1 00:00:00,690 --> 00:00:06,480 So without further ado, I will introduce our evening lecturer is Neil Ferguson. 2 00:00:06,480 --> 00:00:11,370 He currently works at the international level with a number of health technology assessment 3 00:00:11,370 --> 00:00:17,430 agencies really with a remit to try and align process and mechanism across those agencies. 4 00:00:17,430 --> 00:00:24,240 He is the chair of the HDI International Patient and Citizen Involvement Group, 5 00:00:24,240 --> 00:00:33,000 so he has a strong working relationship with patients and involving patients in health technology assessment processes. 6 00:00:33,000 --> 00:00:36,990 And he's also on the board member of patient focussed medicines development. 7 00:00:36,990 --> 00:00:45,270 And that's really trying to better promote how evidence can be generated for all different stakeholders views in a decision making process. 8 00:00:45,270 --> 00:00:49,560 So without further ado, I will hand you over to Neil. Thank you very much. 9 00:00:49,560 --> 00:00:54,840 Thank you, Stuart, and thank you for having me here and thank you for being here. 10 00:00:54,840 --> 00:00:58,900 So what I really want to take you through today is, you know, I've seen that, 11 00:00:58,900 --> 00:01:04,530 you know, the kinds of information those of you that run on on the MSE course, 12 00:01:04,530 --> 00:01:09,360 the kinds of information you've been looking at and you've been going into sort of health economics, 13 00:01:09,360 --> 00:01:20,670 really understanding some of the mechanisms behind what I want to do tonight is took it a bit of a higher level about how bodies work with each other, 14 00:01:20,670 --> 00:01:27,810 how they learn from each other and how sometimes they can't learn from each other and they can't 15 00:01:27,810 --> 00:01:34,410 work with each other because I think that's really important to understand where the barriers are. 16 00:01:34,410 --> 00:01:41,790 And in the second half, one of the areas where we're seeing a lot of alignment in HK is around patient involvement in the process. 17 00:01:41,790 --> 00:01:48,360 I want to take a deep dive into patient involvement in how to see the perspective I'm bringing. 18 00:01:48,360 --> 00:01:50,670 This just builds on what Stuart has said. 19 00:01:50,670 --> 00:01:58,740 But really, I work with chapatis and regulators, with patient groups, with industry, with academic researchers, 20 00:01:58,740 --> 00:02:04,050 all with the aim of getting people to play nicely together for a change and learn from one 21 00:02:04,050 --> 00:02:09,900 another and really understanding the different perspectives and the different limits. 22 00:02:09,900 --> 00:02:22,650 Different stakeholders have so often what I hear from the industry or from patient groups is they often say to me, why can't he change? 23 00:02:22,650 --> 00:02:29,410 And he often finds it really difficult to change because of the rules behind which they're being set up. 24 00:02:29,410 --> 00:02:35,250 There's a lot of reasons why it's quite hard to change processes like to, 25 00:02:35,250 --> 00:02:45,180 and we have to understand what those limits are and why it's hard to change HCA before we can convince them to at least try to change sometimes. 26 00:02:45,180 --> 00:02:54,720 So I work with HCA International. So for those of you that don't know, that's the International Scientific Society for Health Technology Assessment, 27 00:02:54,720 --> 00:03:02,550 Stewart and I, that's how we got to know each other on Paradigm, which was an international collaboration and in particular, 28 00:03:02,550 --> 00:03:10,710 the area that I was looking at was how patients can be involved in early dialogues with bodies and those 29 00:03:10,710 --> 00:03:18,690 early dialogues that first time that a company that's developing a medicine can go to a party and say, 30 00:03:18,690 --> 00:03:23,460 Can you give me a bit of advice here? What kind of evidence would you be looking for? 31 00:03:23,460 --> 00:03:30,900 And we worked with about 15 bodies to develop that piece of work and with patient focussed medicines development, 32 00:03:30,900 --> 00:03:39,240 I just wanted to flag up a new and emerging discipline of evidence, which is called patient experience data. 33 00:03:39,240 --> 00:03:46,890 And it's new is coming along. And one of the things we're trying to do a patient focussed medicines development is say, 34 00:03:46,890 --> 00:03:56,310 let's not make this one of these situations where the regulators have one set of evidence that they look at and bodies have another set of evidence. 35 00:03:56,310 --> 00:04:00,390 So from the very start, we're trying to pull everybody together to say, 36 00:04:00,390 --> 00:04:10,110 let's make sure this new class of evidence is usable, both for regulatory and to purposes. 37 00:04:10,110 --> 00:04:14,100 So what I'm going to take you through today, I'm going to talk a little bit about the context, 38 00:04:14,100 --> 00:04:23,490 the context within which to sets and what is the impact of that context for international alignment. 39 00:04:23,490 --> 00:04:29,910 Then we'll take a deeper dive into patient involvement, and I'll go through a couple of case studies. 40 00:04:29,910 --> 00:04:37,050 So when we think about the context, you've probably seen this, but this is the 2020 definition of. 41 00:04:37,050 --> 00:04:44,910 And I want to just flag there's two elements that you can see in bold that this is a multi-disciplinary process. 42 00:04:44,910 --> 00:04:51,180 So not no. One stakeholder is able to do on their own. 43 00:04:51,180 --> 00:04:59,250 They need the other stakeholders to come together in order to make that process and informs decision making. 44 00:04:59,250 --> 00:05:05,720 And I've. Really want to focus on that inform, because there is some myths that people have about. 45 00:05:05,720 --> 00:05:14,750 I'm sure you don't have them because you've been studying this, but the Mid-South bodies decide which medicines or similar technologies we have 46 00:05:14,750 --> 00:05:22,280 access to or that determining access to medicine becomes purely evidence based. 