highlights of the new proposals that have been presented in the consultation.
Next slide. Yes thank you Rich so yes this slide just introduces uh the uh the breadth of the proposals that we're presenting um for this consultation and looking across the five sort of areas or headings that we presented in the first consultation you can see that we've introduced a number of proposals spanning the full breadth of those. So for the most part in a large number of cases they are developing from and seeking to implement those proposals uh in the case for change um that met with uh positive responses in the consultation we are able to move from those cases for change into fully developed methods that we present present here. Um as I said this spans across the the five headings looking at the valuing the benefits of health technologies there are proposals across modifiers and uncertainty and discounting which I will uh talk about a little bit more in a moment for the evidence base and under how we understand and improve the evidence base again we have moved to implement a number of changes and improvements in how we handle evidence, how we look at things like costs and health related quality of life and present guidance on different types of evidence we also present information and proposals on a structured decision-making framework and approaches in specific circumstances and build on those cases for change for challenging technologies to implement really valuable proposals and refinements and some additional clarifications for those technologies. Um and what I'd like to do now if I could move on to the next slide is pull out a couple of those highlights to talk in a little bit more detail.
One of the topics that I think will be of interest to a number of people on this webinar is the the severity of disease and we presented a case to introduce the severity of disease as a as a key consideration or a modifier into our into our methods we propose an approach here that's built on additional waiting for those treatments and health benefits in the most severe diseases based on a technique known as QALY shortfall and we're going to present many more details on this at our next methods webinar which you'll hear about later so perhaps won't talk uh further about that topic here but that rather move on to the the topic of health inequalities and this remains a really critical priority I think uh it's it's really uh important for NICE and for many of our stakeholders that we consider health inequalities we identified that there is a case to to include consideration of health inequalities but there are important challenges that need to be resolved first before we can do that in a formalised way there remains the flexibility within our methods and within our with our principles and within our statutory uh and framework to consider things like health inequalities where they are relevant within uh individual evaluations. But we're not yet at the stage where we can introduce a specified formalised modifier just yet so there's a little bit more work to do and we need we've identified what tasks need to be done before we can go about doing that. A further topic of interest is that of discounting this is a technique that is used to reflect uh how different costs and health effects are seen at different stages when they occur in the in the future at different times and we believe there remains an evidence-based case for changing our approach to discounting, changing the rate at which we uh um discount uh health effects but there are wider implications and interdependencies of that of that proposal that are beyond the reach of this review and that was identified in that case for change and it remains the case now. So our proposal then is to retain the current methods um while um while further conversations around those wider implications are pursued so we retain our current methods in the meantime.
Moving on to the next slide then um a further topic of a very wide interest is that of uncertainty as many of you will be aware it's very important that we think about uncertainty uh in the evidence when we're evaluating health technologies, knowing that where the the evidence is uncertain that can create a risk for for somebody having a technology or for somebody else elsewhere in the NHS whose health care might be displaced by additional costs for a health technology so it's critical that we think about uncertainty but it's critical that we think about it in the right way and proportionately. There are two aspects to this the first is how we go about understanding and presenting that uncertainty within our evaluations into our committees and we present proposals here to to fully and robustly characterise the uncertainty and clearly identify uh what that uncertainty looks like with the nature of it and how it affects uh and how it impacts on the the health technology under consideration. We also identify some further work that we want to do to implement uh different ways to present that information or visualise the information around uncertainty and knowing that that doesn't necessarily need to be done through the the methods guidance, the methods manual that we present that could be done through other techniques for example in our template so we identify some further work that we'd like to do there. And in this at this stage we now present a slightly reduced emphasis on a technique known as value of information analysis, this is something that we identified a case to to to change to expand our use of in the first stage but we heard through the consultation some of the concerns that stakeholders raised about how we would go about doing that and how that would impact and we as a result of that I consider that it's appropriate to reduce our emphasis on that technique at this stage. Then having understood and presented the uncertainty we need to think about how that is considered within a decision within it within an evaluation and we present a proposal to to use flexibility when evidence generation is particularly difficult so this reflects the fact that there are some circumstances such as those examples shown on this slide where generating the full full picture of the evidence is particularly challenging we want to avoid those circumstances being disadvantaged by uh by excessive sort of going too far and considering uncertainty and so allowing a degree of flexibility that really reflects what our committees were already doing and were already able to do but we now clarified that and codified that and described it in further detail about what that flexibility looks like so those examples are in particular diseases affecting children and rare diseases and also for innovative and complex technologies where it can be particularly difficult to generate uh generate particular types of evidence. Moving on to the next slide then please there a further highlight I wanted to talk about is how we want to support a comprehensive evidence-based broader ranges of evidence and we suggest we propose broad improvements to how we source and synthesise evidence, how we bring it together, how we present it and that most importantly presents improved clarity and predictability both for people submitting evidence to us and for our committees in the expectations that we have of evidence, emphasising the importance of the the highest quality of evidence that it we're able to to source, but considering it proportionately to the context that it's being looked at.
