Tonight on Sunday ` how would you feel? You're seriously ill, there is a cure, but they won't pay for it. Without this drug, ultimately, you'll die. Without this drug, ultimately, you'll die. Yeah. But it costs too much. The fluid going into Maria's arm is like liquid gold. Available worldwide,... It's a scandal. ...but not here. Pharmac are telling us, 'Your life isn't worth saving.' What price is life? (SNORES LOUDLY) There's no doubt that if a child snores, it's really bad for them. It's bad. The perils of snoring. He's waking up 500 times a night. # Need some sleep. # What you need to know about snoring. (SNORES) Copyright TVNZ Access Services 2013 Kia ora. I'm Miriama Kamo. How much is your life worth? It's priceless, right? Well, actually, no. There's a tiny and desperate group of Kiwis ` one could even be your neighbour ` who face death from a rare disease called PNH. Now, they could be saved by a new, entirely effectively medical treatment. But Pharmac, the funding body, doesn't want to pay for it. Why is that? The price tag ` $600,000 per person per year. This from Ian Sinclair. This is a horrible thing to live with. It really is. When you see some of the effects, uh, long-term, in terms of debilitating fatigue, chronic pain, um, espec` I guess the most serious concern is` is of death. Daniel Webby would like to see his 7-year-old son, Wu, grow up. But to Pharmac, this 33-year-old father may be too expensive to keep alive. Daniel is one of eight Kiwis who need treatment for an extremely rare disease called PNH. It's a bone marrow disorder which results in red blood cells being produced which are defective. Um, those blood cells, in turn, get destroyed by the immune system. This solo dad has to live with the knowledge that time is running out. Mwah! I'll see you later. See you, Dad. The median survival is 10 years, so on average, people will survive around 10 years post-diagnosis. SLOW MUSIC PNH strikes only five in a million people. It struck Daniel without warning. How long have you survived? > How long have you survived? > So far, I've lived with the disease since diagnosis for three years. However, there is a lifesaver ` a new wonder drug called Soliris that will keep PNH at bay. But there's a catch. The cost of these two vials of Soliris, making just one dose, is over $20,000. And the asking price to keep Daniel alive for one year? Up to $600,000 ` a price Pharmac so far is not prepared to pay, meaning Daniel and the other Kiwi PNH sufferers could be left to die. I guess the feeling certainly is that` that Pharmac are telling us, you know, 'You're not worth it. 'Your life isn't worth saving.' That's the feeling we're getting. I think, to be honest, it's a scandal, and I think Pharmac really need to do something about it. Waikato specialist Dr Humphrey Pullon says Soliris is only expensive because it cost hundreds of millions of dollars to develop. I think we have to be realistic that drugs that are very effective for relatively rare disorders unfortunately cost a lot of money. And NZ has to come to terms with that. PNH is not the only rare disorder in that category And there's no denying Soliris works. This is a superb therapy. This is a drug that, you know, on average, can prolong a PNH patient's life by in excess of 20 years. UNSETTLING MUSIC Daniel is living life one day at a time. On his neck, deadly blood clots leave tell-tale scars. And quite significant wounds around` around my body, which, yeah, essentially were a direct result of PNH. Come on in. Daniel used to have a full-time job in IT. Now he's unable to work. It actually took three months to heal, so... At the moment, his clots are only skin deep. The longer something takes to heal, the greater the risk of scarring. The worry is where they'll spread to. Clots can occur in all sorts of organs, but particularly within the abdomen. UNSETTLING MUSIC Clots caused as the body attacks its own red blood cells. particularly affecting the liver, and also affecting the brain. And we know that patients that die from PNH ` the commonest cause of death is from clots or the complications from clots affecting vital organs. Do you have an idea of how much time you have left? I-I can't say how long I've got, but I clearly feel worse. My` My health is deteriorating. UNSETTLING MUSIC For some, it's already too late. Dr pullon told Sunday that Pharmac's refusal to fund Soliris may have led to the death of one NZ woman. we've had the experience of a patient who was at North Shore hospital, being looked after there, who applied for Eculizumab Soliris and was turned down. I'm sorry to say that that patient has since died from their PNH. Now, that is a travesty, and I feel very sad that that's happened to somebody in NZ, whose life could have been saved and certainly prolonged by having Eculizumab available and funded. And she would have known she could have been saved? She would have