47 00:05:22,280 --> 00:05:32,180 And I know that's how a lot of people think about it. But in fact, if the politicians who ultimately decide, OK, this is from a report, 48 00:05:32,180 --> 00:05:41,330 it's done by the World Health Organisation and the European Union, and they look at this interface between state and health policy. 49 00:05:41,330 --> 00:05:45,710 And they say H.J. provides evidence based input. 50 00:05:45,710 --> 00:05:52,400 So again, this input informing it promotes evidence informed policymaking. 51 00:05:52,400 --> 00:06:01,400 So it just promotes it. He doesn't make it happen, and they say it offers a bridge between the research and the policy community. 52 00:06:01,400 --> 00:06:06,920 So you can already see that state lives in this very unsafe world. 53 00:06:06,920 --> 00:06:13,040 On one side, you've got all these people creating evidence and on the other side, you've got all the policy makers. 54 00:06:13,040 --> 00:06:19,220 And it's to that sort of sits between those two and tries to make sense of it. 55 00:06:19,220 --> 00:06:27,140 So policymakers decide within each country the overall resources available to health care. 56 00:06:27,140 --> 00:06:32,180 They decide what is more spent on schools and less on health care or vice versa, 57 00:06:32,180 --> 00:06:37,970 for example, within health care, they will decide where the priorities should be. 58 00:06:37,970 --> 00:06:45,770 So in one country, they might say we have a real problem with long term conditions like diabetes and heart disease, 59 00:06:45,770 --> 00:06:51,110 so we want to put more money into treating diabetes and heart disease. 60 00:06:51,110 --> 00:06:57,890 Another country could say, we think that's OK, but we think we have a real problem with rare diseases. 61 00:06:57,890 --> 00:07:07,790 So we want to put more money into understanding and treating rare diseases that policy decisions, they're not decisions. 62 00:07:07,790 --> 00:07:15,560 Importantly, it's also the policymakers and the governments that decide how much resources a body has, 63 00:07:15,560 --> 00:07:20,570 how many people can work in that body, how much money do they have? 64 00:07:20,570 --> 00:07:26,540 And that has a massive impact on the methods that that body can use. 65 00:07:26,540 --> 00:07:32,630 They can't do anything too complicated if they only have a few people in order to do all those things. 66 00:07:32,630 --> 00:07:39,860 And finally, it's the policymakers that decides what kind of happens in their country. 67 00:07:39,860 --> 00:07:44,780 So what's the focus of that is only going to look at the clinical evidence? 68 00:07:44,780 --> 00:07:49,460 Or is it also going to look at the health economic evidence? Is it going to look societal evidence? 69 00:07:49,460 --> 00:07:55,490 Some of them do that to all of those are political decisions that are told to the FDA. 70 00:07:55,490 --> 00:08:01,400 This is how we want you to operate. And so I think it's really important to keep that in mind. 71 00:08:01,400 --> 00:08:07,970 And I want to show you one example to show how much politics determines access to medicines. 72 00:08:07,970 --> 00:08:19,850 So it's a U.K. example I've picked because we're here. But in the early 2010s, there was a real problem in the U.K. of accessing cancer medicines. 73 00:08:19,850 --> 00:08:26,900 So new cancer medicines would come out. They would be assessed by the Health Technology Agency called Nice. 74 00:08:26,900 --> 00:08:36,860 Here in the U.K., a nice would say usually we will not recommend this for reimbursement within the National Health Service. 75 00:08:36,860 --> 00:08:42,050 And yet people in France had access to the medicine, and people in Germany had access to the medicine. 76 00:08:42,050 --> 00:08:46,400 So this suddenly became a lot of political pressure. 77 00:08:46,400 --> 00:08:55,070 Why do we in the UK not have access to the same cancer medicines as people in other countries? 78 00:08:55,070 --> 00:08:59,930 And there were three strategies the government could have tried here. 79 00:08:59,930 --> 00:09:06,380 The first thing they could have tried is to say, we're going to do nothing because we believe in our process. 80 00:09:06,380 --> 00:09:14,060 And if the medicines don't bring value, then we shouldn't pay for them, so we should carry on with the system as it stands. 81 00:09:14,060 --> 00:09:22,400 The second thing they could do is to say, we think this shows as a problem with how to is conducted in the UK. 82 00:09:22,400 --> 00:09:31,190 So we want to change the methods of stay in the UK so that we get more access to cancer medicines. 83 00:09:31,190 --> 00:09:32,660 So they could have said the third thing, 84 00:09:32,660 --> 00:09:41,570 which is we will just throw money at this problem and we will sidestep the process using separate pots of money. 85 00:09:41,570 --> 00:09:47,660 And of course, they went for the Option C, so they created something called the Cancer Drugs Fund. 86 00:09:47,660 --> 00:09:57,680 And you can see this is a quote from The Lancet at that time, saying this is a triumph of political expediency over rationality. 87 00:09:57,680 --> 00:10:05,230 So instead of solving the. The original problem, they just threw a lot of money at the problem to hope it went, it goes away. 88 00:10:05,230 --> 00:10:09,310 But it didn't go away. So this is just a few years later. 89 00:10:09,310 --> 00:10:17,830 You know, the government at the time having to inject another 400 million pounds into that fund just to keep it going. 90 00:10:17,830 --> 00:10:26,710 And you can see from the headline that this is seen as a party. Political movement is not seen as a movement. 91 00:10:26,710 --> 00:10:35,950 So you can see how politics can determine the access to medicines regardless of what a body says. 92 00:10:35,950 --> 00:10:46,480 And this creates a challenge when we're thinking about aligning systems, because if systems are not purely they're evidence based systems, 93 00:10:46,480 --> 00:10:54,310 but if they sit within political context, then it becomes much harder for alignment to happen. 