Real world evidence is an area of particular interest and we highlight here the really broad range of application applicability of real world evidence across different evaluations in different circumstances, different types of evaluations different types of real world evidence and we want to support that and encourage that and to do that we present guidance on appropriate ways to use this type of evidence and how to develop it such that it's as high quality as possible for the circumstances being used. And that guidance comes both through this methods review and through wider activities within NICE going beyond beyond this piece here. Then thinking about broader types of evidence again we're presenting improved guidance so we can give further clarity and predictability for people developing evidence on things like surrogate outcomes for example also expert elicitation techniques where we can bring evidence from from experts into into evaluations and other broader sources of evidence that I haven't mentioned here things like qualitative evidence for example and these together present that package where we really support that broad and comprehensive evidence base uh getting the best out of the information and evidence that we we can. Thank you so if I could move on to the next slide then please.
The final step then is to bring uh bring together the methods across our evaluation programs with the objective here of future proofing our methods across the different types of evaluations as the distinctions begin to blur or to change between different types of technology or different types of treatment and to that end we then present a combined manual where we bring together the the four programs that Richard highlighted um into a single manual and we present it here for information it's in it's a draft manual here for information that describes the shape of that manual and how it sits together and how we propose to present our methods guidance in the future. And then looking forward from that the next step is of course once we've completed this consultation and heard your views and taken them on board to begin to implement this in evaluations looking to do that once the the final methods guidance is published beyond that then we propose this become our last sort of large-scale methods review, single methods review but rather to move to something that's more uh more agile, more modular so we can take individual developments um more more quickly more promptly rather than a single um all spanning methods review, and that's a that's a project for the future and with that I'll hand back to Richard for the next slide. Thanks Ian, so I guess the question that you're asking is yourself is what does this mean for me so just a reminder this is only one component of a number of broad improvements within health technology evaluation and access that NICE is working on. However um specifically the changes that we are proposing in this consultation will have several effects and that includes being able to support prompt access to new and innovative health technologies, um the new methods will also provide a clear value benefit in in a fair and equitable way where treatments get treated in a fair and equitable way and also in an evidence-based manner. They will also help clarify how we explore uncertainty in the way that we present it contextualise it, and ultimately how committees consider it.
The methods will also provide further support for the adoption of a more comprehensive evidence base as Ian was mentioning and that will take full advantage of the best evidence that's available at the time and then the methods will also provide an increased increased clarity and consistency and transparency for the companies that are involved, for patient groups that are involved and also for for the NHS and across committees as well. And finally um they'll be able to help ensure that the decision making is responsive, flexible, predictable and as robust as possible so um I think Meindert already mentioned this but if you have any questions please submit them via the Q&A function and with that I think we'll take questions now. Thanks very much Richard and Ian for a very comprehensive insight in these proposals and again just to reiterate we're talking about the methods today but there's three further webinars where we will be talking about highly specialised technologies, the process proposals and go in a little bit more depth into the modifiers you reiterated that Ian. We're going to be joined by Mark Rasburn he is a senior advisor a senior public involvement advisor I should say in the public involvement program at NICE. Mark are you are you online, hello hello thank you very much for joining us um and it is appropriate I think to to therefore kick off with the first question we've received that specifically addresses the impact on patients.
So could you could you perhaps clarify how we have assessed what impact the methods will have on different patient groups so say for example, people with cancer, people with rare diseases or indeed people with other conditions. Yeah thanks Meindert and it's a good question to start us off with so in the development phase of this review we establish task and finish groups which had participation from patient organisations on each one and these groups explored the impact of possible outcomes and options one being the benefit realisation group. We do have a webinar specifically looking at modifiers as Meindert has previously said which will go into a lot more detail but as an overview in this area we explored how different options might affect different types of condition and different patient groups.
