NICE topic selection methods and processes review explained virtual event series HST focus

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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.

2021-09-06

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