94 00:10:54,310 --> 00:10:59,470 So let's talk about that. When I think of alignment, I think of it in two ways. 95 00:10:59,470 --> 00:11:07,090 On the left hand side, I call informal alignment and on the right hand side, the more formal kinds of alignment. 96 00:11:07,090 --> 00:11:15,730 So on the left hand side, you share best practises. A body might speak to another body and say, We tried this, it was really good. 97 00:11:15,730 --> 00:11:18,100 You should try it too. 98 00:11:18,100 --> 00:11:25,930 They partner on projects together doesn't mean they're going to change their system, but at least they partner and projects together. 99 00:11:25,930 --> 00:11:30,370 And in terms of discussions with regulators. Yet they can have discussions with regulators. 100 00:11:30,370 --> 00:11:36,970 They can hear what each side is doing. Does it mean that they have to change anything on this side, though? 101 00:11:36,970 --> 00:11:42,910 On the right hand side, you have more formal networks that have been created. 102 00:11:42,910 --> 00:11:51,310 A lot of these actually in Europe, because that's where a lot of the challenges first happened, where we saw the divergence of parties. 103 00:11:51,310 --> 00:12:00,490 So you had formal networks created like you netter really charged with coming up with a common model for how you do, 104 00:12:00,490 --> 00:12:06,220 which would hopefully then filter out to all the countries. So alignment would happen. 105 00:12:06,220 --> 00:12:11,890 That didn't work at all. And so they said, OK, let's come up with some legislation. 106 00:12:11,890 --> 00:12:18,910 Let's make laws at the EU level that force people to align the way that they do. 107 00:12:18,910 --> 00:12:23,500 And I'll talk about that later, and you can see that that's not really going to happen either. 108 00:12:23,500 --> 00:12:29,530 And then what is successful is what happens in a country so often. 109 00:12:29,530 --> 00:12:38,410 The really good alignment you see is at the national level between different parts of the health care system, including how to. 110 00:12:38,410 --> 00:12:44,350 So it tends to work very well with other parts of the health care system locally. 111 00:12:44,350 --> 00:12:51,850 They just find it really hard to make decisions that change the way they work at the national level. 112 00:12:51,850 --> 00:12:56,050 So when we look at this informal side, you know, different ways that this can happen, 113 00:12:56,050 --> 00:13:02,470 it can happen through organisations like, I think to a lot of best practise sharing. 114 00:13:02,470 --> 00:13:10,090 It can happen through consortium that have multiple parties involved working on a project together, 115 00:13:10,090 --> 00:13:15,160 and it can happen through just discussions that naturally happen between different bodies, 116 00:13:15,160 --> 00:13:23,680 you know, and just as an example, I pull together some things from I, you know, they run a global policy forum twice a year. 117 00:13:23,680 --> 00:13:30,010 They invite bodies from around the world to that forum, as well as people from the industry and others. 118 00:13:30,010 --> 00:13:35,080 And they look at these bigger issues that they don't quite know how to solve yet. 119 00:13:35,080 --> 00:13:39,400 How are we really going to deal with managed entry agreements in the future? 120 00:13:39,400 --> 00:13:47,260 How will they evolve? How do we get better at disinvesting from old technology or inefficient technology? 121 00:13:47,260 --> 00:13:51,790 And how do we deal with manage value based decision making? 122 00:13:51,790 --> 00:14:01,210 What does good look like there? They also have a lot of specialist groups that look at one particular area of HCA. 123 00:14:01,210 --> 00:14:09,970 So there's a group looking just at real world evidence trying to understand how real world evidence is used by HCA bodies, 124 00:14:09,970 --> 00:14:11,590 and they're thinking about the future as well. 125 00:14:11,590 --> 00:14:20,710 How this artificial intelligence and machine learning lead to better real world evidence that creates better information for. 126 00:14:20,710 --> 00:14:23,590 And of course, they have an annual meeting, an annual conference, 127 00:14:23,590 --> 00:14:32,170 and that is probably the place where most alignment happens four days in one place, everyone together. 128 00:14:32,170 --> 00:14:39,910 That's where the magic happens. But the right hand side of that circle, I'm sorry to say, is really, really hard. 129 00:14:39,910 --> 00:14:47,170 You know, this idea that this can change the way they work just by sharing ideas. 130 00:14:47,170 --> 00:14:48,080 It doesn't really work. 131 00:14:48,080 --> 00:14:59,360 So as I've said before, UNITA was charged with creating a sort of common model for how to that would then sort of be taken up by the various. 132 00:14:59,360 --> 00:15:07,250 National bodies. And actually, it always hits up against this wall of in my country, 133 00:15:07,250 --> 00:15:12,800 I think we're doing better than that model, so we want to stick with what we've got. 134 00:15:12,800 --> 00:15:17,090 You know, so it's not invented here syndrome, you know, 135 00:15:17,090 --> 00:15:25,140 we've developed the best that that reflects our societal values and we don't want to change that. 136 00:15:25,140 --> 00:15:33,230 I'll talk about the second one in a moment that the EU legislation, but I believe it's my opinion that it's been watered down too much. 137 00:15:33,230 --> 00:15:40,340 I honestly don't see how it's going to work, but and then we see positive things happening at the national level. 138 00:15:40,340 --> 00:15:51,170 But let's talk about that EU thing. The challenge is still that health care budgets are set and managed by national governments. 139 00:15:51,170 --> 00:15:55,490 They're not managed collectively across multiple nations. 