NICE topic selection, methods and processes review explained virtual event series: HST focus
Good afternoon everyone and welcome to the second webinar we're holding to support our methods process and topic selection consultation for our health technology evaluation programmes here at NICE. My name is Jen Prescott, I am a programme director at NICE, responsible for the process and operations within our health technology evaluation programmes. I will be chairing the webinar session today. I am also joined by four panellists
to discuss some of the proposals we are making to support your understanding of the consultation. This webinar will be recorded, and we'll be having a questions and answer session towards the end of the presentation but let's move on to the first slide. In this webinar today we're going to cover aspects of the topic selection process, the background and focus of the highly specialised technologies selection and routine criteria, the refined criteria that we've proposed in the consultation, alongside interpretation and application of that criteria. We do tend to shorten the term highly specialised technologies down to HST for short. I hope that attendees today have content with us using HST as a shorthand term.
Next slide please. NICE plays a critical role in the healthcare system in the United Kingdom, the work we do is vital in ensuring rapid access to clinically effective and value for money health technologies that benefit patients, the NHS and life sciences. Evolving and improving our methods and processes ensures that we adapt to emerging changes in healthcare and health technologies. 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. We've outlined five reasons for the changes that we're proposing to make in how we assess and appraise and evaluate health technologies.
The first is a recognition that as health technologies rapidly advance so too do the methods and processes that we need to evaluate them. The second is reviewing our processes and methods allows us to remain agile and responsive to support rapid access to health technologies that are clinically effective and value for money. Thirdly our methods and processes underpin robust consistent and transparent decision making by our committee, but they need to evolve as societal preferences change. Fourthly there's a greater need for alignment between the work of regulators, assessors and payers across the United Kingdom to create an ever more receptive environment for emerging health technologies. And then finally COVID-19 has put a significant spotlight on the importance of new and emerging medical technologies in particular devices, diagnostics, and digital technology. These methods and processes in this space allow us to level up how we select and manage these exciting technologies coming forward.
You'll find that through all of our proposals there's really clear benefits for patients for faster access to innovative health technologies and an easier way to understand and contribute to our evaluations. For life sciences they will find a responsive flexible and predictable way of working with NICE and they'll of course also see flexibility when the evidence we find is difficult to both analyse and interpret. Through these proposals we offer much broader support for a comprehensive evidence base including real world evidence. And finally for the NHS our proposals will ensure a fair equitable and evidence-based access to new innovations.
And of course, in the end it's important for NICE's role that technologies are clinically effective in value for money and they can be supported through our proposals. We are confident that the new methods process, and topic selection changes will support the ambition that was formally laid out in the new life sciences vision to make the UK the best place to discover, develop, test, trial, launch and adopt new technologies. This webinar today is focusing on the selection criteria used to route topics to the HST program.
We've already held a webinar on methods and hopefully some of you that took part in that first webinar are taking part today and there are two more webinars planned to take place where we'll be talking about our processes for guidance development and the proposed modifiers used in evaluation methodology, so if you're interested in any more of those we'll give you some more details later on about how you participate in those webinars. With me today we have Sheela Upadhyaya our rare disease strategic advisor, Heidi Livingston our public involvement advisor and Lori Farrar associate director for horizon scanning and topic selection and they will all be talking you through the proposals we are making in our topic selection update for highly specialised technologies. After their presentation I'll be coming back with our fourth guest Helen Knight who is a programme director for technology appraisals and highly specialised technologies, and I will be hosting the question and answer session with all four panellists, so with no further ado over to Lori first. Good afternoon everyone, so as Jen mentioned we are doing two further webinars and one of those next week on the process one will also include topic selection so this slide is just a quick snapshot of what you can expect to see in the topic selection part next week and also in the consultation where we're consulting on the manual at the moment.