We also thought about how these proposals would impact patients across the NHS as Ian earlier mentioned in the presentation such as considering how any additional costs or additional modifiers would displace healthcare from others accessing and using the NHS and along with these working groups we also consulted on these proposals as Richard earlier mentioned to listen to what impact they would have on different patient groups and again this is another opportunity to to get that feedback in the in the next stage of the review and as we develop the final methods and the implementation plans we will continue to explore the impacts of these methods, so for example one option we're exploring is using case studies to consider in detail how different methods interact for particular types of patients and different patient groups. Thanks very much well the really really impressive interaction with with patient organisations and indeed just to reiterate for everyone listening there's the consultation is open to mid October so there's ample opportunity to engage with the with the proposals we're making and I should also re re iterate that there is a brilliant set of supporting information on our website a very easy to easy to comprehend and easy to interpret material that that explains what were what we're proposing in these in these various changes. So perhaps an easy question to Rich in in first instance when can we expect these changes to be implemented in actual HTA submissions because this is this is the second consultation uh when when can we see the change really happening? So we envisage that the changes will be starting to be implemented in the beginning of the year, so in January 2022 uh but between now and then um we do intend to um to do some implementation work so that will uh you know watch the space and we will keep people informed as to how they can get involved uh at in those um different steps during the implementation phase. And and and maybe you could also answer this one then is this only for single technology appraisals or also for multiple, is this for highly specialised med tech how broad is the is the methods update scope? Now this, sorry. Rich you go ahead. Yes this this does cover all the topics, all the areas that you that you mentioned uh so med tech, diagnostics, uh highly specialised technologies, single and multiple technology appraisals yes.
Perfect. Right and just to add I think I spoke a little bit about one of our ambitions to align across the programs and to make things as consistent as possible between the different types of evaluation. Now that doesn't necessarily mean that all will be the same because there remains some a case to to have appropriate differences so there are differences between medicines and medical devices and highly specialised technologies and uh and diagnostics so um we present then a uh a combined manual that aligns as much as possible those methods across the programs but there are some relevant differences so you will read as you explore the consultation some talk for example on the the modifiers there is a there is a case for some relevant differences in how we think about different aspects of technologies of medicines and highly specialised technologies in particular, there's relevant ways that they are different it is right that we highlight those and we're clear about those so hopefully you can explore those in the consultation papers. And and Ian just to follow up on that the the as we're used to now we have methods at nights that are more you know really based on principles and then we have a set of technical support documents and I imagine that if I think about these methods these are very much principle statements aren't they, are you expecting to produce implementation materials that will be helpful for people later down the line? Yes indeed yeah I think that that would be a really important thing that we can do I think Rich described that we we do plan to develop some implementation strategies, some approaches and further work to do on that one of the components of that will be some updates to the technical support documents which will provide some some of that further detail on exactly how you go about doing some of these techniques uh beyond the principles that we describe in the methods guidance and I think there's some other there's some other work that we can do with with individual stakeholders with our committees uh with with NICE colleagues for example to explain and make sure everybody fully understands the methods and support those with materials, documents training sessions for example. I think we can look look forward to a very comprehensive approach to making sure these are implemented knowing that the five topics are quite broad and uh while there are some topics that are particularly prominent we want to make sure that everyone fully understands the full breadth of those methods improvements so we can really get the full benefit from them and really realise that the benefits of those improvements.