140 00:15:55,490 --> 00:16:06,320 And so the danger is, or the fear is that if you have an international process that could force governments to spend money that 141 00:16:06,320 --> 00:16:12,470 they were not planning to spend or to make different investment decisions than they were planning to make. 142 00:16:12,470 --> 00:16:20,570 And so there was a lot of pushback about how much of a limit would this a system have. 143 00:16:20,570 --> 00:16:26,540 And the second thing is the thing I was alluding to a few minutes ago that they are really focussed on. 144 00:16:26,540 --> 00:16:34,040 We have the best model for how to see if you're going to create a European wide to which model do you choose, 145 00:16:34,040 --> 00:16:38,390 whichever one you choose, a lot of people are going to be unhappy, you know? 146 00:16:38,390 --> 00:16:50,120 So what they did was they said, OK, we will take the least controversial part of a and only make that Europe wide. 147 00:16:50,120 --> 00:17:00,080 So the very first, if you like the main analysis of who is looking at the clinical effectiveness relative to what's available today, 148 00:17:00,080 --> 00:17:07,040 so relative clinical effectiveness, that assessment is due to happen jointly. 149 00:17:07,040 --> 00:17:16,310 So it can happen in one place and everyone will accept that report and say, OK, at least we don't have to do that at the national level. 150 00:17:16,310 --> 00:17:23,300 But the economic and pricing in all of those other things that lead to a yes or no that stays at the national level. 151 00:17:23,300 --> 00:17:29,150 So it still means you're going to have very divergent decisions from country to country. 152 00:17:29,150 --> 00:17:36,950 And actually, even the first thing I said is not true because there was a lot of pushback about certain countries saying, 153 00:17:36,950 --> 00:17:40,820 but we think our system is better than what you're planning to do. 154 00:17:40,820 --> 00:17:45,050 So we don't want to take this Europe wide reports. 155 00:17:45,050 --> 00:17:48,410 We don't want to use it. We want to do our own. So they've made it. 156 00:17:48,410 --> 00:17:55,130 So it's not mandatory for countries to use this, this new EU wide report. 157 00:17:55,130 --> 00:18:00,500 And so we've left with a system that's even more fragmented and nobody knows who's 158 00:18:00,500 --> 00:18:07,370 going to take those reports up or not when it actually starts going forward. 159 00:18:07,370 --> 00:18:11,180 I sent them my last slide for this section is really, you know, 160 00:18:11,180 --> 00:18:18,500 I think the fragmentation is part of the nature of having national health care budgets. 161 00:18:18,500 --> 00:18:27,740 I find it difficult to see a future where you're going to have common decisions made that impact the way that governments spend money nationally, 162 00:18:27,740 --> 00:18:34,820 you know, supranational level and you're always going to have for societal reasons and economic reasons. 163 00:18:34,820 --> 00:18:41,360 Different focuses to your to you're going to have to that look. 164 00:18:41,360 --> 00:18:42,620 Some of them will say, actually, 165 00:18:42,620 --> 00:18:49,700 we just all we care about is the clinical outcomes we will pay for the clinical outcomes and we're not paying for anything more. 166 00:18:49,700 --> 00:18:56,300 And you'll have others that say, Yeah, but we think the patient impacts also need to be part of the value. 167 00:18:56,300 --> 00:18:58,580 So we want to look at that as well. 168 00:18:58,580 --> 00:19:05,510 And then you'll have others that say, we believe those two things, but we think we should also be thinking about the societal impacts. 169 00:19:05,510 --> 00:19:13,310 You know, in some Scandinavian so sweet and in some of in some of the results, they go that far along there. 170 00:19:13,310 --> 00:19:17,700 And you're still going to have arguments about what evidence they accept. 171 00:19:17,700 --> 00:19:25,010 You know, some will say, we don't agree with your model. We don't agree with the the surrogate end you've used. 172 00:19:25,010 --> 00:19:29,630 We think you should have looked for actual hard outcomes, 173 00:19:29,630 --> 00:19:36,770 and some will love patient relevant evidence like patient reported outcome measures and some hates it. 174 00:19:36,770 --> 00:19:41,300 And I think we're still in this fragmented world. 175 00:19:41,300 --> 00:19:46,370 But for those of you that are interested in, that's what makes it so interesting. 176 00:19:46,370 --> 00:19:51,470 I mean, if everybody did exactly the same, there would be nothing to learn. 177 00:19:51,470 --> 00:19:59,390 And there would be nothing to compare. So I love the fact that this is still quite difficult to navigate because. 178 00:19:59,390 --> 00:20:07,700 Each of those difficulties, each of those hurdles is an opportunity to learn what works best and what doesn't. 179 00:20:07,700 --> 00:20:11,870 Moving on to part two patient involvement, it's related, actually, 180 00:20:11,870 --> 00:20:21,260 because I see patient involvement and a really good example of where the agencies have aligned and learn from each other and move this forward. 181 00:20:21,260 --> 00:20:29,570 OK, so this idea that alignment doesn't happen. Patient involvement, I think, is an exception to that rule. 182 00:20:29,570 --> 00:20:36,080 So 1970s, would you believe that was really invented by America in the 1970s? 183 00:20:36,080 --> 00:20:43,220 It was mainly. The Office of Technology Assessment was a federal office founded in the early seventies. 184 00:20:43,220 --> 00:20:53,750 I think it ran until about 1992, and in 76 they produced some guidance of how to do health technology assessment. 185 00:20:53,750 --> 00:21:02,270 And they said it's impossible to do it without thinking about patients, families and society as well as everything else. 186 00:21:02,270 --> 00:21:13,820 OK? And yet when the port is in Europe and Canada and Australia and elsewhere started to form in the 90s and 2000s, 187 00:21:13,820 --> 00:21:18,620 they just forgot that advice and they didn't bother involving patients. 