So it has the main top ticket items are those that are in the four boxes where you'll have clarity on the type of health technologies that are appropriate for NICE technical health technology evaluation, a new panel of experts which is known as the topic selection oversight panel that will be brought together to decide on the topics that should be selected for NICE guidance and their appropriate route, and the proposed refined criteria that will be used to consider and route appropriate topics to the HST programme. So that is the vision that is the focus sorry for today and then clearer communication of selection and routing decisions and how in exceptional circumstances a routing decision can be challenged. For more on those next week. Next slide please. I just wanted to really home here on the difference between selection versus rooting because some of the terms can be confusing and that there's quite a lot of criteria for everybody to digest and go through. So, a selection criteria is about a technology that is identified as eligible that must then be considered and assessed against the selection criteria. Each type of technology has different selection criteria which has been listed in the topic selection manual that's currently out for consultation. And then we've got route in to NICE guidance so once the
technology has met all of the selection criteria it's then considered on the appropriate route so NICE determines the most appropriate route for guidance development and in terms of HST, the criteria that Sheela is going to go through shortly is all about the routing criteria and how a technology can be routed to the highly specialised technologies programme for guidance, so I will leave you here and pass over to Sheela for the next part, oh no sorry Heidi, thank you. Hello everybody I'm just going to take you through a couple of poll questions now so I'm hoping they will pop up on your screen in a minute. Okay so the first question is NICE can do more than three HST's a year so please will you let me know whether you think that's true or false? Okay so I can't actually see the answers coming in, so I'm hoping you've all answered that question now and absolutely we can do, oh here we go I can see them, so yes absolutely we can do more than three HST's a year so there used to be a myth going around starting in 2013 that we could only do three a year and that's just because that's how many we anticipated having in the pipeline way back then but now we can do many more if we need to so absolutely we have enough flex in the system to do as many HSTs as we need to. Next question please. Okay so the criteria have been changed for HST's is to reduce the number of topics entering HST. I'm hoping you'll get this one after the last one, is that true or false please? Excellent, it's false. Sheela will take you through much more of the detail in a minute in
her slides but the idea isn't either to increase or decrease the number of HSTs that we do a year, but Sheela will now take you through that in much more detail okay. Away to you Sheela. Thanks very much Heidi. Good afternoon everybody and welcome to the highly specialised technology routing criteria presentation. I wanted to take you all through a little bit of background in terms of the criteria that we've got in place at the moment, they've been in place since the program was introduced NICE in 2013 and we have had a number of challenges where stakeholders have found them unclear and decisions that have been made about routing topics to HST have been difficult to explain and understand. They have been subject to a lot of confusion
and miss interpretation and of course those of you that are familiar with the HST programme will know that there is a higher ICER threshold in comparison to the TA programme of 100 to 300k versus the 20 to 30k in the technology appraisals programme but there is an acknowledgement within NICE that there have been a number of therapies that have increased for rare diseases but there's still a high unmet need that we need to address going forward. Next slide please. I've been, I've been asked to ensure that people who are listening to the webinar feel free to put questions into the questions box during our presentation so we can collate those questions as we go. So in terms of the drivers for change for the HST refinement criteria we're acknowledging the changing rare disease landscape in terms of the increase in the number of rare disease treatments that are coming through, depending in terms of the challenges and the misunderstandings that we've had around the application of the current criteria, we were clear that we needed to have some more clarity and preciseness in the system so that the criteria were easy to understand, interpret and apply. We also wanted to provide predictability and clarity to our decision makers who are going to sit on the topic selection oversight panel and stakeholders who were trying to understand whether their topics would be eligible to be rooted to the HST programme, this in turn should increase the transparency and hopefully make the topic selection routing decisions much more efficient in terms of how those decisions are made.
Next slide please. What we don't want to do is change the essence of the criteria and what this purpose is and I'll, I'll take you through that in in some of the slides that are coming after this one. We're not wanting to increase or decrease the number of topics that are routed to HST and this is not about enabling all rare disease topics to be rooted to HST. We're not looking to address the differences between the standard technology appraisal and HST programme or make amendments to the current HST methods. A number of these things are
being covered in the process and methods consultation which I'll talk you through in the next few slides but some of the things that people are wanting us to undertake require significant policy change and multiple stakeholder engagement to resolve, and these aren't things that NICE can do alone so there are going to be other things that we'll need to do going forward in terms of for example the voluntary scheme that's going to be renegotiated in the future. Next one please. So in the February of this year, we consulted on a proposed vision and principles for the HST programme and in that vision we articulated that we were looking for fair and equitable treatment access for very rare diseases, and we were acknowledging the challenges in rare diseases and the changing disease a rare disease landscape in terms of generating evidence in small populations, undertaking trials and collecting evidence to support the HTA process. Next slide please. The proposed principles that we consulted on during that time was that the topic and the condition should be listed in the manual of prescribed services that the NHS England team use to identify which conditions are covered by highly specialised services.