Mark if you think about these methods and the proposals made how have we reflected patient and public preferences in the methods, you talked a bit about how we worked with patients but but where do their preferences come into the into play. Yes so I suppose it's best kind of starting what we currently have so we do currently have kind of a fairly robust methodology for how we include patients and the public across our health technology programmes um and this is kind of used in patient public involvement which helps kind of understand what these preferences are tell us to develop the appropriate guidelines and the guidance and ensure what we're developing is actually appropriate for what patients need so we want to continue with what's working well so for example we're going to continue having two lay members on our committees, we'll have open consultations and the lay members are equal decision makers but what we found is this kind of disparity between different practices depending on the program so what this method methods review wants to do is kind of standardise the opportunities for patient evidence and patient involvement across all of our programs, so one example is our kind of patient expert submissions which is current which was currently only um happening in our technology appraisals but not for our diagnostics program or med tech programs so areas like that we wanted to kind of expand kind of good practice which is happening um in sound levels across all of it and have that kind of standardised approach. In the methods document you'll also see that we're proposing using more evidence from people's lived experiences and this is designed to kind of better reflect what it's like to live with the condition in real life what the different patient preferences are but also to help clarify the circumstances and the differences in evidence in both their strengths and limitations to help NICE and our committees to kind of fully understand what the patient preferences are and where shared decision making and patient choices might come into kind of the discussions. There is more information about how we're going to do this in the in the processes document uh and we're going to have a webinar on that but I suppose the kind of key areas for patient preferences and patient involvement are using kind of real world evidence, increasing our use of patient evidence and also ensuring that the same opportunities for for NICE to gather that evidence is included across all of our programs and standardised to a higher level, so using what we currently have and building on it to kind of improve both the use and the impact of the evidence to help us kind of decide and understand what those preferences might be.
Great, Ian, Richard, okay so that's the that's the patient evidence coming in and preferences coming in, how are committees supposed to weigh all that evidence are there statements about that in their methods update? Yes I mean I think uh committees have a a lot of experience of weighing up exactly this type of evidence, so um the the deliberative approach the structured decision making approaches that our committees have been using for for a long time, they're proven very valuable in doing that so one of our objectives then is to just really make sure that we make the most of that evidence so we can present it so that they can deliberate on it. I think a further thing that we've done is um for example in building the decision making framework and looking at the modifiers for example um we present four committees an understanding of how society values these particular things so so for example with the severity of the disease we present to the committees an understanding of what values society as a whole places on these particular factors, or these particular considerations to allow the committees to take it into account in line with the preferences for for the country as a whole so to take into account the severity of the disease as an example uh consistently with the way that we understand from the evidence, the people that people do value that as a consideration so that builds into a a structured decision-making framework that allows the committees to deliberate and use their judgment and build on that evidence in a in a holistic way but also reflect the the values of society appropriately. Great of course as we all know real world evidence is is is definitely a great that's a great part of the the methods proposals and and how we're asking our committees to look at a much more comprehensive evidence base. We've got a question here from Dan Ollendorf who we all know is a great colleague from the United States, so then I've got no clue what time it is at your place but it's great you're you're online but you now disappeared, the question the question disappeared.
It's in the answered section. Is it in, Thanks thanks for helping me out which after 18 months of Zoom it still is a surprise how these things work. So Dan you ask at initial assessment, real world evidence will have limited utility in terms of improving the evidence base it does exist how will NICE engage industry and other stakeholders to address limitations in the initial evidence package for example for a rare disease drug or end stage cancer drug studied in only a single armed settings, is there guidance on historical control sets or other supportive evidence that could be provided or that should be provided to NICE. Who wants to answer that one? Ian? Yes I can I can talk about that I mean I think um I think I wouldn't undersell the utility of real world evidence.
