 Hello, everyone, and welcome to this My Loan of Patients here at webinar. Thank you so much for taking the time out of your evening to join the webinar, and we really appreciate it. So my name is Kate Morgan, and I am Head of Policy and Access at NPE, and I'll be responsible for moderating the webinar today on reimbursement, HDA and patient involvement. These are topics which can seem very complicated, I think, to patient advocates, but are really important for us to understand and engage in as they have a material impact on the standard of care in specific countries across Europe and on patient outcomes. Just before I introduce the speaker, I'll run through the agenda briefly in the administrative details for the webinar. So just to let you know, the webinar is currently being recorded and will be posted on the NPE website after the session has finished. The webinar will last for approximately around one hour, and it will consist of a 40 to 45 minute presentation from our speaker, Neil Bertelsen, followed by a 20 minute Q&A session. During the Q&A, please feel free to ask any questions that spring to mind during the presentation. To let you know, there are two ways you can ask questions. Firstly, select the raise hand button to signal that you have a question. This will alert me that you have a question and I'll unmute your microphone so you can ask it directly. Secondly, if you would prefer me to read out your question, please just type it into the chat box, which appears in the panel on your screen. So that's the admin part over, and I'll repeat how to ask questions after the presentation. I'd now like to introduce our speaker, Neil Bertelsen. Neil is former chair and current steering committee member of the Patient and Consumers Interest Group within HDAI International, a collaborative network of HDA bodies and related stakeholders. He has a wide range of experience working with HDA bodies, patient advocates, an industry on patient involvement, and has a lot of experience as a patient advocate himself. So without further ado from my side, I'll just hand over to Neil to begin his presentation, and I'd like to say thank you for joining us today. Thank you so much, Kate, and I hope you can all hear me well. So as Kate said, I'm Neil Bertelsen, and I'll just kick off straight away with a few declarations of interest. I do some voluntary work with patient groups, with HDA bodies, and I'm also, as well as the HDAI, I'm also involved with patient-focused medicines development, which is another organisation looking at similar areas. But I do paid work as well, which includes working with patient groups, working with the industry, working with HDA bodies, and also working on some IMI European projects, again, in the same area. But I'll take off straight away, set in the context. So why am I here? Why am I doing this webinar? Well, as Kate said, I've been involved with the patient and citizens' involvement in HDA interest groups, very long name for a particular group, but we've been going for about 12, 13 years now, and we're a collection of patient groups and patients, academic researchers, people who actually work within health technology assessment bodies, and people from the industry. And we look for gaps. We look and see how there's patient involvement happening in HDA. We work internationally to share good practices, and we bring these disciplines together to see if we can find better ways of involving patients and patient groups in health technology assessments. So the way I'm going to structure the talk today is we'll have a few slides, just really going back to basics around HDA, and that's really for anybody who's on this call who is a bit unsure about what HTA is and why we have it. But then I'll deep dive into patient involvement, and we'll have a think about why do we have patients and patient groups involved in HTA processes, and once we've covered that off, I'll talk about some ways that that involvement happens. And at the very end of the presentation, we'll think about some different approaches from the advocacy groups towards HTAs, and particularly what can you do if there is no HTA body in your country, or perhaps you don't have patient involvement processes within your HTA. So I'll kick off, first of all, with the basics. So why do we have HTA at all? Where did this come from? Well, it came from this thing that we hear about all the time, which is that there's not enough money to go around. So with the best will in the world, healthcare systems would like to provide the best possible care for all the patients within their healthcare system, but there just isn't enough money to go around. And so then you get this question, which is how can the system decide who gets what? Which treatments, which processes, which kinds of healthcare are available to different people? Now, the old way of doing this was very much based on connections and those who shout loudest. So you could get decisions being made around healthcare just because there was a lot of noise around a particular disease area, a particular condition. Or you could say we will make that choice by reviewing the evidence and we'll make a judgment based on the evidence that exists today. And it's this last option, option B, that is HTA. So when we think about HTA, I think we have to accept that HTA, first of all, stands for Health Technology Assessment. And the word technology is a bit of a silly word to use, but it is used because what they mean by a technology is a medicine or a medical device. So it's something that is used, a kind of technology that they've used to improve healthcare and the assessment happens to see whether that has value. But on the left-hand side of this slide, it's just an acknowledgement that health technology assessment differs from country to country. Each country does it in a slightly different way than the other. But in essence, they're trying to do the same thing. They're trying to say, what evidence do we have? What exists to show us how this new technology compares to what we have already, what's already available? And by doing that assessment, they're trying to ask themselves, should we use this technology? Should we use this medicine in our healthcare system? Should we pay for it? And if we are going to pay for it, how should we best use it? When should it be used and in what kinds of patients? Which patients will most benefit from it? And then if we think a bit further about what that means, is that HTA is used really to help make policy decisions, health policy decisions around the provision of healthcare. It determines how healthcare systems can spend their budget. As I say, it's based on evidence rather than lobbying. And they have a process that they can repeat over