We wanted to ensure unmet need was addressed so clarifying which conditions would be eligible and a severely limiting length or quality of life. We suggested that repurpose and multiple indications should be excluded, and we also suggested that all types of technologies should be considered. And we're really grateful for all the feedback that we received from all our stakeholders who gave us some constructive input into whether these visions and principles were the right direction of travel for us to take. Next slide please.
So, as I said the consultation took place between February and April of this year and I'd just like to take you through some of the feedback that we've received from all of our stakeholders. Next slide please. So we got some feedback to say we should retain the flexibility and expert judgment when making routing decisions for HST. We should definitely retain the clarity around the unmet need so the condition should severely limit length or the quality of life and all types of technology should be considered to be rooted through HST. There was an agreement in the proposals to remove the lifelong treatment criteria taking into account that we are looking at a lot of one-time therapies that are coming through the rare disease landscape, the chronic condition criteria, the cost of treatment, CCG commissioned technologies of course now we're going into ICS structures and then any reference to the highly specialised commissioning models was also a proposal that people felt we should consider removing.
Next slide please. What else did you all say, well there were some concerns raised about the suitability of the standard technology appraisals programme for rare disease technologies and stakeholders asked for definitions and clarity on patient numbers eligible for routing to HST, clarity around the conditions that would be suitable, clarity about what we meant by repurpose and multiple condition indications and defining the unmet need criteria much more clearly. Next slide please. What we've done to try and respond to some of these things is the methods review is proposing some additional flexibilities and I'll talk through a very short slide coming after this to explain those but as Jen has said please feel free to engage in the webinars that are talking about the methods the proposes in more detail.
We've suggested that we remove the term ultra rare and replace it with very rare and this is in line with the regulation's terminology and it's more familiar to people. We're hoping that we're in the HST criteria have clearly defined now what we mean by a very rare condition, the population numbers that we consider suitable to be rooted to HST and clarified the unmet need criteria in more detail. We've removed the term and the and the approach around not looking at repurposed or multiple indications and aligned ourselves with the terminology used in the voluntary scheme for branded medicines writing and access, whereby all new license indications should be considered for routing to HST.
Next slide please. So just to summarise the proposals that we've got in the methods review for that would be relevant for rare diseases. There's a proposal to replace the end-of-life criteria with the severity modifier, we're looking to accept a greater degree of uncertainty when it's clear that evidence generation is really difficult, and this is particularly seen in rare diseases.
We're thinking about being more accepting of different evidence sources for health related quality of life, particularly where the rare diseases struggle to meet the EQ 5D and then taking into account qualitative evidence that's often presented in the rare disease evidence base around expert elicitation, surrogate outcomes, real world evidence. It may be particularly relevant for rare diseases and those proposals look at how NICE would be willing to consider those in a standard technology evaluation. For more information, please look at the web page there around the methods review which will give you much more detail and as Jen said please join the webinars or listen to them on the channel.
Next please. So let's get down to the HST criteria, what is it that we're trying to achieve here, well in terms of our focus we really want to use the HST programme to encourage and facilitate access to those valuable treatments for people with serious and very rare conditions, who have substantial unmet needs and who would otherwise be disadvantaged by a standard approach. The HST programme is a deliberate departure from the standard technology appraisals approach because we acknowledge and recognise the challenges in terms of the generating of evidence into more populations.
It's really challenging for developers and industry to secure a good return on investment for small populations because of the market and the number of patients that they would be looking to get access for, and there is also huge financial implications should the local NHS try to provide these treatments. So there's no intention to change the focus of this approach for HST. We're looking to ensure that those patients in very small groups with very very rare conditions are well served and that research and new innovations are encouraged in this area.