I think it has some important limitations of course and it's there's particular limitations in establishing the overall effectiveness of a medicine for example um and we know then that a well-conducted randomised control trial remains really the gold standard for that particular use but there's a number of uses where I think we can get a lot of information from real world evidence I think things like um clinical practice what's happening in the real world of of medicine and how that impacts on the the patient experience and the benefits and the costs of a health technology, it can have some real really important uses in diagnostics context is another example understanding the full pathway of of care and how that might change or with a a change in the diagnostic approach medical technologies real world evidence can be really valuable for people's experiences of using the technologies and how how it works for them in practice what what limitations or benefits they find from it so really spanning the full breadth of a health technology evaluation approach real world evidence does have a lot of potential use I think um and I think it's great that we are able to to support and encourage that but I do absolutely take the point that there are some limitations of it and I think the question around how we're going to engage with stakeholders to make sure we improve that evidence base is really important. So one of the things that we're doing is presenting some guidance straight off on how we can make the best use of real world evidence and set some standards and expectations for getting the highest quality real world evidence that we can so that process begins here but it doesn't end here there's certainly not the end of the road for real world evidence at NICE, there's a there's a lot more work that can be done across the institute I think to provide further information and guidance on that. On the specific questions around single arm trials and historical control sets again there are areas that we can develop the principles that we set out in the methods guide in the methods proposals that we present I do do address that albeit not necessarily directly because there's such a breadth and a variety of different types of study design and study approach that we can take so we set out the principles here rather than dictating specific methodological approaches but then that leads into Meindert your question earlier around the further supporting information and guidance that we can give technical support documents for example and that's another area where we can build even further onto onto the methods guidance to really support that top quality really valuable real world evidence. Brilliant a related question to real world evidence from Felipe Vera, but now it's gone I must I must not be good at this bit about the real evidence approach I know it's still there. This is going to be a more methodological area of effectiveness is this question or in the possible economic evaluations we're modelling and in particular I thought this was an interesting part how does the new approach to QALY lost due to the disease as this is proposed in the modifiers, how will how will that work with real world evidence are people expected to submit further evidence or is it really part of the next webinar, Ian? Yes I think the um the the consideration of displacement and loss of healthcare or displacement of healthcare elsewhere and the system probably will be a topic that we can explore a little bit more in the the next methods webinar where we um where we talk about um where we talk about modifiers uh so that that will be and particularly the severity modifiers so that will be an area um okay where we can explore that but I think the other um part of the the question around um the real world evidence I think as I say there there's there's a really broad range of applications of real world evidence and I would see it having a potential role across any aspect of health technology evaluation so it could be that we're talking about effectiveness evidence but also in the economic evaluations and the economic modelling real world evidence to establish resource use is a really potentially valuable avenue that we can explore so what we have tried to do with the proposals and with the methods guidance is really stress that we're not only interested in a particular narrow understanding or view of how real world evidence can be used it really is across the board but the principle of the highest quality evidence possible and that it be considered and evaluated and explored and critiqued proportionately to the circumstances being used, whether that be a particular type of technology or a particular application or a particular type of uncertainty whether it be something that is a very significant effect on the results or whether it's something that's perhaps more minor and might need less scrutiny that we do that proportionately and reasonably and appropriately in each circumstance.
There's a question about the overarching goal in making these changes and we already discussed that haven't we is there anything that you want to add to that. Just that I mean the whole point of this methods review is to keep NICE's methods as up-to-date, as current, as cutting edge as as as it should be um for to reflect the changes that are happening in tech in the development of new technologies and new types of technologies that we're seeing so this is a response to that and I think that the change to the modular approach of future reviews is also a response to that so that we're more flexible in the way that we can update and and we don't have to make these huge strides in in changing from you know one methodology to a completely new one although this I wouldn't say that this is a new completely new this is an evolution of of the existing uh methodology. Someone asked about the the introduction of the the disease modifiers in NICE's approach to introducing severity of disease modifiers there's a graduated approach where these modifiers are applied to both medium and high severity indications and we're going to talk about that in a different webinar as we've said a couple of times but this specific question is why has this graduated approach to decision modifiers not being considered for a rarity of the disease so, what is the difference why are we doing this quantified work for the severity modifier but not for rarity and there's then the subsequent question what support do NICE proposed for manufacturers developing novel new technologies for rare diseases. So perhaps on the first bits Ian.