and over again for different medicines in different disease areas. And so having this repeatable process is part of the system, is part of the whole role of HTA, is that you can do these assessments over and over again with different medicines in different diseases. But obviously, if the evidence doesn't convince the HTA body that a new medicine has value, then that usually means there's no access to that medicine in that particular country. So it does become a real gatekeeper for our access to medicines. I just want to cover up just a few terms that you might hear. And I don't want to cover this in a great deal of detail, but just to acknowledge that there's different ways that health technology assessment is conducted. So some countries say we're going to use a term that they call clinical effectiveness. And what they're saying is we want to see how this new medicine performs clinically compared to what we have already. So what are the clinical benefits of this new medicine that are better or that are new compared to what we have already? There are other healthcare systems that use something they may call cost-effectiveness. And in cost-effectiveness, what they do is they calculate what are the supposed health benefits of this new medicine? How do we measure those outcomes from the new medicine? And let's look at the cost of the new medicine and is the cost of the new medicine reflective of the better outcomes that this new medicine is promising? So that's cost-effectiveness. As well as that, sometimes on top of that, you have a very simple question which they call budget impact. And budget impact is as simple as can we afford it. We have so much money this year to spend on healthcare. If we were to stop paying for this new medicine, will it break our budget? I just want to give you those terms because when you're reading about HTA, you often come across this terminology. So I just wanted to explain the difference between those systems. So let's move on to why. Why do we need patients involved in HTA? What's the process there? We looked at that within HTAI, the group that I've been involved with. And there was an international piece of research that asked lots of different stakeholders in different countries why patients should be involved in HTA. And it came down to these five areas. Relevance, because patients have knowledge that's unique, that the doctors don't have, that the health economists don't have, and that the HTA body doesn't have. It's fair. Patients should have the same rights to contribute as other stakeholders such as doctors within the process. It helps balance equity. So understanding how you trade off paying for treatments for one disease against another. If you can speak to patients and understand how patients experience their condition, it helps understand what are those trade-offs that the healthcare system needs to make. It's about legitimacy of a decision. So HTAs make decisions or make recommendations that lead to decisions. And obviously, if patients are going to be affected by those decisions, then it's legitimate that patients should be involved. And the final one is around capacity building. And this was recognition that if health technology assessment bodies don't involve patients, they will never learn how to involve patients. And similarly, if patient groups don't get involved in HTA, then they'll never learn how to get involved with HTA. So this final reason was an understanding that actually the involvement process of having patients involved in HTA systems helps to bring capacity, helps to build capacity to have more patient involvement. And I just want to focus on that very first one, because I think this is the key. This is the key to patient involvement in HTA, is that patients and patient organisations, they have unique knowledge about how a particular condition, in this case myeloma, affects everyday life. And they can make that specific and really pull out the kinds of impact that myeloma has on people's lives, on people's ability to work, on essential tasks. So patients and patient groups are really good at understanding how to quantify and how to describe the impact of a condition in a way that HTA bodies find really valuable. Obviously patients understand the real pathway that they go through in getting diagnosed, getting healthcare, and what some of those challenges are. And they have experience of different treatments. They've been through various, often various stages of treatments, different kinds of treatments, and they can share their experience, good and bad, of those different treatments. And finally, but it's very important, only patients can tell you what they expect, what they're hoping for, what things need to be made much, much better in terms of symptoms or quality of life that they're really looking for for new generations of potential treatments. That relevance is important. But I would argue that it's not just about doing the right thing, HTA bodies actually need patient involvement. So it's not just a nice thing to do, they need to do it. And why do they need to do it? Well, when HTA bodies first get formed, usually, in fact, in every case, they say, we can do this on our own. We're experts in this. So we go to the pharmaceutical company and we get the evidence from them. And we know lots of doctors and we know people who are experts in health economics. And so we can all sit down together, we can look at the evidence together and we can make a decision. And they try to do this. And it falls down, it goes wrong because there are certain questions that these people cannot answer. So questions like, are there specific needs of patients that this committee doesn't know about that we need to know about to help us make this decision? Often, in the evidence presented by a pharmaceutical company, they'll have lots of different outcomes and benefits they're saying that this new treatment brings. But which of those are most important to patients? Which of those really matter? Sometimes, these new treatments companies will say, this could change clinical practice. It could change the way that you deliver care for this particular disease. And patients need to be consulted to know how that change in clinical practice will really affect them. Often, one of the critical questions HTA bodies are asking themselves is, is there a subgroup of patients, a particular kind of patient that is most in need, that would most benefit from this new technology? And it's not always about clinical measures. It can be, for example, people who live in rural communities may particularly benefit from a new kind of treatment than others, for example. And so, really, they want to find out from the patient perspective what is likely to be the impact, both