NICE topic selection, methods and processes review explained virtual event series: Methods focus

NICE topic selection, methods and processes review explained virtual event series: Methods focus

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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 And finally our final slide is to  really encourage you to share your views   on 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.

2021-08-31 05:04

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