Petitions and activism
to think if a 'miracle drug' is out there it should be accessible.
sudaname · 01/11/2012 20:09
Have just read that a new drug has been developed that is to be a huge step forward for Cystic Fibrosis patients. Then l thought 'Ere we go again' as l went on to read the drug company and NHS have yet to have talks about the costing of it. No doubt it will be ridiculously highly priced out of most trusts budgets and so wont be readily available to CF sufferers for a long time, sadly too late for many. Like my beautiful DGD aged 12 for one 
. There just seems no sense of urgency whilst it is agonising for the sufferers and their loved ones to know there is something out there but beyond their reach.
I mean should the drug companies be allowed to do this 'Nuh, Nuh, Nu ,Nuh, Nuh look what I've got' routine and then put an impossible price on it. They did this a while ago with an 'amazing MS' drug a while ago. My son has MS too btw ( l'm not the best person to be related to obviously, so much illness in the family - so if you discover me on your family tree , erase me immediately ! 
).
It's just so sad that years later these 'miracle breakthroughs' etc are still not out there or you read about one or two people who managed to get it by taking on the might of the NHS and winning their case.
Sorry rant over.
Now the real reason for starting this thread Anyone care to sign this e-petition for this drug to be priced reasonably ? Not to lay the emotional blackmail on too thick but if you could all meet my DGD l am sure you would, to help lift that cloud that hangs over her beautiful head, if only a little.
www.ipetitions.com/petition/campaign-for-kalydeco
sudaname · 01/11/2012 20:15
l imagine lots of Mumsnetters frantically searching family trees right now to check for the dreaded 'Great Aunt Sudaname, twice removed' - leaping off the page at them.
nocake · 01/11/2012 20:22
Drug companies aren't charities. It costs a lot of money to develop new drugs and they only have a limited amount of time before other companies are allowed to make the same drug so they have to recoup the development costs in that time. If the companies were forced to sell the drugs at dramatically reduced prices they wouldn't bother developing them in the first place. So, your petition could lead to fewer new drugs being available which means the next person's child won't get a treatment for his/her illness. Is that what you want?
Dozer · 01/11/2012 20:28
The MS Society and others campaigned hard to get NHS funding for beta-interferon (?) and in the end DH agreed to fund a large clinical trial, not sure what the trial results were. Do CF organisations have a campaign going?
morethanasong · 01/11/2012 20:42
Thank you for posting this. My baby ds has just been diagnosed :(
FantabulousFryingPan · 01/11/2012 20:44
Do you know anything about drug discovery, OP? The cost of drugs not only takes into account the cost of the discovery, development, manufacturing and clinical trials of the drug in question but also has to pay for all the research and development of drugs that don't make it to market.
There really isn't much ' nuh nuh ' going on.
FredFredGeorge · 01/11/2012 20:57
Well it's only relevant to 5% of CF patients and as people have said the cost of it was extensive, so unfortunately that means the reality is it will be expensive. 250 patients for 20 years will not bring in much money for the drug company I'm afraid.
And yes it's got to take some time to decide if it's economic to start the treatment, money is not unlimited.
merlottits · 01/11/2012 20:59
You sound completely ignorant to the complexities of what goes into a new drug. I know this is an emotive subject for you but there is no massive conspiracy.
It costs millions and millions to take drugs to trial and market them. Only then do drug companies get a return.
Often 'miracle' drugs are really not and someone has to decide whether they are affordable or not. My background is in Oncology - sometimes 'miracle' drugs only offer people a few more weeks to live yet many patients feel they are entitled to these drugs even if they cost tens of thousands of pounds.
The NHS cannot afford to give every new medicine to every person who needs it I'm afraid. Sometimes hard decisions need to made.
We should be glad that drug companies are not charities and are highly competitive. That's how they make real advances.
sudaname · 01/11/2012 21:00
Yes l know Fantabulous l know, but it's just how it feels sometimes and it just adds to the anxiety of having or a loved one having a disease/condition where time is of the essence.
I sometimes wish they just wouldnt tell you about these things but then again we live in a world of readily available information so l know that's not a solution.
Ahh l'm sorry morethan if it's any comfort there have been so many advances in its management in the last decade or so between even my dgd aged 12 and her cousin born 25 yrs ago that who knows ? by the time your little boy is 12 this latest drug and other new treatments will probably be available.
Thank you Dozer Will look on CF site and see not sure just literally seen this new drug.
HilaryClinton · 01/11/2012 21:05
This reply has been deleted
Message withdrawn at poster's request.
Dozer · 01/11/2012 21:11
An overly positive picture of drug companies is painted here. They are doing OK profit-wise!
It's the CF Trust campaigning on this, which is clinician-led and funds research, doubt they would be campaigning without considering the evidence.
I would much prefer public money to be spent on CF drugs - even if for "only 5%" () of patients - and indeed other drugs with potential to help a lot of seriously ill children than a lot of other NHS or government stuff.
FredFredGeorge · 01/11/2012 21:19
Dozer The 5% shows what a limited market is - and how much the cost of development needs to be offset against that market (the profits being what funds the research that helps the rest)
CFSKate · 01/11/2012 21:26
I saw this in the news on Monday about CF and Kalydeco
Cystic Fibrosis sufferer denied free drug
"Cystic Fibrosis sufferer is being refused a "chance of life" drug by the NHS despite the manufacturer offering it to her for free, it has emerged."
Is this the MS drug? Alemtuzumab The article seems to be saying that the price is going up because it works for MS? "Researchers at the University of Cambridge say a cancer drug, which wipes out and resets the immune system, has better results than other options. However, there is concern that a drugs company is about to increase the cost of the drug as a result."
onceisneverenough · 01/11/2012 21:28
I've signed. My 13 year old cousin has CF and I know what it's like watching them get so poorly knowing nothing can help them.
I have just searched the drug to read about it more, (according to the daily mail) and the drug company are already trialling a drug that will help 50% of CF patients.
RebeccaMumsnet · 02/11/2012 13:16
Hi all,
We have moved this to our shiny new petitions noticeboard now.
morethanasong · 19/12/2012 19:02
I'm not the op, but I wanted to come back and update - the drug has been funded in England! Thank you to everyone who signed the petition 
Let's hope that it will be funded in other countries, and that drugs for people with a different cf mutation can be developed www.cftrust.org.uk/pressoffice/pressofficepo/kalydeco_updates/kalydeco_eng_win
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