positive and negative, of a new treatment. And so, they start involving the patients, and in some cases, caregivers, to give their point of view, to give their perspective. And when I speak to HTA bodies that do this, they say it improves the quality of the decisions they make. That actually that input from patients and sometimes from caregivers as well helps them make better decisions. And I'll go through an example of that later in the presentation. OK, so if that's why we're involving patients in HTA, how does it happen? Well, it happens really differently from place to place. And I have to say, in a lot of countries, it doesn't happen at all. And you might be living in one of those countries. But first of all, before I go into the exact how, I just want to just explain what we, in HTAI, what we mean by patient involvement. So there's two ways of looking at this, and we think that both are important. The first, which is the top row, is patient participation in the process. So that's being, you know, attending meetings, being invited to the committee meetings, being part of a consultation process. So the actual taking part is a really important and crucial part of patient involvement. But alongside that, there is what we call patient-based evidence. So that's evidence that exists out there in the literature about patient experiences, patient preferences. Maybe other studies have been asked, using, for example, patient registries, that gives more information about patient's experience and quality of life. So you have these two things working in tandem. You have the involvement itself, and you have evidence that underpins that. And I think the role for patient organisations is obviously in taking part. So it's in that first row. But where it crosses over is increasingly, we see HTA bodies using what we call patient-group submission templates. And those submission templates are a sort of standard form that is given to patient organisations to say, can you answer these questions for us? Can you tell us more about the disease and its impact and provide that in a written form to us? And we will consider that as part of the evidence package that we consider when we're making our decision. In terms of how that engagement process, how that involvement process works, as I've said before, in some countries it doesn't happen at all. There is no opportunity for patient involvement. And there's a lot of work that happens behind the scenes in trying to get more countries to have patient involvement processes. But often where it really starts is on this left-hand side with informal discussions. So maybe a HTA body doesn't have a patient involvement process, an official one. But they're looking at a particular treatment and they think, actually, we have too many questions about this. We need to speak to some patient groups and get some clarity. And so they may have informal occasional meetings to discuss a particular disease area or informal meetings to discuss their HTA processes to see whether patients can be involved. The second step is open consultations. This is often the first step that HTA bodies make before they go to full patient involvement. It's usually done via a website and they will say, you know, we're looking at this particular drug for this particular disease area. If anybody wants to comment, you can use this website and comment. And that's usually open to everybody. So it's open to patients and patient groups. It's open to nurses and doctors. And it's open to the general public. So that generates an awful lot of responses. And what tends to happen over time is that the HTA body realises that there's too many responses from too many different kinds of organisations and stakeholders. So they start to build formal processes and say, oh, we do want to hear from patient groups, especially about this. So they build a process in to take that feedback from patient groups. And I should just say that most of the HTA bodies do mean patient groups. There are a few situations where they want to speak directly to patients. But actually, for assessing a new medicine, it is mostly patient groups that they go to. They want that patient group perspective. And then the final box here is being represented at a committee meeting. And I realise I'm going to have to speak a bit faster. I'm going too slow. So this is how it can look in a more sophisticated HTA body. You have all of these different steps. I won't necessarily go through every one of these. But I think the important one is gathering evidence for more stakeholders. This second arrow from the left, that's really where patient groups are invited to provide detailed information about how patients experience their disease and what kinds of things that the committee should think about. The next box along is the committee meeting where they discuss all the evidence. Some of these agencies invite patient group representatives who can bring patients with them to answer any questions that the committee has or to give a presentation to the committee. I should just say that when a decision is made, most HTA agencies do not allow patients to be part of that voting on a decision. They often say citizens can represent the general public and be part of that vote, but the patients can't. And they're trying to separate conflicts of interest here. They're trying to say the health technology assessment process is there to protect the funds of the whole healthcare system. And so they want citizens to be part of that decision-making process for other patients because it's in the interest of the citizens to protect that healthcare system. I just want to look at these questions. So these questions that you can see on the screen now are from Scotland, but I don't want you to think about them as Scottish questions because actually you see these questions used in many, many HTA bodies around the world. How does this condition affect day-to-day life of people living with it? HTA bodies want to know that. How well do the medicines currently work that you have today? Has the patient organisation been able to find anybody who's tried the new medicine, perhaps in a drug trial, for example, who can give us some insight what that was like? Would this medicine be expected to improve the quality of life of patients and their experience of care and be specific? If it's going to lead to an improvement, exactly what is it going to improve? What do you expect that improvement to be? What kinds of impacts would treating a patient with a new medicine have on the family and carers? And a really important one, which is really vital this is covered off, is one of the disadvantages of the new medicine. So if a HTA body hears from patient groups, this is fantastic, it's