Next slide please. So, the criteria as it now stands in terms of the proposals we're putting forward and to be considered for HST normally all four of these criteria need to be met, so the condition is very rare and this is now defined by one in 50,000 in England normally no more than 300 patients in England are eligible for the technology in its licence indication and no more than 500 across all its indications. The very rare condition significantly shortens life or severely impairs its quality and no satisfactory treatment options exist so that's around the unmet need and if it does, the technology is likely to be a significant benefit. Now you'll see in these criteria that there are a number of different types of population that have been mentioned and I thought it might be useful if I ran through a few slides just to explain the difference in the different types of population that we're talking about so please humour me and it's particularly for those people who might find the terminology confusing.
Next slide please. So, when we say prevalence what do we mean? This is the total number of people who have the condition in total so when you do the one in 50,000 and apply it, you'll get a number and that gives you the total number that have this particular condition in question that the technology will be used for. Next slide please. The eligible population. This is a subset of the number of people who have the condition
and meet the criteria to receive treatment so particularly technologies will have particular treatment criteria or criteria that have to be met in order to receive the treatment and that's generally in in most cases a subset of the total population, so that's the eligible population. Next slide please. And what do we mean by incidence well incidence is the number of new patients that would be eligible for a treatment identified over a given time, so these are the three types of population that we'll be talking about in the criteria as I explain its application. Next slide please.
So, the first criteria. We've tried to future proof as much as we possibly can in terms of just developing the criteria, but the condition is very rare and it's defined by 1 in 50,000 in England. The very rare means that it has a prevalence of one in 50,000 or less than 1100 patients approximately in England. We're using this threshold um and this this kind of approach it's used by other countries particularly the SMC and we've done some work looking at other countries in different jurisdictions and that they're using this approach as well. It seems like a really good place to start. However there is the opportunity to apply discretion in this situation. In exceptional circumstances NICE can apply some discretion and flexibility to this criteria as long as the technology meets all the other criteria within the selection of the routing criteria. So we've got situations where you may have a condition that's
over 1100 patients and if the other three criteria are met exceptionally highly then we could apply some discretion if we wish to. Next slide please. The second criteria that needs to be met is normally no more than 300 people in England eligible for the technology in its indication so you'll remember this is about a subset of the population that would be eligible for treatment. The smaller the eligible patient population the more likely the criteria will be met. Normally we're saying no more than 300 patients should be eligible for the treatment to be routed to the highly specialised technology programme. There is also situations where we may be seeing one-off treatments particularly gene therapies or cell therapies where they may only be given once in a patient's lifetime and in those situations what we're saying is that there should be a prevalent population so those patients who are already diagnosed with the condition and eligible for treatment of up to 50 and an incident so new patients over a given time of no more than 40 a year would be acceptable, normally.
Again, there is an opportunity for NICE to apply some discretion and that could be in situations where the topic exceeds 300 patients in terms of eligible patients. The exercise where we gather evidence and information on those technologies informs the topic selection panel that there is an opportunity to apply discretion for example where it's slightly over and the unmet need is very high and the other three other three criteria are very highly met. The eligible population is also cumulative for multiple indications so when you've got a technology that has multiple indications up to a maximum of 500 eligible patients would be seen in HST after that there would be a stronger consideration for future technology future indications to be seen in the standard technology appraisals programme. Next slide please. And the last two criteria the first one is around the very rare condition the technology is indicated for significantly shortens or severely impacts quality of life.
We haven't defined what we mean by significantly and severely because we know it requires a level of a judgment, this is based on the information and the evidence that is collected during the scoping exercise when we're undertaking a selection and routing decision. And then the unmet need around treatment options that exist or don't exist and how significant the additional benefit will be. Again, we're looking at things around significant not being defined because again we feel that it requires some judgment. Next slide please. So just in terms of a couple of slides now just to talk you through the application of the criteria in an example format. So, example one talks
about where the all the criteria have been met. The condition is very rare and defined by one in 50,000 so in this example we're saying it's approximately 500 patients in England, 235 are known to be eligible for the treatment based on the criteria for eligibility and there is clarity and understanding that the quality of life for patients is very very poor with a high chance of early death and there are no treatment options available. In this scenario all the criteria are met and NICE would potentially seek to route this through to HST. Example two is the example where you may have over 300 eligible patients, so you've got a 500 patients who have the condition, 320 patients are eligible for the therapy, again quality of life is poor, significant decline, multiple comorbidities. There is a treatment available however it's suboptimal in outcomes or the treatment targets other aspects of a particular condition. In this situation NICE would have some discretion to consider routing to HST if they felt the other three criteria, so that's the eligibility number, sorry the rare condition, significantly shortening life or impacting its quality or that no satisfactory treatment exists the unmet need was very highly met and if there if there was evidence to suggest that then discretion in those situations could be applied to route to HST.
