The National Institute for Health and Care Excellence (NICE) has issued preliminary guidance stating that three drugs used to treat cystic fibrosis (CF) should not be used within the NHS.
Kaftrio (ivacaftor/tezacaftor/elexacaftor), Orkambi (lumacaftor/ivacaftor), and Symkevi (ivacaftor/tezacaftor) are the three drugs in question, produced by Vertex Pharmaceuticals.
Jess and Will Meyrick have a 2-year-old daughter called Ottilie with the condition.
01:24 Keeping Otte healthy is a routine and involves a lot of time and effort.
01:30 It is a very debilitating condition.
01:34 Yeah.
01:35 She requires lots of different medications, physiotherapy for her lungs.
01:40 She's also pancreatic insufficient, which means that she has mucus sort of leading to the fattest ways of her pancreas.
01:48 So she requires daily pancreatic enzymes, which help absorb the fat in her food.
01:55 And that requires so much planning because every meal that she has, sorry, every meal that she has, we have to weigh out the fat content.
02:05 And if we get it wrong, there's repercussions in her bowels and it's so hard to manage and balance.
02:12 I mean, her physiotherapy is also, you know, it's a big part.
02:16 It's all about keeping their airways clear and clearing out that mucus.
02:21 And, you know, we've got a trampoline in the room because, you know, she's always having to do physical activity to try and keep that mucus shifted.
02:29 Antibiotic nebulisers once a day.
02:31 Yeah. And it's a progressive disease.
02:38 So, you know, the older they get, the harder that becomes and the more the CF takes over.
02:46 The damage.
02:47 And, you know, that's why we do it to try and prevent the damage done as much as possible.
02:58 In the UK, the biggest charity for supporting people with cystic fibrosis is the CF Trust.
03:10 And they did an Instagram post on Friday afternoon stating that the outcome of NICE is reported.
03:22 And I think it was just a huge shock to the whole community and a very difficult time because, obviously, the CF Trust don't control when the information from NICE is announced.
03:37 So, yeah, we were kind of left for a few days with really not a lot of information.
03:42 But still over the last couple of weeks, the information is not clear.
03:46 There is a lack of information.
03:47 Yeah.
03:48 I think there is.
03:49 There is, yeah, there is quite a lack of information.
03:51 The family is a little bit lost about what's going to happen.
03:54 I mean, it's certainly, the NHS and NICE keep reiterating that anybody that is already on Caftrio will maintain access.
04:06 But, and that's great, but there is something like 300 people every year, I think, I'm not sure if that's right, but there's a lot of people born every year with CF.
04:18 And if the funding is withdrawn, every single year there's going to be more and more people that don't have access to it.
04:26 And eventually there will be thousands and thousands of people that don't have access.
04:31 So it's great that the people who are on it will have access.
04:34 But what about everybody else that is going to be born, that is not yet born with CF?
04:40 I mean, what are they going to do?
04:41 So, yeah, it's really, really important that something is sorted out.
04:49 It is a lot of money, but...
04:52 It's a life, it's a life.
04:53 It's a life, yeah, I mean, the drug is phenomenal.
04:59 It's life changing.
05:01 We're not talking about a couple of years of extra life with, you know, the life they have been better.
05:07 We're not talking about doubling their life expectancy.
05:11 We're talking about, instead of them being in hospital multiple times a year, having, you know, week, two week stays in hospital at a time.
05:21 You know, some of these people that are now on Cafetrio are not going to hospital hardly at all apart from for checkups.
05:27 You know, their quality of life is hugely improved.
