Table 3 Supporting quotes
Expectations | |
Exi202 | Why would I trade [what I have] for having to hike up to London every week or something for bloods for six months? |
Exi203 | I think if it’s a cure… It doesn’t cure all the joint replacements, it doesn’t cure the arthritis, it doesn’t cure the hepatitis, it doesn’t cure the cirrhosis—it just solves a problem, which is bleeding. That’s what it does, it solves a problem. It means that you don’t bleed anymore. Does that mean that haemophilia is cured? It may do. I just don’t… The word doesn’t sit right with me for some reason |
Exi205 | And then also, just the process itself. I thought at the time… I mean, when I was doing some reading, I was still at Uni and I thought no way I’m doing anything like this at Uni, going in and out of that process |
But just the process of going in and out of hospital multiple times… And then even when I went to [names hospital] last year and did some more reading about it at that stage, I thought it was going to get mentioned in some form of a conversation about what it is and the future, as it always does now. But I just thought I’m not so sure I want to make my immune system go to tatters when we’re part of a worldwide pandemic, and also, I’m not so sure this is the most responsible thing to be doing in an NHS Covid crisis, really | |
There’s not enough evidence, not enough data behind that, and not enough people really going through the process of it yet | |
Exi208 | At the moment, it’s a little bit, I feel, like… not a waste of time, but I just feel like if you’re going through it you’ll put a lot of strain probably on your family as well as your work. I just feel that’s probably quite a massive reason why I wouldn’t feel comfortable going along with it |
My lifestyle at the moment, it fits around my treatment process. So, I just kind of want to keep it as much as normal as I can | |
Exi211 | They might be quite open to it, but I’ve only just started this job so… I’d have to… probably give it a while before I… |
Exi212 | Yes… I mean, thinking of where personally I am now with college and where I’ll be next year, if—if—I were to be, say, going on it next year, next year is one of the most important years for not only graduating college but then also setting up my professional life. So, that’s not something I’d really want to sabotage by going onto this, and then having the next X, Y, Z. The pay-off just wouldn’t be worth it when the medication that I’m on is allowing me to live the lifestyle that I currently am |
Exi213 | If we’re speaking exclusively on haemophilia, then I think it’s quite a gold mine because there’s just so many options out there. It’s better now to be growing up with haemophilia than it was ten years ago, and obviously ten years before that and so forth. Hopefully, children that are born with haemophilia A now, for example, they’re not going to have as much joint damage as I do |
Outcomes | |
Exi201 | It’s an interesting question, because certainly when they talked about [grandson] having gene therapy, his mother had said a flat no until another few years until they see if there are any side-effects that come out |
Her reason is give it 20 years and see what happens. Because the last time we discussed it was in the very early days and she was saying we don’t know what the side-effects are, we don’t know what the long-term effects are, we don’t know how long the effects will last, we don’t know whether it will be worse after if they do come back | |
Exi202 | Maybe in ten years when it is one and done, maybe that will be different |
If my treatment wasn’t performing as well, maybe that would be a different question, a different equation almost | |
Exi203 | If they turned around and said, ‘Right, gene therapy is for you for all these reasons,’ and I ask, ‘Well, what are the chances of it working? What are the chances of it failing? What’s the chance of me never needing to inject again?’ You know, the fact that I’m 57… ‘What’s the risk of bleeds?’ To me, all the answers there are all unknowns because it’s gene therapy, it’s new |
I don’t think. I think, for me, it’s the way… I guess the way the language is all about curing haemophilia, and I find that a little bit uncomfortable in that haemophilia is therefore defined as this problem to be fixed, and therefore I am… there’s something wrong with me that needs to be corrected, rather than ‘Here’s some drugs that can allow you to live a flourishing life’ | |
Exi205 | For starters, everyone’s saying it could work, it might not work, so ok we’re in a 50/50 situation, flip of the coin anyway. And then it might work but you might only get to 12 percent. You might only get to 12% potentially, or you might be 95%. I mean, it really is a flip of the coin, and we don’t really know… And there’s no logical reason… there doesn’t seem to be a logical reason right now about where you sit |
I think there are so many other things that could come first to make haemophilia better at a far more reasonable cost and would have a far more direct impact on 99 percent of people if we started doing it tomorrow morning | |
Exi206 | The same with gene therapy. There’s a lot of talk of roughly eight years because that’s as much data as we have, and maybe levels dwindle. So, especially getting older, would I take that one jab for eight decent years and then go back to being a normal haemophiliac? Maybe. It’s difficult to quantify it and to make the decision based on that because… it does work for a lot of people… it’s just you don’t know… It’s like a lottery, though—you go into it and you don’t know what level you’re going to get |
I think it’s the finality of gene therapy. Moving onto [Names FVIII Mimetic Product], if it doesn’t quite work for me—I know, and I’ve discussed it with my centre about switching to an extended half-life because that’s still an option. But if you go onto gene therapy and it doesn’t quite work, what situation are you left in? | |
Exi212 | But then I also think… well, I also kind of like the security that I have with my medication, knowing that if I take my medication like I’m supposed to then nothing’s going to happen. Whereas with gene therapy I feel like there’s still a lot that’s kind of unknown |
Access | |
Exi202 | It’s not been mentioned to me. Again, I have struggles trying to get them to elaborate on basic things like what’s happened to those scans you did, no I don’t want to be changed from the current treatment I’m on. So, no—no discussions about gene therapy |
I changed centre during Covid, so that’s been a huge barrier to just accessing a lot of things in general | |