And the last example I have is where the prevalence is over 1 in 50,000 so there may be situations where a condition doesn't quite meet the 1 in 50,000 it's over the expected average 1100 patient number but the eligible population may be really really small like in this example of 76. There's an acknowledgment that the quality of life again is poor with significant decline or other challenges in terms of the life shortening situation and there may be no treatment available. Again, in this situation there can be an opportunity for the topic selection oversight panel to apply some discretionary departure from the normal policy and there could be a situation where they felt strongly that it was the right decision to route this to HST.
Next slide please. So what have we tried to do here and what does it mean for rare diseases? I'm hoping that we've achieved more clarity and consistency and transparency for stakeholders on how the criteria need to be met and how they can be applied. I hope that we're going to be able to achieve some predictability so that there's an early ability to predict which technologies could be eligible for the HST programme and then more efficiency in decision making. I know that there have been some frustrations by stakeholders around how much longer it takes for us to make these decisions and hopefully the criteria will provide us with a much more streamlined and faster process in terms of those decision making processes. Thanks very much, Jen over to you. Thank you Sheela, and thank you Lori and Heidi and so I think we can open the question and answer session now so I just invite all of the panellists to put their cameras on and get ready to take themselves off mute, you don't want to be the ones that speak whilst you're on mute and we do have a few questions coming through and I think someone came through quite early and I think Sheela you may have addressed some of those in your presentation anyway and there are a few themes around the question.
I tried and theme them together as much as I can so thinking about the first question Sheela, I'm going to come to you for this one and a few people have asked this question is what is the rationale for limiting the eligible population to 300? Thanks Jen, so it's been quite a challenging task to work out how to provide clarity for stakeholders in this criteria and those people who've had their fingers burnt in trying to do this will know how difficult it's been but what we've tried to do here is look at the past appraisals and evaluations that we've undertaken in HST both in those topics that were routed to HST and those that weren't. We've looked at current topics that are going through and future topics that may be entering into the HST programme, this has given us a really good flavour of the of the kinds of numbers that we would be expecting to see and based on that information is how we've come to this number. Now it may not be the perfect number and it's totally up for consultation so if there are alternative views, we are open to getting those views back. OK thank you Sheela. Jen can I just follow up on that slightly and I think it's always going to be difficult to find the right number but I think the feedback that we did have is that it would be really helpful to at least have numbers available and we know that there are challenges in actually getting accurate figures for those estimates and so it is going to be difficult but you know as Sheela said the paper tries to explain the rationale of how we've landed there both for one indication and for a technology that might have a number of indications but please feel free for the responses to consultations to advise us of that we you know we've tried to explain how we've got there and why it's reasonable also to have a number attached to it. Thank you Helen.
Sheela there's a couple of other questions around the population sizes as well which I'll come back to you on if that's okay and this is more about the additional kind of restriction if you will or a number around one-off treatments for cell and gene therapies. Are you able to articulate why that's been set at a lower figure? Again, it's a similar response really Jen. We've looked at the technologies that we've seen come through that are one-off within the programme, that are currently in the programme and that are coming in on the horizon now obviously we can't predict what's coming much further away but we've looked about three approximately two and a half three years away and we've seen that the number of technologies that are coming through in that one-off space and the types of eligible populations we'd be looking at in those conditions are around that figure, so the analysis shows that that that's about the right figure in terms of setting the prevalent and the incident populations that we can see coming along in in the horizon going forward. So that again it's a similar rationale really that we've deployed here and as Helen said if that's inaccurate or if there's other evidence or information to show we should be thinking about that again then please, please feel free to feedback and give us evidence to support any changes that we should that we should consider. Thank you Sheela. Helen I'm going to come to you next. There are a couple
of questions about oncology products. Are oncology products now acceptable before routing into their highly specialised technologies programme? Thanks Jen, I mean what we've said in the consultation is that we will you know assess all technologies as to whether they're appropriate for the HST and that would include oncology products. That said you know it would have to meet all four criteria and because of you know the populations for whom the you know oncology treatments are um designed for we don't anticipate that there would be many that would actually meet all of those criteria and as they're laid out at the moment and I you know. So, looking forward yes we would consider them they're not automatically excluded from being considered for a HST. Thank you Helen.
