Genetic Drug cure
Discussion
Heard a news article today and it got me thinking after reading the 'Logans Run' thread.
A 5 month old baby has been treated with a drug for a genetic condition at a cost of £1.8million,without the drug life expectancy is around 2 years.Obvioulsy it is good news for the parents but where is a line drawn?
If the gene therapy doubles or triples life expectancy how much on top of the £1.8m will it cost for ongoing health care?
Story here...https://www.expressandstar.com/news/uk-news/2021/06/01/five-month-old-baby-is-first-nhs-patient-treated-with-drug-for-genetic-condition/
This bit stood out to me “The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers.
This isn't me slating what's been done or will be done more a is there a line we should draw at some point?
A 5 month old baby has been treated with a drug for a genetic condition at a cost of £1.8million,without the drug life expectancy is around 2 years.Obvioulsy it is good news for the parents but where is a line drawn?
If the gene therapy doubles or triples life expectancy how much on top of the £1.8m will it cost for ongoing health care?
Story here...https://www.expressandstar.com/news/uk-news/2021/06/01/five-month-old-baby-is-first-nhs-patient-treated-with-drug-for-genetic-condition/
This bit stood out to me “The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers.
This isn't me slating what's been done or will be done more a is there a line we should draw at some point?
Saleen836 said:
This isn't me slating what's been done or will be done more a is there a line we should draw at some point?
NICE guidance on value for money on interventions:https://www.nice.org.uk/Media/Default/guidance/LGB...
Biggy Stardust said:
The NHS was never set up for this purpose, just basic healthcare without worrying about a doctor's bill.
It shoud never have morphed into 'anything for anyone, regardless of cost' & needs to revert closer to its original model.
Per my post, it isn't 'anything for anyone, regardless of cost' - there are clear guidelines set by NICE.It shoud never have morphed into 'anything for anyone, regardless of cost' & needs to revert closer to its original model.
vaud said:
Biggy Stardust said:
The NHS was never set up for this purpose, just basic healthcare without worrying about a doctor's bill.
It shoud never have morphed into 'anything for anyone, regardless of cost' & needs to revert closer to its original model.
Per my post, it isn't 'anything for anyone, regardless of cost' - there are clear guidelines set by NICE.It shoud never have morphed into 'anything for anyone, regardless of cost' & needs to revert closer to its original model.
It's way beyond its original intent. Some of the original assumptions used when setting it up failed decades ago and contributions have been unable to keep pace.
A radical rethink is desperately required, especially for the less well off IMO. The wealthy will be able to afford private care should the system fail.
The NHS won’t be paying a penny towards the treatment, this will be funded by the Pharma company developing the treatment.
SMA is a rare disease, costs for specialist genetic treatment are high because of this.
Ignore the click bait headline - pilot tests like this have to happen to better understand how to treat the condition and also create a scalable / affordable treatment.
Also the GOV recently published their rare disease treatment framework, this is the upshot. Companies taking the initiative and developing treatments for often overlooked conditions.
SMA is a rare disease, costs for specialist genetic treatment are high because of this.
Ignore the click bait headline - pilot tests like this have to happen to better understand how to treat the condition and also create a scalable / affordable treatment.
Also the GOV recently published their rare disease treatment framework, this is the upshot. Companies taking the initiative and developing treatments for often overlooked conditions.
Edited by moustachebandit on Tuesday 1st June 22:11
moustachebandit said:
The NHS won’t be paying a penny towards the treatment, this will be funded by the Pharma company developing the treatment.
SMA is a rare disease, costs for specialist genetic treatment is high because of this.
Ignore the click bait headline - tests like this have to happen to better understand how to treat the condition and also create a scalable / affordable treatment.
One of the advantages of the NHS. We are a perfect setup for a lot of research. There is a lot of interesting stuff coming from big data and machine learning analytics of NHS records. Because the NHS exists the U.K. is a world leader in many areas of pharma and medical research.SMA is a rare disease, costs for specialist genetic treatment is high because of this.
Ignore the click bait headline - tests like this have to happen to better understand how to treat the condition and also create a scalable / affordable treatment.
I'm on an experimental biologic drug, Interleukin 17, not a million though, i think 1k per injection after discount. To get to this point I've had to go through 5 other treatments, multiple doctor assessments, basically standing in a room half naked whilst 8 doctors look at you.
You then have to meet criteria for the drug, before could even get on it. This has taken about 7 years. it is not simple and involved me escalating a few times as you find if you don't actually ask you won't get.
The one thing that i noticed is this drug has some controversy in testing, it is weird really, each drug developed, billions invested they can't afford for it not to work.
You then have to meet criteria for the drug, before could even get on it. This has taken about 7 years. it is not simple and involved me escalating a few times as you find if you don't actually ask you won't get.
The one thing that i noticed is this drug has some controversy in testing, it is weird really, each drug developed, billions invested they can't afford for it not to work.
Since we have just spent £372 billion pounds (and rising), ruined everyones life for a year and a half and all to save us from a virus which 99% of people survive... money is NOT an object.
If you supported lockdowns and the governments policy for the last 15 months then I don't see how you can against this baby receiving this treatment, taxpayer funded or not.
If you supported lockdowns and the governments policy for the last 15 months then I don't see how you can against this baby receiving this treatment, taxpayer funded or not.
Another rare disease here.
Gene therapy is seen as the golden bullet to improve things long term, so very interested in these sort of things.
As others have said, some treatments are gateways/pathfinders for treatments that might in the future have wider applications and/or improve understanding.
If I live long enough it might spare me from fortnightly infusions of replacement enzyme, funnily enough the nurse has just put the cannula in for this weeks adventure, so after lunch expect to spend a few hours getting a top up that will hopefully keep my muscles from wasting away too quickly.