Exi203 | And I don’t know the politics behind the decision-making that different treatment centres have got. I don’t want to get involved in that, but I assume and I’m guessing that different treatment centres have got different views because of different levels of funding or whatever it might be |
But it is kind of ‘We think this is going to be good for you for these reasons,’ and I go, ‘Yes, if it stops me from bleeding then great, fantastic.’ I did push the [names treatment] one though. I did say, ‘I want to do this as soon as possible.’ So, that’s the only one time that I’ve gone in and said, ‘Can we stop faffing around with this once-every-two-days stuff? I want to change because I’m hearing great things.’ And they just said, ‘Yes, we’ll do it as soon you’re fit and ready.’ And we did and it’s been great | |
Exi204 | So there is a financial part of it, but [my centre] is a big centre, similar to other areas, so I presume they have leverage in terms of how it would work… So, it’s just understanding how the decisions are made. But we’re not given a choice |
I think the NHS has to ultimately derive the greatest value from the smallest amount of money | |
Exi206 | There are still some times where we butt heads, which… rightly so, because they’ll say one thing and I will try and argue my point against it. But it’s a much more open conversation. I’m not being dictated to as to what I need to do. They will listen to me and offer their advice, so it’s much better |
From everything I’ve heard about gene therapy, it costs a lot of money and it’s a bit of a 50/50 thing | |
Exi207 | The centre I was at was not very helpful, would not refer me to the centre to get onto the trial—which at the time I was very annoyed about |
Exi208 | I don’t think I’d be one of the main patients to benefit from it in their eyes. So, when you look at the list of priorities—because it’s not going to go to everyone on that patient list—I wouldn’t be top of that list |
Exi211 | So, I didn’t even know it was a thing until I went on Facebook, to be honest. And then I spoke to a few more people around my age and they said they’re all on it, so I just wondered why I’ve not been offered it |
Exi212 | It was that they got a cheaper deal buying a bigger bunch of, say… I can’t remember what any of them are called, but they get a bigger deal for buying more of the same product, so they’ll buy that, put more patients out on it, because obviously it saves them more money at the end of the day. So, I understand that it’s not really a possibility to have “Oh, here’s all these different products for all these different patients who need what they need.” I understand the constraints on that |
Exi213 | At all the centres that I’ve been to it’s just not something that’s been brought up with me at all |
Information | |
Exi201 | I learnt about it when my daughter told me about it. I’ve always ignored my haemophilia, I’m not part of any real haemophilia group or…I’m not a member of the Haemophilia Society and I don’t read things about it. And I don’t have any things that come into my inbox about it. It’s just one of those things I’ve tried to… well, I just have ignored. So, I heard about it when [she] told me about it |
Not that I’m aware of it. Mind you, I only have a five-minute conversation with my guy anyway, about every six months. He phones me up and says, “Are you doing all right?” and I say yes. He says, “Have you had any bleeds?” and I say no. And that’s it | |
Exi203 | I think what has always frustrated me a little bit is there’s no sort of education about the different treatments in layman’s terms. I’m headlines and not detail, just like a lot of other people—I do the headline and the detail I expect layman’s terms information |
Exi205 | I think they should be putting more information out, but I’d like to know if they actually have the information themselves. That’s the thing I’m really curious about because I’m not so sure they do. I think that’s why they might all be… none of them are putting information out because I’m not sure they’ve potentially got this actual data to show us all yet |
And doing some reading about it… The thing I’ve found with reading about it is… like I said, being a person who’s focused by data, there just never was anywhere that told me the exact… not an exact number, but ranges were very coy and ranges were too big that I don’t want to get involved in that | |
Exi207 | I don’t think so. I’d have to go search it and do a bit of a… like, you’d have to do a dive in the studies because it’s still in trial period, so the information’s not that easy to ask, like at the snap of your fingers |
So, my first consultation there took about three and a half hours because we literally sat there and went through everything. There are still some times where we butt heads, which… rightly so, because they’ll say one thing and I will try and argue my point against it. But it’s a much more open conversation | |
Exi208 | But I think the thing for me is… I think I’d want to just see a bit more of the longevity results. And I think if I was to ever make the move… I think this time last year I was a definite no, but I think the idea around it is actually… I think it is going to probably be the future. So, for me, I’d probably actually want to speak to someone –when I say ‘someone’ I mean not my direct team but maybe someone who’s gone through the process, as such—first. But short term, the next 12 to 24 months, I probably would still be a no if it was offered |
Yes. I mean, I’m sure there is a vast amount of evidence and results out there. But the next step is probably accessing it and how easy it is to access it. I’d like to think I’m at a reasonable level, where I can go out and find that sort of stuff. But other than the AGM for The Haemophilia Society, I have found it quite hard to kind of find information, or up-to-date information anyway | |
Exi209 | So, I think it’s a mixture of there is the info out there, but you’ve got to look for it, it’s not fed to you on a plate, and also hearing experiences from people who have actually been through it. I think if you’re not proactive to find someone like that then there’s not enough information |
Exi211 | I feel like there’s still not enough to go on here—which is fine, because it’s still new, they still don’t have as big a pool of people that are on it to say, “Here’s what our research shows” |
Getting the consultants to speak about it a bit more, really. [My consultant] doesn’t actually really tell me about anything that’s new. The nurse tries to but the consultant obviously leads the consultation, so… |