known. She was certainly well aware this medication was potentially available in the worldwide, you know, market. We're going to the zoo. A lethal disease ` a remedy out of reach for Daniel and young Wu. I don't want him to grow up without a father, so I'm-I'm hoping that` that I can secure access to a treatment which I know will prevent premature death. My plea to Pharmac is please look at this seriously, because there are, you know 10 or so patients in NZ who have an enormous amount to gain from this medicine, and they're currently at risk of dying from their disease. EASY-GOING MUSIC Next up ` too scared to come home. If I go home, I will potentially die. How England has rescued a Kiwi PNH sufferer. It's so hard to believe that NZ is taking this position. EASY-GOING MUSIC Expat Kiwi Maria Piggin lives in exile. She fears that if she quits London for NZ, Pharmac will let her die. We don't live in, you know, the Third World. It's just` It's so hard to believe that NZ is` is taking this position. GENTLE MUSIC Okie-dokes. Sharp scratch. Every fortnight, England's national health service gives Maria a lifesaving medication that NZ is refusing to fund. So today, um, I'm having an infusion of Soliris. Soliris ` brand name for the drug Eculizumab. Without it, Maria will probably die from PNH, the same rare and deadly blood disease that affects a handful of Kiwis back home. This is the magic stuff, really, that I need to be able to have some sort of quality of life and not live in fear of thrombosis or any of the other complications that PNH can deliver. So in other words, what you're saying is that without this drug, ultimately, you'll die. Yeah, basically. But there's a price. The fluid going into Maria's arm is like liquid gold. What does it cost, do you reckon, this` this, what, this amount you've had today? So, without maths being my best subject, I think Eculizumab is worth �300 per person per year. That's about NZ$600,000 per person per year. This bag's probably worth about �10,000. Expensive, yes, but Maria maintains NZ also has a duty of care. I mean we're the only country in the OECD who don't pay for it. I mean, there's countries like Lithuania, Algeria, Greece, who pay for this drug, and you couldn't say that NZ was poorer than those countries. REFLECTIVE MUSIC It's left this successful London lawyer caught in an awful dilemma. London's been good to Maria Piggin, and she'd be the first to admit it. It's given her opportunity. It's given her a career. But like most Kiwis, she'd love eventually to fly home. However, unless NZ, like Britain, is prepared to fund the lifesaving medication that she needs, a trip home would be a death sentence. So she's stuck here in London, in limbo. As a NZer, I always imagined that I would always have the option to go home. So it was always something that I thought would happen in the future. If I go home, I will potentially die without this drug. Once a patient's started on` on Soliris, if they can't access it again, you` the deterioration in health by stopping the drug is` is clearly dangerous. Professor Peter Hillmen, England's top authority on PNH, says the $600,000 price tag on Soliris is relative. Do you fund leukaemia, where the care can cost, you know, hundreds of thousands of pounds? Is that acceptable? You know, paediatric intensive care units ` we often spend �500,000 on a` on a child, or a million pounds looking after them through intensive care. Is that acceptable? So where do you draw the line? BOTH CHAT SOFTLY Back in NZ, without Soliris, Kiwi father Daniel Webby's struggle with PNH worsens. I do the best I can. I-I often, you know, feel that I haven't got the energy that I'd like to have when I'm looking after my son. This is a very destructive condition. UNSETTLING MUSIC In May, Pharmac published a proposal to decline funding Soliris on the basis of cost. They called for public submissions in response. One of those respondents was Dr Humphrey Pullon. He questions Pharmac's costings. Well, the figures put out in the consultation document said that 20 patients needed this drug, whereas, you know I believe it's considerably fewer than that. How much fewer? Well, in the order of eight to 12. And, you know, the other thing is the price that they quote in terms of $600,000 a year, I think could be considerably less if meaningful dialogue with the drug company occurred. Have you been given any indication that` that that could be the case? Well they're certainly prepared to discount it. I guess the question is how much further might they be able to go. I mean, I think, you know, if Pharmac were serious about trying to get this medicine funded in NZ, I think there is room to negotiate with the suppliers to try and get a better price. Have they done so? Appallingly, no. And the interactions between Pharmac and the drug company have been very few and far between, and to my mind, there's been no