There is an infant form (I presume zero/near zero enzyme production) which pre-infusions being available: 100% would not make it to a year old. With diagnosis and treatment: 100% survive beyond year one and into later life.
Should it be NHS funded? I am very grateful that it is, in some countries like USA there are the terrible battles with insurance companies to get treatment, even in the UK it is not consistent. But there are countries like New Zealand that have not supported it and the only way patients get treated is when on trials or through the drug company covering it. They leave people to waste away and die... and they do.
I can see there have already been a variety of opinions. I'm not expecting to change any views, just thought my perspective was relevant. My treatment is also regarded as expensive, but as someone has already said; there is an approvals/costing/benefit assessment which I can only be happy that I am (currently) in the 'we think it is worth trying to keep you going a bit longer....'
Gene therapy is seen as the golden bullet to improve things long term, so very interested in these sort of things.
As others have said, some treatments are gateways/pathfinders for treatments that might in the future have wider applications and/or improve understanding.
If I live long enough it might spare me from fortnightly infusions of replacement enzyme, funnily enough the nurse has just put the cannula in for this weeks adventure, so after lunch expect to spend a few hours getting a top up that will hopefully keep my muscles from wasting away too quickly.
There is an infant form (I presume zero/near zero enzyme production) which pre-infusions being available: 100% would not make it to a year old. With diagnosis and treatment: 100% survive beyond year one and into later life.
Should it be NHS funded? I am very grateful that it is, in some countries like USA there are the terrible battles with insurance companies to get treatment, even in the UK it is not consistent. But there are countries like New Zealand that have not supported it and the only way patients get treated is when on trials or through the drug company covering it. They leave people to waste away and die... and they do.
I can see there have already been a variety of opinions. I'm not expecting to change any views, just thought my perspective was relevant. My treatment is also regarded as expensive, but as someone has already said; there is an approvals/costing/benefit assessment which I can only be happy that I am (currently) in the 'we think it is worth trying to keep you going a bit longer....'
pavarotti1980 said:
What should happen to these patients with very rare conditions if reverting back to a basic healthcare model?
I consider it should be equivalent to the bit where your insurance company says "I'm sorry, this isn't covered". We can't afford everything, something has to give.I'm sure your beliefs are closer to 'treat everything' but that's not sustainable & is perhaps the main reason the NHS has been "in crisis" pretty much every year since its inception & will never have sufficient funding.
(I accept there are outliers where treatment will advance general medical knowledge but the basic point remains.)
Biggy Stardust said:
I consider it should be equivalent to the bit where your insurance company says "I'm sorry, this isn't covered". We can't afford everything, something has to give.
I'm sure your beliefs are closer to 'treat everything' but that's not sustainable & is perhaps the main reason the NHS has been "in crisis" pretty much every year since its inception & will never have sufficient funding.
(I accept there are outliers where treatment will advance general medical knowledge but the basic point remains.)
It has nothing to do with "my beliefs". Put simply if someone has a rare medical condition your suggestion is to self fund (which is already available for drugs which are not approved on the NHS) or no treatment at all?I'm sure your beliefs are closer to 'treat everything' but that's not sustainable & is perhaps the main reason the NHS has been "in crisis" pretty much every year since its inception & will never have sufficient funding.
(I accept there are outliers where treatment will advance general medical knowledge but the basic point remains.)
It will be interesting to know what you think is acceptable for the NHS to treat.
pavarotti1980 said:
It has nothing to do with "my beliefs". Put simply if someone has a rare medical condition your suggestion is to self fund (which is already available for drugs which are not approved on the NHS) or no treatment at all?
No- if someone has a rare condition that is extremely (excessively) expensive to treat then yes. Please don't misrepresent what I said.pavarotti1980 said:
It will be interesting to know what you think is acceptable for the NHS to treat.
It was set up for the likes of broken bones &illnesses/diseases.Something along those lines would be good.I believe that the likes of boob jobs & sex changes should be self funded or not treated- I think the psychological necessity for ops like this is somewhat overstated. I'd add to that list treatments costing millions that prolong life for a very short period; I'm sure the NHS could employ someone to draw the exact line subject to a suitable brief.
You might call me lacking in empathy- I'll respond by pointing out that funding is not a bottomless purse & a line has to be drawn somewhere.
Edited by Biggy Stardust on Wednesday 2nd June 17:18
Biggy Stardust said:
It was set up for the likes of broken bones &illnesses/diseases.Something along those lines would be good.
I believe that the likes of boob jobs & sex changes should be self funded or not treated- I think the psychological necessity for ops like this is somewhat overstated. I'd add to that list treatments costing millions that prolong life for a very short period; I'm sure the NHS could employ someone to draw the exact line subject to a suitable brief.
You might call me lacking in empathy- I'll respond by pointing out that funding is not a bottomless purse & a line has to be drawn somewhere.
If by “boob jobs” you mean reconstructive surgery following mastectomy, yes, I will say you are lacking empathy.I believe that the likes of boob jobs & sex changes should be self funded or not treated- I think the psychological necessity for ops like this is somewhat overstated. I'd add to that list treatments costing millions that prolong life for a very short period; I'm sure the NHS could employ someone to draw the exact line subject to a suitable brief.
You might call me lacking in empathy- I'll respond by pointing out that funding is not a bottomless purse & a line has to be drawn somewhere.
Edited by Biggy Stardust on Wednesday 2nd June 17:18
I’m not convinced by some of the things the NHS does, such as the special fund for expensive cancer drugs that NICE won’t approve (a purely political funding of a specific illness based on media pressure) but to say “the NHS wasn’t set up for this” is just wrong. That argument makes as much sense as refusing to fund Typhoons because when it was set up they only has Sopwith Strutters.
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