perfect, it's everything we wish for, there's no downsides to this, they get really suspicious. Because there's no medicine that doesn't have some kind of downside to it. What HTAs are not looking for for patient groups, and they complain about this a lot, is patient groups sometimes look in the scientific literature, look at the published clinical trial results for this new medicine and they repeat all that information in their submission to HTA. But HTA get that information anyway from other sources. So it's really important when you're involved in a HTA process, that you're bringing really clearly the patient perspective. And you don't muddy that with data or evidence that the HTA body are getting from other places. I'm often asked about this third point, which is about emotion. It's not that you can't be emotional, it's okay to say to a HTA body, this disease is devastating, this disease is ruining people's lives. That is absolutely fine, but you need to go a step deeper and be really specific in how it is so devastating. Exactly what is it about this disease or the treatments that make it so devastating? Be as specific as you possibly can. And I'll do an example of that now. So this is from England, but again, I don't want you to think about this just as being from England, because I want to focus on the kinds of information that a HTA body finds useful. So this was a treatment, costly treatment for a disease called ulcerative colitis. And what happens in this disease is that at some point in time, the symptoms get so bad that you have to have surgery, you have part of your bowel or colon removed, and then you have a stoma bag or a claustomy bag. You live for the rest of your life with this bag attached to your side. And this new treatment came out, it was expensive. And what the data, what the evidence showed about this treatment was that it worked, but it worked before time. After a time, a few years depends on patients, how they responded, but the symptoms would come back and the patients would still eventually need to have this surgery. And so from a health service perspective, this is a clear no because you are, you're still going to have to pay for the surgery in the end. In the end of the day, the patient's quality of life ends up exactly where it would have done, with the surgery, with the bag. The only difference of having a new treatment is that you delay that surgery by some years, but you have this massive cost of paying for the drug. So in principle, when you look at this objectively, the new treatment doesn't add anything. Patients end up in the same place. It's just cost a lot more money to get there. But the patient groups, and there were patient groups in attendance at this meeting, and so they were able to take part in the discussion. And what they were clearly able to show is that there was a particular value for patients to delay that surgery. And in particular, what they were able to do, and if I go to the next slide, is they were able to answer those questions that I showed you earlier with specifics. So they were able to clearly say how the disease debilitated people. So exactly what happens to people who are not treated properly or can't get rid of their symptoms? How does it affect their day-to-day life? What was really important here was the comparator was surgery. It wasn't another treatment. It wasn't another medicine. It was surgery. And what this group were able to show is that surgery has immense problems for people. People often get really bad with ulcerative colitis in their late teens, early 20s, going into their early 30s. This is exactly when people are studying, going to university or starting a career or starting a family. The surgery also makes women infertile. And so the patient group were able to clearly specifically point to different patient populations that would be really harmed by surgery. And particularly this concept... Well, it was not a concept. This reality that women, young women could be made infertile for the rest of their life by this surgery was a real point that hadn't been considered in the discussion so far. So by being specific, they were able to show that there was a need to delay surgery. And that delay in itself had a value, an immense value on people's future life experiences. Let's just cover off some advocacy approaches. And this is really top line. And we can talk about this more in the Q&A. But obviously, you may or may not have HTA in your country. You may or may not have patient involvement processes. So I think what is the opportunity? Well, if you're in a country that has HTA and has a patient involvement process, then it's being visible to that HTA. So most HTA bodies will have a list of patient groups that they have a database of and they contact whenever there is a particular treatment that they're reviewing in their disease area. So the first thing is make sure that they know about you. Make sure that you're on that list. And the second thing is be prepared. If you can see that a new treatment's coming down the line, you know, it's in the news amongst the myelomid community, then you're going to know that sometime soon it's going to come to your HTA body. So be prepared to develop the evidence that really shows what currently is the gaps in patient need for people with myeloma specifically in your country. But if you don't have this opportunity, what if you can't be involved in a HTA process? Well, it all starts with making the arguments as to why the HTA process needs to have patient involvement. And we do that at HTAI at an international level. We put HTA bodies that have no patient involvement process. We put them in touch with others that have won or have started one recently and we encourage them to learn from each other. But I think from a patient group perspective, it's making those political collections with, you know, the Ministry of Health, et cetera, to show and make them hear over and over again that patients need to be involved in these kinds of healthcare decisions. Also, if there's any consultations happening, might not even be about HTA, it might be a national consultation about the healthcare system and healthcare delivery. Be part of those consultations. So put your name forward and start... So people start seeing your name and your organisation as a partner in these kinds of consultations. Even if there's no patient involvement process, approach the HTA body and let them know you're there. Let them know you're there. And if ever they're assessing a myeloma treatment, that they can always call on you. They can always pick up at the phone or set up a meeting. So be really mindful that sometimes you have to push yourself out and keep reminding the HTA bodies that you're a potential