I'm just going through the questions here just to make sure I pick up where there are potential themes that have come through. So Lori can I come to you next? A question about topic selection in general and I think challenging historical decisions. So even the revised criteria and if a technology does fully meet the new criteria is it possible to challenge earlier decisions on technologies that may have been previously routed to the single technology appraisal process with a view to rerouting to HST? Thanks Jen I think that this really would depend at what stage the the topic is in so if it is in guidance development then that is not really an option that we would stop guidance development at that time. The criteria also just out for consultation at the moment and are proposed and they still have to go to the board for sign off before they're implemented which will be early next year 2022.
So as part of that we will then look at when how we will implement them and where the top and we will do that internally as to the reconsideration of topics before they're in that guidance development stage but for anything that's historical and in that guidance development stage we wouldn't go back and change the re-routing. More so because of when the criteria will come into play as well. Thank you Lori, Helen what does nice consider as significantly impairing quality of life in respect of the criteria for selection for HST? Yeah, it's a difficult one and I think you know when we take the proposals through towards the final development, we can think about perhaps putting examples around that. We know that you know we we're not looking at a full evidence base at the point that we're making these decisions so I think you know we're looking back at the types of topics that we have been taking through HST already and I think it's very clear it's you know they're quite obviously severe diseases and you know it's important that there is deliberation around it but Sheela I think you want to come in don't you? Yeah I think in this criteria I think we've acknowledged that in the very rare diseases they are often very complex, they often have multiple co-morbidities associated with them and therefore the kind of impact on the quality of life is much more magnified in these than it would be in more prevalent or common conditions and I think that's what we're trying to capture in here so as Helen rightly says it's really difficult to articulate on paper because each rare very rare condition has its own complexities and its own unique kind of co-morbidities that are associated with them and we want to make sure that we're not disadvantaging those patients so that's why we've kept it as more of a subjective piece but in terms of our scoping exercise that we undertake when we're collecting evidence this is where we get to see how complex these conditions are so patient groups, clinicians and other people who respond to these exercises are the people that we kind of turn to help inform us of this. Thanks Sheela, thanks Helen. Helen I'm just going to come to you next in terms of Sheela's description of needing discretion and some of the criteria requiring a form of judgment from the topic selection oversight panel when making their decisions. Can you explain how the
panel would go about applying that discretion and how we would communicate where that has happened? Yeah you know we know this is complex, it's difficult as Sheela's just said you know we look to our stakeholders as well to help build that evidence base when we're considering all of this so we're you know we're not doing this you know in an isolated room with no input from anybody but in the same way that committees kind of deliberate over evidence you know we want to make sure we have the right people on the topic selection oversight panel and we've you know we've made proposals to amend the people who are around the table when we're taking those decisions and we have to weigh up the information that we've got. And take a judgement on the criteria and how that fits. We are proposing that we will be much more transparent about the decision making so where we are taking those deliberations and where we are applying judgments, we will be explaining that to people so it will be much more understandable why we've made a decision on whether a criterion is met or not. So you know hopefully people can start to see how we take those decisions and build up a bit more of an understanding. Thanks Helen and Lori? Thanks Jen, I just also wanted to say that where Helen is saying that we will be more transparent that is not just necessarily with the company who we do have quite a lot of dialogue with, that will be for all stakeholders and that will be displayed quite clearly on the NICE website as part of topic selection changes on the transparency that we're doing as well. And what I should have said about the challenge point earlier was that we're looking at trying to mitigate a lot of these challenges by being more transparent and having that earlier dialogue with companies and stakeholders and part of that is maybe changes to how we may scope, so rather than putting a label on it very early on we may just go out as a health technology evaluation per se with no label and gather that evidence through scoping before the topic selection oversite panel actually try and make that decision on a route in because it's that scoping exercise that's really important in giving us that that extra layer of detail for us to really be reassured that we're making that right routing decision for everyone.