188 00:21:18,620 --> 00:21:29,750 And yet, by the time you get to 2017, this man here, Brian O'Rourke, he was the head of Catalyst, which is the Canadian health technology assessor. 189 00:21:29,750 --> 00:21:36,410 And he said quite clearly, if you're not involving patients, you're not doing. 190 00:21:36,410 --> 00:21:45,320 So there was a massive change that happened between the 2000s and by the time we get to 2017, 191 00:21:45,320 --> 00:21:52,640 OK, and today what we see is we see patients being involved in multiple parts of the process. 192 00:21:52,640 --> 00:22:00,890 Yes, they input into the assessment process, but they also get active in the early dialogue's years before an assessment, 193 00:22:00,890 --> 00:22:07,640 their input and increasingly into managed entry agreements, especially to make sure that they're manageable for patients. 194 00:22:07,640 --> 00:22:11,840 The first managed entry agreements included a lot of monitoring trips and extra 195 00:22:11,840 --> 00:22:17,060 visits for patients in order to prove that their medicine was still working. 196 00:22:17,060 --> 00:22:25,640 And if they missed some of those visits, they were taken off the treatment. You know, so, so so patients need to be involved in these. 197 00:22:25,640 --> 00:22:36,860 They're increasingly providing their own data into HCA processes, helping HCA bodies understand what it's like to live with this disease. 198 00:22:36,860 --> 00:22:40,100 They advise on the impact of the healthcare system. 199 00:22:40,100 --> 00:22:51,380 The drug companies often say to bodies on medicine is so fantastic that it's going to change the way that this this disease is treated. 200 00:22:51,380 --> 00:22:53,810 Patients will not be treated in the old way. 201 00:22:53,810 --> 00:23:00,980 Once we have this treatment, and that's great because you need to check if the health care system needs to change, 202 00:23:00,980 --> 00:23:04,880 what's the impact on that change on patients? 203 00:23:04,880 --> 00:23:12,620 And finally, when you have a team that's covering a disease area that they've not looked at for a long time or they've never looked at before, 204 00:23:12,620 --> 00:23:18,410 they often do scoping meetings to understand from a patient what it's like to live 205 00:23:18,410 --> 00:23:24,810 with that disease so they can think about that before they get into the HCA thesis. 206 00:23:24,810 --> 00:23:27,200 This changes all the time, this map, by the way, 207 00:23:27,200 --> 00:23:34,340 so I wouldn't focus too much on how this all details out because next year it will be different again. 208 00:23:34,340 --> 00:23:39,710 But you have a lot of countries have something called patient group submissions, 209 00:23:39,710 --> 00:23:47,840 so that's where they invite patient groups, not individual patient patient groups, to submit evidence into a HCA. 210 00:23:47,840 --> 00:23:53,480 Others have consultations, so public consultations where the whole public they could be nurses, 211 00:23:53,480 --> 00:23:58,850 doctors, patients, family members, whoever they can input into a process. 212 00:23:58,850 --> 00:24:09,470 Usually it's like a website where you can you can sort of look at your your comments and we so we are seeing Germany is a bit of an outlier, 213 00:24:09,470 --> 00:24:14,150 but a really interesting patient involvement process. They do it on two levels. 214 00:24:14,150 --> 00:24:19,850 They say our assessment will only consider evidence that we think is patient relevant. 215 00:24:19,850 --> 00:24:27,440 So there's always this argument with drug companies and the GBA in Germany about what is patient relevant, you know, 216 00:24:27,440 --> 00:24:32,870 but they also have a really interesting mechanism for involving patients where 217 00:24:32,870 --> 00:24:38,510 they they really basically give money to full patient associations and say, 218 00:24:38,510 --> 00:24:43,310 with this money, you make sure we have patients in our process. 219 00:24:43,310 --> 00:24:52,040 And it's a way of ensuring that the patients in the process have not received any funding from the industry. 220 00:24:52,040 --> 00:24:57,530 OK, so they do it. So why do parties involve patients? 221 00:24:57,530 --> 00:25:03,420 Well, as I've said before, if? First, they said, we don't want to do this, we don't want to do this. 222 00:25:03,420 --> 00:25:07,230 So they said, OK, you know what? This is dead easy. We're clever people. 223 00:25:07,230 --> 00:25:12,780 OK, so the industry provides us with the evidence and we can look at the evidence. 224 00:25:12,780 --> 00:25:19,590 We can start reviewing it. We can bring in clinicians to the table and we can bring in health economists to the table. 225 00:25:19,590 --> 00:25:27,150 And together we can debate the evidence and then we can make a decision, you know, should we reimburse you recommend that this is reimbursed. 226 00:25:27,150 --> 00:25:31,740 And if so, to whom? And how do we reimburse it? 227 00:25:31,740 --> 00:25:42,330 OK, dead easy. Except it's not because the clinical evidence never tells the whole story, especially when it's a new product. 228 00:25:42,330 --> 00:25:50,910 The clinical studies only provide part of the story they need to be extrapolated to match the health care population in that country. 229 00:25:50,910 --> 00:25:58,710 So the clinical trial population is usually very tightly defined. And so there's this big question how would the results from this clinical trial 230 00:25:58,710 --> 00:26:05,250 really going to be reflected in real people on the ground in our country? 231 00:26:05,250 --> 00:26:10,020 The second thing is that clinical trials, because the regulators are getting faster and faster, 232 00:26:10,020 --> 00:26:16,030 approving medicines, clinical trials and shorter and shorter in length, you can almost see it on a graph. 233 00:26:16,030 --> 00:26:23,820 Yeah, and it means that there's virtually no evidence about the duration of response from a medicine at launch. 