resource for them. Now, the media, this is something I feel quite difficult about, to be honest. When we... Before we had patient involvement in HTA, the way that patient groups dealt with decisions made by HTA bodies was use the media. So a drug would be turned down and the patient groups would go on the media and talk about why this was unfair and how this was impacting people's lives. But then what happened quite quickly is that for every disease, every time this happened, different patient groups would be coming out with the same story. And so it started to lose its value as a strategy. And what also happened at that time was... Because these decisions are very difficult decisions to make. A HTA body doesn't say no because they're evil. They say no because the evidence provided by the company didn't convince them that there was value. That's really why they say no. And so what started to happen was it created a divide between the people who make the decisions and the patient groups. The patient groups sort of shouting really loudly from the media while the HTA bodies were trying to do their best and work out how to make better decisions. So when you use the media, you can do it. I'm not saying it's wrong to do it, but think about using them in a way that really exposes the fact that patients are not involved in this decision-making process. So it's not just that it's unfair that a treatment hasn't been approved for reimbursement. I mean, that is unfair, but it's also unfair that this decision was made without any representation from patients or patient groups to make this decision. And I think that more nuanced approach would be my recommendation. In either situation, whether you have patient involvement or not, think about the evidence. So this sort of evidence ladder we have on this slide, this is what I've observed over many years as HTA bodies have started to include patients and patient groups. The involvement starts, and initially what patient groups did was bring stories, was bring sort of verbal stories and perspectives and quotes, really focusing on that emotional component of a disease. And what they learned through this process was, actually, that's not getting us far enough. What we need is evidence. We really need evidence to take this to the next level and to show that we're not just bringing stories to HTA bodies, but we're actually substantiating the main points of those stories with our evidence. And then the evidence becomes more robust over time. And then they develop capabilities to actually generate, you know, quite complex evidence that sometimes is evidence that the HTA bodies and the industry cannot generate, as they don't have the same resources and capabilities in terms of access to patients. I would argue that this ladder also applies if you don't have a HTA body or you have a HTA body that doesn't include patients. Because when you're doing, for example, you're meeting with a policymaker around HTA patient involvement, actually having evidence to back up your policy asks is just as important as it is for HTA. Or if you're developing a media article to show why patients are being left behind because of decisions made by HTA, then having the evidence to back that up makes your story so much stronger. So I would argue that you need evidence either way. And it's, if I want to sort of make it simpler, you go from we want this to we want this. And it's specifically important because we have evidence to show X, Y and Z. Rule two, being really specific about what it is you want. So our evidence highlights that it's this need and that need and the other need that is really causing problems for patients with this disease. And that's why we're asking for A, B and C. So being as specific as you can with your asks and the reasons why you're asking for what you're asking for is for me the key to this. And it works with policymakers and it works with HTA. I won't go through this in detail, but there's lots of ways of generating this evidence. Interviews, surveys, focus groups, some patient groups, you know, quite big, well-financed patient groups actually create their own patient experience registries so they can capture this information over time. You know, and how this is provided to decision makers. Obviously, I've talked about patient submissions to HTA, so that's one way, expert testimony. And that could be at a committee meeting, but that can also be at these one-off meetings, these ad hoc meetings that HTA bodies sometimes organise. Yes, it's okay to have patient stories, but include the evidence in there. Back up what your patient stories are telling you with some evidence. And you can use that with policymakers to highlight the need for change and you can use that with the media to create a pressure for change. And I'm still doing okay for time, nearly finished, you'd be glad to know. So this is just more details about if there's no patient involvement or no HTA. So again, think about those political connections. Think about how you make the argument for more patient involvement or some patient involvement in your reimbursement or HTA decision-making process. Think about getting case studies of how it works in other countries so that you're not just asking for something with a blank sheet of paper, but you can say, for example, in this similar country, they have a patient involvement process and this is how it works. So you actually come there with ideas and solutions and examples. If you're not invited, introduce yourself anyway. So as I've said before, try to set up a meeting with the reimbursement authority or the HTA body and just introduce yourself. If they don't want a meeting, just develop a CV, talking about your group and the connections you have and the kinds of evidence you've generated locally so that they see you as a credible and valuable resource that they could turn to you in future. And as I've said before, use the media appropriately. Yeah. I'm really going to finish up now with just a quick point of some useful resources. So the group that I'm involved with, HTAI, has a lot of information for patients and patient groups. And I'm assuming you'll get these slides afterwards. So I'll check that with Kate, but you can have these slides afterwards. And so that's the links here are all the latest links. I've checked them today. So that link will take you to the HTAI patient and citizens involvement group and it will take you to resources that are designed for patients and patient groups. This is a really good resource. So this was developed by Pee Coda, which is the name of an agency in Canada. But again, don't just think of this as a Canadian resource. This has some really good general advice about how to submit information to HTA