serious attempt at negotiating a reasonable price and a reasonable deal to have this drug available. PENSIVE MUSIC Sunday approached Pharmac for an interview on their proposal to decline funding for Soliris. They declined to appear, saying public submissions are still under review. We wanted to ask Pharmac why did Pharmac claimed it might have to support up to 20 PNH patients when the specialists say the likely figure is less than half that? Why did it say each patient would cost $600,000 when Sunday's been informed, in fact, the manufacturers are ready to negotiate a lower price? And why, when 40 other countries have been funding this drug for years, was a NZ woman left to die last year? I think they've really put this in the too-hard basket and said, 'It's too expensive. We can't go there.' My response to that is, well, here are a small number of NZers who really need this treatment ` highly effective treatment. Please, please go back to the drug company, please try and get some sort of deal together, where this drug can be made available to those who really need it. You have to accept that if you make a decision not to fund Soliris for PNH, that you're making a decision that some of those patients with PNH will not survive. Hard decision. GENTLE MUSIC Back in NZ, Daniel Webby is left not just with fears for his own health, but for a son who may well lose a father. Does Wu know about your situation? He was there while I was in hospital for about three weeks. He's certainly seen me in far worse conditions than I am currently. It certainly worries me, um, thinking about, you know, even if it's not death, having a serious stroke or losing some` some organ that's quite useful, and then all of a sudden, you know, I'm permanently debilitated that` in a way that I can't look after him. A father who knows our health system has the power to save him. He just fears they think he's not worth it. GENTLE MUSIC Well, Pharmac has received more than 300 submissions on its proposal not to fund the drug. It says it's a difficult and important decision, and that it will consider those submissions. This, it says, will take time. Hopefully, of course, for the patients, there is time to play with. Next ` snoring. It's not cute, and it's not funny. One thing he's dead-set scared of... LOUD SNORING OMINOUS MUSIC ...is going to sleep. (SNORES, BREATHES LABOUREDLY) Because when Geordie sleeps, he snores. And between the snores, he stops breathing. That could be a simple mistake. Or a deliberate move for a long-term advantage. Or a deliberate move for a long-term advantage. Perhaps. But, no, you're someone who plans ahead. I'm thinking you don't make a move unless you've thought things through. And if you don't have an answer, you'll find someone who does. Make the right move. Ask how the ANZ KiwiSaver scheme can help you achieve your long-term goals. Nice move. Nothing simple about snoring. Apart from the nuisance value, it can cause daytime drowsiness, irritability , lack of focus, decreased libido and quite serious psychological and social damage. And that's just for you and me. But for kids, snoring can have devastating effects lasting a lifetime. Here's Kerri-Anne Kennerley. GENTLE MUSIC Meet 4-year-old Geordie Brown. His sleep story is a beauty. The bunny one. The bunny one. < OK. He likes to help Mum in the kitchen,... You hungry? ...likes cheese sandwiches,... Hey, bubby, how are ya? ...likes to see Dad when he comes home,... You had a good day? READS: First plane to fly was made of wood. ...and likes a bedtime story. You comfy? Hang on. Let's do this. But one thing he doesn't like, one thing he's dead-set scared of... LOUD SNORING OMINOUS MUSIC ...is going to sleep. (SNORES LOUDLY, BREATHES LABOUREDLY) Because when Geordie sleeps, he snores, and between the snores, he stops breathing. CONTEMPLATIVE MUSIC He's a gorgeous little boy, as any mother would say that, but, um, he's... He's a really beautiful personality, but he's obviously not been sleeping,... (CHUCKLES) for his whole life. For Jen and Johnson Brown, one of the signs something wasn't right was that Geordie never ever slept like a baby. I kept a sleep diary when he was 3 months old till probably 9 months old, because he would sleep for 20 minutes and wake up screaming, and there was just nothing we could do. I tried Baby Mozart, I tried every sort of technique available for a child that has trouble going to sleep, and he just did not want to go to sleep, full stop. But Geordie's lack of sleep was taking its toll. Whoa! Whoa! (LAUGHS) > He took longer to learn to talk than other kids his age. Going backwards now. > Then there was his class photo from preschool. Oh my goodness! My poor little bubba. All I can see is the bags under his eyes. Like, they're just so dark, and he just looks tired. It would be months before they'd find out why going to sleep scared Geordie so much. I want to go for a