234 00:26:23,820 --> 00:26:31,740 So we know it might be expensive and we know it works at first or within the first 24 or 48 weeks, but we don't know much more than that. 235 00:26:31,740 --> 00:26:38,340 So if you cannot pay this much for it, can we at least start to think about whether it's going to work in the longer term? 236 00:26:38,340 --> 00:26:42,390 How many years is it going to work for that sort of thing? 237 00:26:42,390 --> 00:26:49,470 Quality of life and patient reported outcome measures are really hard to interpret, especially for a tag. 238 00:26:49,470 --> 00:26:53,310 There's often really big challenges, with missing data in patient. 239 00:26:53,310 --> 00:27:00,390 Reported outcomes is often not talked about, but there's always a lot of missing data in patient reported outcomes and how that's 240 00:27:00,390 --> 00:27:07,020 dealt with statistically can completely change the shape of that evidence point. 241 00:27:07,020 --> 00:27:15,060 There's also been a move over the last five 10 years to really build new patient reported outcome tools that are specific to 242 00:27:15,060 --> 00:27:23,280 each particular disease for good reason because you want to know as much detail about what it's like to live with that disease. 243 00:27:23,280 --> 00:27:29,160 But when you have so many different patient reports that controls, how can a body keep on top of them all? 244 00:27:29,160 --> 00:27:36,990 How can it compare the results of one tool that was used by one company compared to the results of a different tool used by a different company? 245 00:27:36,990 --> 00:27:42,360 So it becomes really, really complicated. And then there's this old chestnut here. 246 00:27:42,360 --> 00:27:50,010 This is this is the one that always brings down the is is what did you use as your comparator in your clinical studies? 247 00:27:50,010 --> 00:27:58,860 You know, and so you often have really credible questions to ask, why did you pick that comparison? 248 00:27:58,860 --> 00:28:05,010 We don't use that. We don't use that drug in our country. So actually, your data means nothing to us. 249 00:28:05,010 --> 00:28:14,340 We can't really understand what is telling us. And as I mentioned before, when you have a lot of innovation and in particular disease area, 250 00:28:14,340 --> 00:28:18,540 the clinical trials never reflect the most recent innovation. 251 00:28:18,540 --> 00:28:24,630 So if you let me give you an example, you've got a new treatment that comes out for skin cancer. 252 00:28:24,630 --> 00:28:30,810 But just two years ago, there was a great new treatment that came up for skin cancer that changed the landscape of skin cancer. 253 00:28:30,810 --> 00:28:35,550 But your clinical trial never measured that because that treatment wasn't there then. 254 00:28:35,550 --> 00:28:45,270 You know, so as a stay, that creates a massive problem. Now, how does this latest innovation compare to a recent innovation? 255 00:28:45,270 --> 00:28:49,260 And we have to have the answer. So we have all these uncertainties. 256 00:28:49,260 --> 00:28:55,770 And so the approach breaks down, and you start to ask in the committee meeting these kinds of questions. 257 00:28:55,770 --> 00:29:02,460 You know, I can see your data, but I'm really starting to question what is the most meaningful part of your data. 258 00:29:02,460 --> 00:29:07,650 I should be focussing on what outcomes are important. 259 00:29:07,650 --> 00:29:12,180 You mentioned all of these different populations that we could potentially be reimbursing, 260 00:29:12,180 --> 00:29:15,930 but do we understand the particular needs of those populations? 261 00:29:15,930 --> 00:29:20,760 Is there a particular patient population that is really underserved as much higher needs? 262 00:29:20,760 --> 00:29:25,860 For example, in many diseases, skin diseases are a good example. 263 00:29:25,860 --> 00:29:30,900 So dermatology, skin diseases, you often have disease scores, you know, 264 00:29:30,900 --> 00:29:36,660 numbers attached to have severe there's this disease is and it's sometimes really hard to 265 00:29:36,660 --> 00:29:42,390 link what those disease scores mean and improvement of one point in that disease score. 266 00:29:42,390 --> 00:29:46,440 What does that really mean for a patient? 267 00:29:46,440 --> 00:29:54,360 If you're saying you're going to change the way that this whole disease is treated, have we thought about how that impacts the people who are treated? 268 00:29:54,360 --> 00:29:59,180 Ultimately, we're trying to answer this question quite clearly. Who will? 269 00:29:59,180 --> 00:30:06,090 Benefit most, and let's focus on them and obviously, what is the real value of the treatment? 270 00:30:06,090 --> 00:30:11,060 So in the round, based on what we know, what do we think the value is? 271 00:30:11,060 --> 00:30:16,100 And I argue that patients are the missing link in these deliberations. 272 00:30:16,100 --> 00:30:23,660 They really could, explaining how a disease affects day-to-day life, what they can do, what they can't do. 273 00:30:23,660 --> 00:30:33,140 They can translate the endpoints being measured. The patient reported outcomes as well into specific impacts that they experience every day. 274 00:30:33,140 --> 00:30:37,700 They can highlight if there's any change in these scores. What does that mean? 275 00:30:37,700 --> 00:30:44,900 A two point scale? Oh my god, that's transformative. I could do so much more if I improve by two points. 276 00:30:44,900 --> 00:30:53,030 And they can clearly articulate how current ways of managing their disease is not good for them is not meeting their needs. 277 00:30:53,030 --> 00:31:01,340 And you can see here I've highlighted specific twice here, and it's probably the words if I'm if you were a patient patient group, 278 00:31:01,340 --> 00:31:08,990 people here, I've probably set up at least 50 times already now because I cannot emphasise enough. 