bodies. This could apply anywhere that you have a patient involvement process in HTA, really, really good, really well laid out. And they give examples of the kinds of sentences they look for when they get information from patients. So throughout the book, it'll say, OK, this is your point. Think about seeing it like this. Think about using your evidence this way. So really clear guidance. NICE also has lots of guidance. Again, it's based on the English system, but a lot of their guidance applies more generally. So I pulled up this one, which is their hints and tips when preparing to be a patient expert. And it particularly has some guidance on what to do if you're invited to a meeting, what kinds of things to concentrate on. You know, how do you bring the facts to life as a patient expert for the committee? This is an old resource, I think it's 2010, but don't let that put you off. The reason I've included it here, first of all, is seeing multiple languages beyond English, it's Spanish, Mandarin, Italian, Polish and Greek. It's quite a meaty document that goes through what is HTA and what is the patient contribution to HTA. So a really good background read if you're wanting to find out more information. And then the final one here is the ethical guide. We developed this at HTA AI, but obviously if you're thinking of doing things like surveys or interviews with patients to develop the evidence you need, there are some ethical considerations you need to keep in mind. And we wanted to make sure there was a simple bit of guidance that you could refer to. It talks to all of the main stumbling blocks that you might not think about around the ethical challenges of gathering information directly from patients. And I'm going to stop talking now, you'll be pleased to know. And we'll hand back over to Kate for the questions and answers. But thank you. Thank you so much, Neil. That was a really comprehensive and interesting presentation. And I can confirm that we will be circulating the slides after the webinar. And the webinar will also be available on our website. Just before I open the floor for questions, just so you're clear on how you ask a question, firstly, you can select the raise hand button, which is literally a button on your screen that has a hand on it. And that will alert me that you have a question and I will unmute your microphone. And secondly, if you would prefer me to read out your question, please just type it into the chat box and that will come directly through to me. So does anyone have the first question to ask, Neil? Oh, Fonzo, I can see you have a question. Thank you, Kate. Can you hear me well? Yes, we can. OK, thank you. And thank you, Neil, for this very, very good presentation. I think it's been pretty clear. So my question, I hope it's not out of a scope, but it has to to see a little bit with the initiative that the European Commission together with UNET started like a couple of years ago, if I'm correct, of having an HTA in joint actions or joint decisions. So I mean, I know that the patient involvement in this process has been quite low to my understanding. So I have a question from you and I'm probably with your expertise to be able to answer. So as I understand the UNET, what they are trying to do in the European Commission is to have a kind of joint decisions manner to produce a kind of common HTA reports at the European level that can serve as a basis for the different member states to make their decisions. So my question is to which extent do you think these actions are really going to have an impact on the access scheme to new treatment for patients taking into account that each member state can later make their own decisions on their own, given that they have different HTA models and so on. So do you think this is something that is going to change the status quo of the HTA and improve the access schemes or is just something like a good initiative that probably will end up in nothing? OK, so that's a really good question. It's something that we worry about, to be honest with you. So to be clear, the proposal, the HTA harmonization proposal is looking to split the decision making into two parts and to say at the moment what happens is in your country, if you have HTA, the HTA body will look at all the clinical data they will decide where the value is and then they will make some assessment on whether they're going to pay for it, whether it's affordable, whether it's cost-effective. What the European Commission would like to do is to have that first part, the clinical analysis of the evidence to happen jointly across the EU states. And that would produce a report which is around the clinical effectiveness of the new treatment. So very similar to the kinds of ways it works in France and Germany where they look at clinical effectiveness. But then the second half of the assessment, which is deciding the economics of whatever the treatment is, that will still happen locally so that you'll still have a HTA body in your country and it will be that HTA body that decides still whether we're going to pay for this or not. So the idea behind the proposal is that it stops duplication around assessing the evidence, but you still have local decision making. And excuse me, I'm going to cough, so I'll just go on mute a second. It's not corona. What we worry about is that we see the need for patient involvement at both those levels. So we think that patients should be involved in the clinical assessment because patients have particular perspectives about what's important and relevant to consider in that clinical effectiveness assessment. And obviously we think patients should be involved at the local level because it's at the local healthcare system that patients have got knowledge about how they experience healthcare for that disease in the local setting. And we worry that patient involvement gets watered down, especially at that first level, because at the moment we have some countries that have very strong patient involvement processes throughout and we have other countries that have less or no patient involvement. So we worry that when you try to pull all this together into a harmonized system, that you'll end up having less patient involvement than we have today. And so that's something obviously that we discuss and we work with HGA bodies and patient groups work with the commission to try and make sure they understand the importance of patient involvement. Did that answer your question? Yeah, thanks. It was pretty clear. It's only the ones who want to apply this, but I have probably a silly question. So you mentioned that in the first part, which is the more fishable one at the European level, it's about assessing the clinical effectiveness of a new treatment