Thank you Lori. Helen I'm just going to come back to you just a linked question really around that judgment and especially criterion 3. Where we've had a question about whether we have considered or whether we could consider using a QALY shortfall method or a quality adjusted life year shortfall method as we have proposed with the severity modifier. Could that help to make criterion three more specific? Yeah, I think when we were going through the methods development and the HST criteria there was consideration around that. I don't think it's necessarily going to be that useful at this
at the point that we would be making the routing decision so it's something that we certainly can take away. We have considered it but we can take that away and reconsider whether you know that type of information would be helpful, but I think we're not necessarily in that position at that stage when we're making the decision and it might be quite challenging to be able to do that. Thank you Helen.
Sheela, we've had a few questions around technologies with potential multiple indications, a wider portfolio than the indication we might be considering for selection for the HST programme. Could you just give us some rationale in terms of why we've set the number at 500 around the total estimated eligible population for all of the different indications? Yeah really good question Jen and again this was quite a complex issue to try and resolve and I think in the stakeholder conversations we had a lot of feedback received from stakeholders is that we should try and discourage and work with industry and other people developing these technologies to make sure that they're getting an appropriate return on their investment and so we've tried to think about how we can ensure that the very rare conditions are well supported and routed to the programme but of course where there are multiple indications that opportunity for return becomes a much wider opportunity so, we're trying to kind of help ensure that the very rare conditions get into HST when they need to be there but of course help manage and support the research and the investment that needs to be undertaken in order to bring them there so it's trying to kind of balance those things out and I definitely recall hearing from a number of stakeholders in that consultation that said that they were supportive of trying to think about how we encourage and secure appropriate and fair return on the investment that's undertaken. Thank you Sheela. Helen if I come back to you. There are a few questions that have come through that reflects on the poll that we did that suggest you don't say that and NICE can do more than three and we do do more than three within any one year if that reflects the topics that are relevant for HST but I think there was maybe an expectation that the review would result in more topics being routed into HST, potentially increasing access to treatments for rare condition. Can you explain why this might not have happened in the way that expectations may have been raised? Yeah, I think we tried to be quite clear about what the scope of this work was about and you know it wasn't about trying to work out you know can we get more topics going through the HST programme or fewer topics going through the HST programme. What we realised
from the experience that we were seeing is that the criteria were had been set for a while you know we've got a lot more kind of history of decision making and that we were you know there were certain criteria that just redundant that were unhelpful and we really wanted to help to try and clarify what the programme really was intended for and I think that in a way previously was probably lacking the vision, the principles why we have the programme and really that was never up for you know for it to be updated or changed in any way and but we did feel what would be the most useful thing would be to help to clarify what the programme is intended for and refresh the criteria to better reflect that and hopefully make the decisions that we're taking more understandable for our stakeholders as well and easier for the people sat around the table you know making those those hard decisions really. Thanks Helen. Sheela you mentioned the manual of prescribed services in your presentation, and we refer to it as well don't we in the consultation documents. We've received a question just asking whether the manual or schedule four as it might now be formally known as does it in fact add a fifth criterion to the proposed four that we've outlined here today? It's an interesting one and we again this was this kind of reflects the original criteria that talked about the you know the technology must be rooted through a highly specialised commission service and this has caused some confusion in terms of how the current criteria have been applied and interpreted so we're acknowledging the changing NHS structures of course with ICSs and various different kind of approaches that specialised commissioning will be taking in that fold and really thinking about the fact that not all technologies that might be considered for HST will have an established highly specialised service in the way that NHS England defines highly specialised services so we've started to see technologies that need some form of national coordination in terms of delivery but not a highly specialised service label and I think the reason for taking out the reference to highly specialised commissioning structures and the manual was to reflect those changing structures and to reflect the changing landscape of the different technologies that we're seeing and really putting the hands of deployment and delivery back into the hands of the NHS because that's the job they do the job that we do is to ensure that it's value for money for the system, it's clinically and cost effective and we then leave the deployment and operational kind of delivery to the NHS so that's the reason we've decided to exclude the reference to the manual and to any commissioning structure sort of backup that the NHS might have. Thank you Sheela.