279 00:31:08,990 --> 00:31:17,510 The emotion does not help. So if a patient group comes into a stay and says, Oh my God, 280 00:31:17,510 --> 00:31:27,680 this disease is so difficult to live with my life is how so many other patient groups with different diseases come in and say those things. 281 00:31:27,680 --> 00:31:32,810 It doesn't help the body understand why is your life? 282 00:31:32,810 --> 00:31:37,250 How? Why is it so bad to live with this disease? 283 00:31:37,250 --> 00:31:44,510 So what I always say to patient groups is, be specific, say exactly because if this symptom, 284 00:31:44,510 --> 00:31:49,670 I can't do this every day and the second symptom stops me from doing this other thing. 285 00:31:49,670 --> 00:31:57,680 And then it's quite easy then for the body to say, I hope with this new treatment actually deals with those symptoms really well. 286 00:31:57,680 --> 00:32:04,820 So it helps them link impact to the, you know, the treatment in front of them. 287 00:32:04,820 --> 00:32:08,630 Oh my God, time's rushing on. OK, I'm going to be a bit quick enough. 288 00:32:08,630 --> 00:32:14,690 So they add patients into the mix and patients provide that context to them. 289 00:32:14,690 --> 00:32:24,650 We looked at this guy. We did a Big Delfi study internationally, multiple stakeholders, and we asked why should patients be involved in HK? 290 00:32:24,650 --> 00:32:29,780 And the list is here, but I really want to focus on this relevance on the first one. 291 00:32:29,780 --> 00:32:36,260 Patients have information and knowledge and experiences that no other stakeholder has. 292 00:32:36,260 --> 00:32:42,710 So the doctors cannot answer the same questions they can answer. The economist can't answer those questions, either. 293 00:32:42,710 --> 00:32:53,690 And it's understanding that missing piece of knowledge that improves the quality of what people used to think that if we invited patients, 294 00:32:53,690 --> 00:32:59,240 patients would just be demanding. We want access to everything. It turns out they don't. 295 00:32:59,240 --> 00:33:05,510 You know what they say is we want new drugs, but we don't want any drug and not any price. 296 00:33:05,510 --> 00:33:12,620 We want drugs that are transformative. We want drugs that are really going to change the way we experience disease. 297 00:33:12,620 --> 00:33:22,620 And they can be as critical, if not more critical of the industry when they think that the evidence that they've collected has not been good enough. 298 00:33:22,620 --> 00:33:30,800 You know, and I've heard lots of very difficult discussions between patient groups in the industry of why did you choose that as your comparator? 299 00:33:30,800 --> 00:33:38,660 Why did you when you run the trial for 24 weeks, why did you have this stupid exclusion criteria that doesn't match reality? 300 00:33:38,660 --> 00:33:44,960 You know, they will be just as critical of the evidence as the other stakeholders. 301 00:33:44,960 --> 00:33:52,190 How do they do it? I'm not going to go through this in detail. It's limited by all the things I mentioned earlier. 302 00:33:52,190 --> 00:33:57,380 What is the remit of that? What is their role in that health system? 303 00:33:57,380 --> 00:34:00,630 Have they got the resources to do patient engagement? 304 00:34:00,630 --> 00:34:06,320 There's no point asking them to do it if they don't have the resources to do it in their country. 305 00:34:06,320 --> 00:34:14,360 Is there even a cultural and societal acceptance of patients and patient groups doesn't exist in every country? 306 00:34:14,360 --> 00:34:21,800 So sometimes you can't just transport a concept into a new country setting and expect it to live. 307 00:34:21,800 --> 00:34:27,020 You have to be respectful of the culture and society. 308 00:34:27,020 --> 00:34:33,020 The way that we see it work is on this sort of spectrum for informal fruits, 309 00:34:33,020 --> 00:34:40,070 the most formal hair where you have people from patient groups actually in the meetings. 310 00:34:40,070 --> 00:34:46,190 And I just want to just flick through this one. But this is a sort of generic process. 311 00:34:46,190 --> 00:34:59,130 Not every day has all these steps and some have more steps than this, but generally scoping phase happens in preparation of an upcoming year only. 312 00:34:59,130 --> 00:35:03,690 Happens, really, if there were big questions about we don't understand this disease, 313 00:35:03,690 --> 00:35:08,460 we've not looked at this disease for 15 years and we need to understand if anything's 314 00:35:08,460 --> 00:35:14,760 changed or this is a rare disease and we've never looked at this ever in our body, 315 00:35:14,760 --> 00:35:24,240 then often then have consultation and workshops with patient groups, usually to help understand what are the main aspects of this disease. 316 00:35:24,240 --> 00:35:30,570 They should be thinking about when they're organising the time. You have the evidence submission phase. 317 00:35:30,570 --> 00:35:38,730 The industry submits their evidence, but this is also when a lot of parties reach out to patient groups and say, 318 00:35:38,730 --> 00:35:45,940 Can you also submit evidence which we will consider alongside the industry evidence? 319 00:35:45,940 --> 00:35:50,280 You have a committee meeting. Patient groups are usually there. 320 00:35:50,280 --> 00:36:00,060 Sometimes individual patients are there. It depends on the day, and it also depends on whether it's a rare disease or more common disease. 321 00:36:00,060 --> 00:36:02,370 There's a lot of distinctions there, 322 00:36:02,370 --> 00:36:13,530 but at a committee meeting the patient's role there are to give testimony and to answer any questions that come up from the other committee members. 323 00:36:13,530 --> 00:36:21,510 They're not official voting members of the committee, just as the industry is not official voting members of the committee. 324 00:36:21,510 --> 00:36:25,530 So just to be clear about this, you know, we have two governance terms. 325 00:36:25,530 --> 00:36:33,480 We have to separate the interest of needing a treatment and wanting a treatment, then voting for that treatment. 