Yeah that's a good question um and I think this um goes back a little bit to the the evidence that we explored in the first stage of the consultation of the the methods review and that that that what stage that Richard described where we looked in detail at the evidence for societal value so looked in detail at how society and people across the country view different factors and what value they ascribe to them and what we found in terms of rarity was there really isn't evidence that society puts greater value on rarity itself uh in of itself uh rarity is not really something that that people particularly put greater value on and for that reason we considered that there wasn't a case to introduce a they modify specifically uh for rarity uh in contrast what we found was there was evidence that society does place a higher value on those those health benefits for the most severe diseases um and that's why we present um the case for change um that we did and hence the the proposal that we presented at this stage. Now having said that in practice a lot of rare diseases are indeed severe and one of the benefits of the the severe disease um modifier is that we broadened the modifier beyond what was previously a relatively narrow view of specifically end of life conditions that we looked at before so we broadened out into a in a wider range of conditions and circumstances and that can include rare diseases so that means that where a rare disease is is um causing very severe symptoms or very severe effect on people's lives that is appropriately reflected in the values that we we ascribe to and the values that our committees can place on it so I think that has an important benefit I think for poor rare diseases. I think there's also um a number of other things in in rare diseases that the that are features of rare diseases and features of evaluations in rare diseases where our methods have been have been improved further elsewhere so for example we know that generating evidence from gold standard randomised controlled trials can be very difficult in rare diseases and that's one of the reasons why we want to really leverage that benefit of real world evidence and also move into other broader sources of evidence such as expert evidence, qualitative evidence, surrogate outcomes for example, so these all put together combined to make sure that we're getting the best of the evidence that we can and as much information and leaving as few uncertainties as possible from the evidence that's available. Then when it comes to the uncertainties we also recognise that that evidence is indeed difficult to generate and those uncertainties are to an extent inevitable and we want to again make sure that those rare diseases are not blocked by a disproportionate approach to uncertainty and a lack of flexibility so we we propose to really clarify and codify and clearly explain the flexibility that we uh that is available and has been used in the past indeed by by committee so committees have used this their judgment uh in the past to reflect this system of flexibility, but we we can clarify that we can codify or can explain it and make sure it's very clear how we are applying that flexibility when it comes to uncertainty in those circumstances where where evidence generation is is difficult where it's challenging. Thanks very much. Mark, do you think if you think about patient involvement and how patients interact with these methods will it become easier for them or more difficult because it feels it feels quite complex doesn't it what we're talking about, what's in the consultation have you got have you got an idea how how we can make it easier if it is ever more complex? Yeah I suppose the simple answer is I hope it makes it easier the whole aim of this is to kind of standardise our approach so whether patient organisations are attending one of our technology appraisal meetings or diagnostic meetings we should have a kind of a standardised approach to what patient involvement and patient evidence is being included in those committee discussions so they have kind of an awareness of the kind of the methods and the processes which we use as an organisation but I think as part of my team the public involvement program there's also the role of kind of improving the support which we have so this looks at kind of the toolkits we make available, templates and the support to essentially make sure that we're we're communicating with patient organisations and our patient experts to identify where the gaps in knowledge are, where the gaps and evidence are, where the kind of patient experience is needed to influence the decisions and influence committee discussions and essentially make it as easy as possible to involve and engage with NICE as we can make possible because we have to kind of appreciate we're working with organisations who were global leaders in their area as well as those ultra ultra rare condition areas which might just be a volunteer sat on a laptop so we need to make sure we're adapting our support depending on the need of our stakeholder so I'd provide more information or if they're they're well versed in a NICE process they might not need as much support from my team but we really wanted to kind of ramp up the kind of I suppose the engagement, the communications to make sure the information that they're giving us is beneficial to the committee and in the same kind of scope of that we're also starting to ask our committees how the patient evidence has influenced the discussion and the outcome so we can feed that back and constantly learn what is having the biggest impact, is it the way we're presenting, is it the type of evidence but essentially making sure that evidence is having an impact and not just a tokenistic approach for involvement.
Yeah because there will be I think Ian and rich there will be more on visualisation and how we present the information that committees have taken into account. I know Polly Moyer asks question specifically to a product or perhaps not not to go into detail here but she wants to understand better how how how these methods work for a new technology that's being introduced into the UK, what what do certain terminology we're using what does it mean and I know the answer is probably you should contact Mark and his team and they can discuss that in more detail with you. There was a question here from Jose Diaz and I mentioned the importance of understanding the societal value derived from innovative, clinically and cost-effective technologies and he wants us to expand on how exactly NICE is planning to identify, evaluate and incorporate in decision-making societal benefit and spill over benefits from innovation, are we going to do that Ian? I think that would be a really good candidate for something for us to look at uh in the um the next stage of a modular of a modular update I think there's definitely a case for us to think again about what evidence and types of value we want to take into account so what we've done at this stage is we have clarified how we approach innovation and how we particularly around the uncertainty piece so this is this is where that's a particularly important consideration but I think there's there's definitely some value in us thinking about things like spill over effects from innovation in the context of a modular update thinking about the implications of that so that wasn't something that we presented in the case of change it wasn't something we we identified enough evidence to work from at this stage but that doesn't mean that it's something that we are we are finished with there's definitely a longer term piece that we can um move on to within a within a modular approach.