or whatever. But isn't this what it's been done in the drug approver process before with the European Medicines Agency, like basically assessing the clinical efficacy and safety? So isn't it a little bit like overlapping? There's always been overlap between these kinds of assessments, but they're fundamentally trying to do something different. So the EMA is looking particularly at the benefit-risk profile. So they're not comparing with what standard of care is in Germany or in Poland or wherever. What they're doing is saying, in this clinical trial, do we have a reasonable view that the benefits that this new treatment is bringing outweigh the side effects and the potential harms that this new treatment can do? So they look at that in isolation. When you come to the HTA context, even if this is across the European level, you're trying to make much more of a comparison between what is this new treatment telling us that they have and what do we see in the real world from the treatments we already have? So it's a different perspective they bring to this. But there are moves. If you look at the EMA's 2025 strategy that they published a few months ago, there's a big element of EMA trying to align their assessments with what HTA are trying to do. So who knows? But I think you might see these worlds joining together at some point over the next five years or so. Okay, thank you. We're very clear. Thanks for the look. Thank you. Perfect. I can see that Nicole has a question now. So I'll just unmute your microphone. There we go. Thank you very much, Kate. And this has been a really exceptional presentation. Thank you so much, Neil. I wanted to ask if in any way pharmaceutical industry partners capture information on patient experience or quality of life or patient preferences, how that information might be able to be used in HTA either being submitted by the company or is it possible that that sort of data could be also leveraged by patient organizations? Really, really timely question this one. So thanks, Nicole. So this is starting to happen. And I say starting to happen because we have the very first few examples of patient preference studies being brought to HTAs. It's quite a new set of evidence for them to look at. And I think there's a lot of learning that needs to happen on all sides about what is the value of patient preference that is? What does a good patient preference study look like? What's the bad patient preference that it looked like? What methodologies become validated? And what methodologies are pushed to one side because they have challenges with them? And we don't know the answers to all of those questions yet. But it's an area that's gaining so much interest at the moment. Mainly driven by the FDA in the US who are really focusing on this idea of patient experience data. So they're encouraging companies at the moment, but it won't be long before they're mandating that companies bring this kind of evidence as part of their regulatory submission. And that means that this kind of evidence will be out there. We'll all be able to use it. So patient groups will be able to use it and HTA bodies will be able to use it and the industry will be able to use it. I think there's still a lot of questions about how it's used in decision-making processes. And there's always, if I'm honest with you, there's always a bit of a suspicion from the HTA bodies around these new technologies, around these new evidence bases. They need to understand them in a lot more detail to be able to rely on them for decision-making purposes. And that takes time. There is the project, the Innovative Medicines Initiative project, I and I prefer, which on a European level has started to look at those different methodologies and see how they can be used for decision-making in these early days. But we're all going to start seeing much more of this data floating around, much more noise around patient preference studies. I think the thing from a patient group perspective that I worry about is I would not like to see this kind of evidence being used instead of patient involvement. If you can think back to that slide I showed that said you have involvement in the process and then you have patient-based evidence. And for me, preference studies are patient-based evidence. And I worry that as decision-makers and the industry and others start to get hold of patient preference studies, they stop engaging with patient groups. So I think there's a clear story that we all have to tell which is that patient preference studies is an evolving field. We need to learn much more about it. But patient involvement needs to happen alongside that. And patients need to be involved in the design and the implementation of preference studies as well. They shouldn't be done in isolation. I'm not sure that answered your question, Nicole, did it? No, I think that's a brilliant response. Thank you very much. That makes perfect sense to me. So we have a few more minutes left for the webinar. So I'll just go to Hans, who has a question now. Hans? Yes, I have one question, maybe a bit in line with the former one. Thank you for your excellent presentation, by the way. I was wondering, because I was confronted with it once, that I spoke with a member of the HAA in my country about the involvement of patient representatives. And he was not very enthusiastic because he argued that patient advocates are biased in the sense that they always advocate in favor of the treatment. So what's your experience with the value of patient representatives? Should they also be involved in the decision-making or just give specific patient input to help the decision-making? What's your idea about that? Yeah, it's a thorny question. This is a difficult question. So we hear this a lot from HTA agencies that they don't want to do patient involvement because they think that patients are too close to the industry and they're suspicious that the patient groups repeat the lines from the industry. And of course, patients want access to new treatments. So of course they're going to campaign for access to new treatment. What I've seen over the years is patient groups are much more independent than that. So patient groups are able to... Sometimes the biggest critics, if you like, of the kind of evidence that the industry is putting forward. And I've seen meetings where it's been the patient groups that have really dug into that evidence and said, why are you not doing this? Why are you not doing that? So that's the first thing. Now, the whole voting right... I have two minds about this, if I'm honest with you. I think that HTA as a construct was there to protect the whole healthcare system. And so I really understand why most HTA bodies have voting rights for citizens