326 00:36:33,480 --> 00:36:42,480 So you often have citizens or patients that don't have that disease in the committee meeting who do have a vote, 327 00:36:42,480 --> 00:36:47,910 but the patients who will benefit from the decision will not have a vote. 328 00:36:47,910 --> 00:36:53,760 There's just good governance. Yep. Oh, sorry, I didn't finish that one. 329 00:36:53,760 --> 00:37:01,140 Discuss, discuss the evidence. Make a sort of draught recommendation. 330 00:37:01,140 --> 00:37:09,780 There's usually a consultation period. Those patient groups that have been involved up to now are part of that consultation, 331 00:37:09,780 --> 00:37:14,010 but usually that's where it stops is not reopened to new patient groups. 332 00:37:14,010 --> 00:37:19,080 So you have to be involved usually in order to be in that consultation step. 333 00:37:19,080 --> 00:37:23,940 And then there might be a second committee meeting in a final sort of recommendation made. 334 00:37:23,940 --> 00:37:31,020 And you can see here that patients are involved, potentially at every step along the way. 335 00:37:31,020 --> 00:37:37,920 Not every body does that, but this is a sort of idealistic general example. 336 00:37:37,920 --> 00:37:45,960 These are the questions, you know, I think you were asking me earlier sort of like, what is it that about this consultation with patients? 337 00:37:45,960 --> 00:37:52,500 So this is from Scotland. But these same questions are asked in France, the US, in Canada that, you know, 338 00:37:52,500 --> 00:38:01,380 most bodies that have this kind of approach where patients have a written submission are similar questions to this. 339 00:38:01,380 --> 00:38:09,270 They want to know about what it's like to live with the disease, how well is currently managed without the new treatment. 340 00:38:09,270 --> 00:38:12,750 They want to know if they've been able to talk to anybody who's tried the new treatment. 341 00:38:12,750 --> 00:38:18,870 Maybe it's already been launched in a neighbouring country or somebody who's been on a clinical trial. 342 00:38:18,870 --> 00:38:23,550 They want to know how it's expected to improve the quality of life or the medical 343 00:38:23,550 --> 00:38:29,910 care of a patient and what they think the impact would have on families and carers. 344 00:38:29,910 --> 00:38:35,730 And finally, a really important one they want to know are there any disadvantages to this new treatment? 345 00:38:35,730 --> 00:38:42,900 And they're very suspicious. If the patient groups don't answer that question because there's no treatment ever made, that is only positive. 346 00:38:42,900 --> 00:38:50,280 There will always be something to plug there. Case studies very briefly. 347 00:38:50,280 --> 00:38:54,180 Right? So this one is a right. It's reasonably recent. 348 00:38:54,180 --> 00:38:57,660 It's a rare disease treatment. Very, very expensive. 349 00:38:57,660 --> 00:39:07,680 And I wanted to use this case study to show how patients can bring new concepts to the table that none of the other stakeholders have mentioned. 350 00:39:07,680 --> 00:39:15,000 OK, so this is for lipodystrophy rare condition quite complicated. 351 00:39:15,000 --> 00:39:22,560 Different patients have different symptoms. It's poorly understood by the body because it's a rare disease. 352 00:39:22,560 --> 00:39:29,250 This is quite common for rare diseases and the industry and and the clinical 353 00:39:29,250 --> 00:39:35,100 partners were having this massive conversation about blood values lab results, 354 00:39:35,100 --> 00:39:44,010 saying We can tell this medicine's working or not. But by looking at, you know, the liver enzymes, the blood sugar, the triglycerides. 355 00:39:44,010 --> 00:39:49,770 So the conversation was very technical and was really technical about lab values. 356 00:39:49,770 --> 00:39:51,330 And what's the difference? 357 00:39:51,330 --> 00:39:59,060 What does it mean to a patient if this lab value changes and the patients there were saying, hold on a second you've not even mentioned? 358 00:39:59,060 --> 00:40:07,340 The most important thing for us with this disease, and the most important thing is we have this constant feeling of hunger. 359 00:40:07,340 --> 00:40:11,660 It's like being starved to death. Does it matter if we eat, it never goes away. 360 00:40:11,660 --> 00:40:17,690 OK, so we cannot stop eating. We cannot stop feeling this intense starvation. 361 00:40:17,690 --> 00:40:22,970 For us, that means we have no social life. It means we can't go to work. 362 00:40:22,970 --> 00:40:28,940 It means that we cannot interact with people in a way that they can understand because we just can't stop eating, you know? 363 00:40:28,940 --> 00:40:40,310 And so they they were fantastic. They brought to life the psychological, the daily, the social, the economic to their own economy. 364 00:40:40,310 --> 00:40:54,200 The economic impacts of this symptom hadn't even been measured by the company wasn't even part of the health economic model. 365 00:40:54,200 --> 00:40:56,690 So nobody brought up this subject. 366 00:40:56,690 --> 00:41:02,750 But they were so passionate that actually, if you look at the final report of this, it's what the committee focussed on. 367 00:41:02,750 --> 00:41:08,840 In the end, they focussed on this symptom and what an awful impact it had on people's lives. 368 00:41:08,840 --> 00:41:13,940 And they agreed that they were going to reimburse or recommend to reimburse the treatment 369 00:41:13,940 --> 00:41:19,640 because at least it seemed to have some effect against this symptom or they hoped it would be. 370 00:41:19,640 --> 00:41:26,510 But it was just a shame that the company making the medicine never bother to measure this. 371 00:41:26,510 --> 00:41:32,390 Bring it to a close as we've come to the IMF, I just want to thank you all so much for coming all this way. 372 00:41:32,390 --> 00:41:37,130 You've come from outside the UK today, and what a great deal. I learn something every time. 373 00:41:37,130 --> 00:41:42,698 Thank you, Neal. Thank you.