drive, Daddy. Oh, you want to go for a drive? Why is that? Things got so bad that the only way to get Geordie to sleep was to get in the car. Why? # I can't get to sleep. # I think about the implications. # Jen and Johnson felt very alone. Sleep-deprived themselves, they got little help and fewer answers from doctor after doctor. It wasn't until they were referred to one of Australia's leading child sleep specialists that they discovered what damage sleep deprivation was doing to Geordie. You can expect around about a 10-point decrease in intelligence quotient, or IQ, as it's known. Wow! So it's really quite a serious reduction in your ability to learn. And that's just the beginning. You're doing OK. Beautiful. Paediatrician Dr Jim Papadopoulos and his team at St George Private Hospital in Sydney test and treat children who aren't getting enough sleep. And what they're discovering will jolt you wide awake. He's not struggling` He's not struggling` WOMAN: Not struggling and not screaming, not crying. A lack of sleep... and can lead to... What about in babies? What about in babies? Babies ` it affects their development. It can slow them down so that they don't smile at the right time and don't roll at the right time. Some of the patients are only a few weeks old when they arrive. What they all have in common is snoring. When you ask, 'When did this child start snoring?' Usually, it's from the age of around 2. Occasionally, it can happen from infancy. So when should you treat it? As soon as you know. Here's why. This is what a healthy airway looks and sounds like in a normal sleeping person. And this is what happens in the airway of someone who snores. It begins closing up, reducing the amount of oxygen reaching the lungs, the bloodstream and the brain. At times, no air gets through at all. A lack of oxygen can damage your brain and your body. Yet most parents think snoring is a sign their child is in a deep sleep. You go and check on them, and if you hear snoring or they're still asleep, it's like, 'Well, that's, uh`' You know, 'We are doing well.' What's interesting is that snoring and sleep apnoea is often genetically passed down through the mother. Nici wears a CPAP mask to bed. So does James. Now you turn it on. I'll get a book for us. So, too, does baby Ethan. He's been snoring since birth. What went through your mind when you've gone, 'He's snoring?' 'Here we go again.' 'Here we go again.' BOTH LAUGH Dad Jeremy is the only one not masking up. He wears something else. In May, when Ethan was six weeks old, he was admitted for a sleep study. It's almost every minute that he will stop breathing while he was sleeping, so... per hour. He held his breath for 14 seconds at one stage and, thankfully, started breathing again, but it was quite scary. This is video from the study. Now try holding your breath for 14 seconds. SILENCE (CHOKES) He basically chokes himself awake. He basically chokes himself awake. That's it, yeah. He basically chokes himself awake. That's it, yeah. That's pretty scary. Well, there's an adrenaline rush that wakes him up. Seeing Ethan gasp for breath brought it home for me. It's very distressing. It's very distressing. It's distressing just to look at it, let alone experience it. (CRIES) Breathing difficulties like Ethan's could also be linked to sudden infant death syndrome, or SIDS, so Ethan was fitted with a CPAP mask which immediately helped his breathing. There. The snore's gone. But the long-term solution will be surgery. The full-throated cure seems to be, as doctors are discovering, removing the tonsils. Together with the adenoids, they were once routinely taken out. The surgery is now less common, but in severe cases of sleep apnoea, losing them is the start of a more restful life. So, you're ready to have your tonsils out? Three weeks ago, Geordie went into surgery. All right, sweetie. All right, sweetie. Give him a cuddle. Cuddle. Cuddle. OK. Want another one? Want another one? OK. Brave boy. His dad was nervous. (CHUCKLES) No tears. (CHUCKLES) He didn't need to be. The results ` immediate. DAD: 'There's no snoring.' Um, but he's also... You can... He's dreaming. Because also, with sleep apnoea, obviously waking themselves up every two minutes, they don't` they don't dream, because they don't go into a deep sleep. They don't release growth hormones, so we've been told that we should see, in the next six months, Geordie shoot up. Whoo-hoo! And the Dahms, the family that used to snore together, now know a good night's sleep. Their sweet dreams are reward enough for Dr Jim. By treating the children and educating the parents, I know I've saved marriages. < That must be very gratifying. < That must be very gratifying. It's fantastic. I've got the best job in the world! SILENCE Oh, isn't that great? All right. That's us for tonight. Join us on Facebook ` Sunday TVNZ, and have a great week. Nga mihi nui, hei kona.