but have input from patient groups. But I also understand from a patient group perspective that you want a voting right. You want to have a say in this decision. From a governance perspective, it creates a massive conflict of interest because obviously there is an interest in having a new treatment available for patients. I think it's possible to square that circle. There are lots of different techniques, such as citizen juries, other methods that can be used where you can take a variety of views in to help you make a decision. And I know some HTA bodies are looking at that. And maybe where we'll go to in the future is less of a voting approach to this and more of a multiple input to help make the final decision. And so there's lots of different methods being looked at at how you can do that. I don't think I answered your question, but I think that's deliberate because I don't think there is a solvable answer to that conundrum. Yes, I think that's more or less the answer you could give. It's a difficult one, but we sometimes get comforted with this. But thank you for answering. No problem. OK, we'll just go for one last question with Mirjana and before closing the webinar. So I'll just unmute your mic, Mirjana. Hello, Kate. Hello, everyone. Thank you for this webinar. I would like to ask a question about patients' organisations in countries like Macedonia, where HTA is not available at all. So how do you think that lobbying from patient organisations should be to the healthcare institutions where we have a very low budget for access to drugs? I mean, we are trying as a patient organisation to be in media constantly sending letters and all that we can do from our side, but still we are living in a low GDP country with very low budget for purchasing the drugs. How do you think we can handle this situation? So I think there's two sides to answering this question. So I think on one side you have what can you do to encourage patient involvement in the decisions that are made? You can't waive a magic wand and suddenly there's more money in the country than there is. So that's not in your power. But what is in your power is to work probably across disease areas. So not just thinking about this from a myeloma perspective, but across disease areas on having a system where at least how that small pot of money is allocated, that decision-making process involves patients and patient groups. So that's one element. The other thing is it's not just putting pressure on the system and the politicians. It's also thinking about what can the industry do? So there are some companies who are now starting to really think about do they have different prices in different countries depending on the GDP of that country. And there's been some experiments at that, especially in vaccines, looking at how you can do that. There's challenges with that. But it's not just pointing the fingers at the government and saying we want more access to treatments. It's about saying how can we improve the system so that at least we have a voice? It might not always be a yes, but at least we have a voice in how those decisions are made. And it's also thinking about what can other stakeholders in the healthcare system, including the industry, do to help access to medicines in countries such as yours in Macedonia? But that's just my point of view. I mean, I don't have the perfect answer for this one, I'm afraid. Neil, could I ask a follow-up question to that? What do you think organisations like Miloma Patients Europe can be doing to support national patient organisations such as Mirjana's group in Macedonia to get more access to the political environment and to help them get involved in decision-making on new medicines? Yes, I think there's several things really. One is across your network, there will be different parts of your network creating evidence around the unmet needs, the challengers' experience of care, and it's making that as generalised and usable across the countries as possible, or creating that in a way that can be interrogated by different countries who are wanting to find supportive evidence for their asks. I think in terms of the process for reimbursement, whether you've got a HTA process or not, I think it's going across your network and finding examples of where that change has happened. So a good example for you is a HTA example, but until actually just a few years ago, France didn't have a patient involvement process at all. But by networking, France got in touch with other countries that did have this process and they started doing it. And we've seen that happen in small countries as well, so don't just think about this in terms of big countries. In South East Asia, we have lots of examples where small countries are actually starting to come up with quite innovative patient involvement processes. They're not expensive, they're not as complicated as what we have here in Europe, but there are certain examples that we can put people in touch with. So it's finding those examples, whether it happens at HTA or whether it happens with the Ministry of Health as part of a reimbursement committee. But I think the first step is always to have the confidence to approach the policy makers directly, to just start building that relationship. So maybe there's something, some experiences, some case studies, some things that you can pull together as MP Europe to really get that learning, those top tips, maybe a buddying system. So the people who are new to this or are not that confident working in policy can be buddied with somebody in a different country who's got more experience in this. But you maybe do that already, I don't know. That was really helpful, that's it. We do do that through our access programme, to an extent, but it's good to get some additional feedback on how we target that programme to support our members in countries that need our help. And I've just realised that we're nearly 10 minutes over time. So I think what I'll do is I'll close the webinar now. So I'd like to thank you again, Neil, for joining today and for your really helpful presentation. I think all of us found it really useful and very comprehensive. The webinar will be posted, as I mentioned previously, on our website. We've recorded it, so you'll be able to re-watch it and disseminate it through your networks. And if you have any follow-up questions, we're happy to help answer them. So please just email us at info at mpeurope.org. And the last thing is to just thank everyone for joining. I know it's a bit of a difficult time and we really appreciate your support and your participation. So thank you very much, everybody. Thank you for inviting me. It's been a pleasure. Wonderful. Thank you very much. Have a good evening, everybody.