But we are taking innovation into account in our uncertainty proposals yeah exactly. Yes absolutely yes no no we absolutely take it into account but I think it's that longer term piece around the the value of spill over and scientific um externalities those sorts of technical concepts but build on um really important societal value um that we can bring into our evaluations in a meaningful way but does require that more sophisticated longer term deliberate deliberation. There's a couple of questions around the modifiers that perhaps are more for for next time so for example one around what would happen if if future research on societal value of severity and that we're signalling we will be doing if it showed that people do not value severity more highly what would happen how do we how can we make sure we don't then go backwards in getting patient access? How will we balance displacement costs and providing adequate incentives for advancing existing exciting not existing new technologies which have high initial costs so that second is perhaps a different question so the first one about severity we can talk about in the in the modifier webinar but but maybe the second one how will NICE strike a balance between considering displacement costs and providing adequate incentives for advancing exciting new technologies with with high initial costs, cell and gene therapies is there anything you can say about that Rich? I mean there are a number of different mechanisms that NICE has in place to encourage um this this sort of development so so um apart from the methods update that we're engaging in now there are things like the innovative drugs fund and other mechanisms that would allow for that to happen so so so um I guess thinking about this as one of many a suite of options that are available um this this is this is not one a one-stop shop it's it's it's we do we look at the wider view. Yeah there's a couple of questions around the implementation of when when when these proposals were applied to either reviews or new technologies so we should we should discuss those on another on another occasion I think perhaps in the webinar around process there's also a couple of questions around discounting and specifically what are the overlapping issues and how we're intending to support further discussions I think that's probably beyond the scope of this this this specific webinar considering that you know that that's that's something that isn't really instigated by NICE those discussions but has to be happening between the health system partners that we work with there's also a note about the method standards framework using real world evidence and of course that's important work that's happening already and perhaps it's perhaps a very final question about the modular updates how do we envisage those to work? I think that's a really important question and it's something that we need to design um to be honest it's something we haven't yet established how it's going to work but I think we we need to do a process of designing um how we go about that something that's really important is making sure that we have the right engagement with stakeholders it's not something we would ever want to do on our own in a silo we want to have engagement with patient groups and patient representatives and industry and academic academia academics um and the wider world as well I think there was a question somewhere about um international HTA organisations that's something that we can we can uh design into that modular approach as well I think I'm sorry some of the ideas that I was thinking around uh are designing different approaches for uh different types of updates so where there might be smaller clarifications we might have a smaller approach that would be proportionate in that case whereas something larger such as a modifier and might need a more sophisticated longer term piece and we can be really dynamic with this the whole idea is that we can be very flexible and dynamic and move quite fast with the with these things and not be stuck to a a very fixed long-term large-scale um project but but make the right changes at the right time um in the in a way that's meaningful so we can keep those methods uh right on right on the cutting edge and moving as quickly as possible and as dynamically as possible. Okay there's a there's a very final question I think I should I should put that out there could you explain the rationale a motivation for keeping the proposal on subgroups given a strong objection from a variety of stakeholders? Rich is that this can you just briefly attend to that and then we'll close the meeting. Well I think uh there there wasn't um opposition I think that there there was a mixed views but the but in terms of what is included in the um the consultation it's it's already what um committees can do so they can already uh make recommendations um that are based on the evidence that's available to them uh and and if a population if a population is is uh not cost effective then then it should not be uh recommended in that population um so so yeah so I think it's it's consistent with the approach that committees already take.
Brilliant well thanks thanks very much everyone for participating, I'm sorry we haven't been able to answer all the questions you put in but we should move to the to the final slides we've got as we mentioned very at the very beginning of the of the presentation so next next slide I hope we can see those still, the future events. We've got the highly specialised technology selection criteria focused on the first of September that's next week from four to five then we have on the 8th of September our process and topic selection proposals from 1 to 2 and the methods review a focus on modifiers and there have been so many questions today already that will be on the 9th of September and you can register at www.nice.org.uk/news/events And finally our final slide is to really encourage you to share your views on www.nice.org.uk/methods-review the consultation closes on the 13th of October and there is an email address if you have any further questions thanks everybody for participating in particular Rich, Ian, Mark and the team behind the scenes and we hope to see you at the next webinar next week on the 1st of September thanks very much have a good day.