Helen, just coming to the criteria that talk about where other treatments may already exist in terms of making a judgment as to whether to root a new technology to the highly specialised programme. Can you talk about what it means to have a significant additional benefit for those affected to then be selected for highly specialised technologies and also, I'm considering what gene therapies may present as well in terms of additional benefits? Yeah it's again difficult and particularly with these types of conditions you might have different treatments working on different aspects of the condition as opposed to kind of treating and underlying the you know the underlying cause of the disease as well so I think there has to be kind of recognition about what current treatment or current standard of care looks like, how that impacts patients at the moment and how do we think the new technology will or could change that and I think quite a bit of that will come from feedback as well about the expectations of the treatments from clinicians, from the patients and from other stakeholders. So you know it might be different in different circumstances but you know you have to take the base level of what's there and take a judgment on you know how we might expect this treatment to have such an impact on a patient and take it from there but Sheela, I think you want to come in? Yeah, I mean you've kind of nailed it but I think it just goes back to what I was saying earlier about these very rare conditions having multiple co-morbidities so we may have a situation where there's a treatment available for a particular condition but only addresses one part of those co-morbidities and another treatment may come along that addresses something different and the benefit, the combined benefit might be you know different. It might be greater, and it might have a different impact and I think that's the assessment we're trying to make when we're talking about significant additional benefit. Acknowledging those multiple co-morbidities in that space.
Thank you, thank you both. So Lori can I just come to you for a question around we've been asked whether the changes proposed have been sense checked if you will using previous topic previous topic section considerations of both highly specialised technologies and standard technology appraisals and what was the outcome of that? Yeah so the team did what we what we call an impact assessment and I think Sheela may have also mentioned it in when she was going through the slides as well where we looked at the current criteria and the new proposed criteria and we looked at those against historical topics particularly those ones that have been challenging so that we knew that they made sense and they've also been looked at current topics that are in in the programme work, programme now and also those that are that are looking coming through horizon scanning. So in the horizon scan of the topic selection, we're looking at topics that are two years ahead of their anticipated marketing authorisation so we've looked at those as well and what the what the team come up with is that there is no less topics coming through and that they are very similar to the same topics that are getting routed now. It's just that our hope is that the new refined criteria are clearer and more predictable with those numbers of eligibility that stakeholders have asked us for. Great thank you Lori and Helen I think final question just noting the time that we're getting close to five o'clock and the end of the webinar. What support do NICE provide to manufacturers that are developing new innovations in this very rare space? Yeah good question and you know we want to be there supporting and facilitating so I mean companies have got a number of options to talk to NICE and so there's the NICE Scientific Advice to start with and we've got the Office for Market Access where we can pull together in a confidential environment and the right stakeholders to talk about a particular technology and you know of course we're when we take a topic through an evaluation we do have lots of support, the team do support companies particularly companies that haven't experienced NICE processes before to help support them through that so I think there's kind of a wealth of opportunity for companies to come and speak to NICE and we would always advise companies to come and talk to us early about this. You know there are a number
of initiatives new initiatives that are happening. The MHRA changes so the innovative licensing and access pathway where stakeholders are getting together much earlier so hopefully you know that those early discussions will continue through the life cycle through to you know health technology evaluation so lots of really promising changes coming. Thank you Helen. So just as we're drawing to a close of the webinar we spoke about this earlier but just to remind everybody that there are two more webinars that we're running over the next week so we'll be focusing on the process review changes that we've proposed in the consultation next week along with a separate webinar on methods again so we started with methods and we'll finish with methods and but on the final webinar for methods review we will be focusing on the modifier element of the consultation as well so please do feel free to register attendance at those webinars but again they'll be recorded so if you're not available to attend live you can watch them again later on when we publish them on our YouTube channels. And so, the final thing just to say as well as saying thank you very much for coming here today again and it's just to say please do get involved in the consultation and please do go and take a look at the consultation documents on the website. Please also do go and look at the draft manuals that we propose so the draft guide to methods and processes for health technology evaluation and the rough topic selection manual also and please do inform us of your thoughts and if you're able to send your responses in before the 13th of October absolutely that would be absolutely brilliant because we'll be ready and waiting to receive them and work on them as quickly as possible.
So thank you very much again for your time today and for all of the really great great questions that we've received. We'll be taking those forwards in terms of further thinking as well and getting ready to receive your responses thank you very much everyone bye-bye.