THE ROLE OF THE BRITISH GOVERNMENT in respect of VERTEX.

[Austen742]

Why was the company, Vertex, allowed to conduct Trials and access the facilities of the NHS, without this company being committed to share the benefits of success?
This NHS is OURS!
It does not belong exclusively to the Government of Britain or to any of the devolved governments. We have now got this ridiculous situation of having successfully trialled Phase 1 and 2 of the drug in Britain, and find that we CANNOT AFFORD TO PAY the cost demanded by Vertex.
Vertex have acted unethically, in breach of the Helsinki Declaration, having used UK Patients, many of them children, in trials, achieved success, manufactured the drug, taken it into the market place, brought it back to Britain, Kalydeco/Ivacaftor - the miracle drug! And it is!
Guess what?
IT IS SO EXPENSIVE - WE CAN'T AFFORD IT!

Why do we have to have stories like this in our press? http://www.dailymail.co.uk/health/a...refused-wonder-drug-save-life-NHS-chiefs.html

Article 19 of the Helsinki declaration ' research is only justified if there is a reasonable likelihood that the populations in which the research is carried out stand to benefit from the results of the research'.

The price of Kalydeco/Ivacaftor is out with the reach of 99.99% of the CF population in this country.
ARE THE NHS AWARE OF THIS ? and what do they propose to do?
What about the next round of trials by Vertex (recruiting now) - is there a safeguard in place?
These points need addressing.

 

[MaddiesFamily]

Just to add context (particularly for non-UK readers) a key committee is due to decide next week whether to recommend that Kalydeco should be available on the NHS in England. At list price, Kalydeco for the 270 eligible patients in England (one copy of the G551 mutation and aged 6 and over) would cost nearly £50,000,000 - which is about 45% of the entire NHS budget for CF. The UK government is strapped for cash and the NHS simply does not have £50M of loose change in its pockets. There are therefore real concerns that eligible CF patients in England will simply be denied access to Kalydeco because of a combination of the NHS's lack of funds and Vertex's determination to generate massive profits. As anyone who visits this forum will know, such an outcome would condemn those patients to needless suffering and avoidable (but irreversible) damage to their lungs and other organs.
Should the decision go "the wrong way", it would certainly raise an issue as to whether it would be ethical for UK patients to be enrolled into, or continue with, clinical trials involving Vertex products.

 

[CMC64]

Let's not forget the alleged means tested element for patients in the USA on low incomes who are provided with Kalydeco at no cost. This could be taken into account by Vertex when agreeing an acceptable costing with the NHS, as all G551D patients are eligible for health care under the NHS regardless of income. I'm sure that a lot of the UK patients would fall into the category for means test based on the alleged income threshold of what I understand to be $100,000 p.a. Apologies if I'm incorrect with this figure. My daughter would certainly fall into the category for free Kalydeco were she a US citizen. We need a quick resolution to this in the UK, Vertex have used us for their trials but have priced Kalydeco to be out of reach for almost all patients who would benefit in the UK, yet from other posts on this forum, it's apparent that Kalydeco is being prescribed for patients with mutations other than G551D in the USA.

 

[blue_eyes]

The problem with the NHS? They are viewing Kalydeco as an additional expenditure, not a revolutionary treatment.

The problem with Vertex? GREED, pure and simple GREED.

 

[CrisDopher]

The problem with the NHS? They are viewing Kalydeco as an additional expenditure, not a revolutionary treatment.

The problem with Vertex? GREED, pure and simple GREED.

Well, those are two very sweeping statements and, I think, unsupportable.

 

[cfsucks]

Well, those are two very sweeping statements and, I think, unsupportable.

disagree

edit:
kalydeco is a revolutionary treatment 100% no questions asked. the fact that kalydeco can cut down on other costs outside of healthcare is huge- like allowing a patient to go back to work and not be on disability, etc

it's 100% greed- they want profits now for their shareholders- not the long run- if it was more affordable more cf patients would be able to access it.

 

[blue_eyes]

Well, those are two very sweeping statements and, I think, unsupportable.

I welcome an alternative viewpoint, however, you fail to explain your thoughts – allow me to share mine (please do tell me if I am wrong on any points).

Vertex was given via venture philanthropy $75 million. They did not have to dig into their reserves to fund this research and development. Any financial layout made by Vertex has already been covered by profits from the US alone; share prices and subsequent profits support this. The manufacture of each Kalydeco pill is MINIMAL – single digits minimal. Vertex is offering Kalydeco FREE OF CHARGE to those in the US who do not earn enough (reportedly under $100,000).

To recap - No dipping into their own reserves to an excessive amount, rise in profits and share price, low manufacture costs and offering the drug to some people for free. None of these factors point to a company who are struggling financially, far from it. So it begs the question – why set such an exorbitant price in the UK. Sadly, the only conclusion I can make is greed.

The NHS and the specific commissioning bodies (notably the Clinical Priorities Advisory Group) are only taking into account trial data not case studies taken from those individuals who a) took part in the trial and who continued on Kalydeco and b) have started on Kalydeco due to a lung function of less than 40% (the compassionate programme). The NHS are expecting the £50 million (the amount it would cost to pay for the 270 people, over the age of 6, with the G551D mutation for a year) to be an additional cost. They are not considering fewer hospital admissions, fewer prophylactic medications, fewer medications to treat new infections and, hopefully, fewer medications to treat CF related complication such as faecal impaction.

One clinician has stated this medication (depending on pre-existing lung damage) has the potential to turn children with CF into non CF children and adults with CF into mild COPD patients.

I stand by my initial statements which as you can see are NOT unsupported in any way shape or form.

But everyone is entitled to their own opinion.

 

[CFSanDiego]

It takes over 800 million, probably more like 1 billion to bring a dry to market. Vertex invested a great deal of heir own money. Fortunately, or unfortunately, if there isn't a potential for profit, drug companies won't be incentivized to make them. Now that Pfizer, Glaxo and others have seen the potential windfall they are all aggressively working on the df508. I think as a result of this success of Kalydeco, we will all benefit a lot sooner.

 

[2005CFmom]

Vertex is offering Kalydeco FREE OF CHARGE to those in the US who do not earn enough (reportedly under $100,000).

This is to patients not covered by insurance. The vast majority of CF patients have private insurance or are covered under government sponsored plans. They are making money in the US AND helping people that are not insured (under insured). The problem with the UK is the single payer system. Someone has to pay for it before they can offer other "assistance" to others. Vertex and your health system need to work out a "fair price". Maybe pay 2/3 the current price. I get that figure because Vertex offers co-pay assistance in the US up to 1/3 the cost of the drug.

 

[MaddiesFamily]

It takes over 800 million, probably more like 1 billion to bring a dry to market. Vertex invested a great deal of their own money.

I do not think anyone disputes the entitlement of any pharma company to make a decent profit. I also think that everyone recognises that without such incentives innovative treatments such as Kalydeco will never see the light of day. There is, however, a big "how much?" question here. If it really cost $1Bn to get Kalydeco to market then Vertex should say so. I would personally be gobsmacked (not sure if that word travels across the Atlantic) if it were so, but am more than happy to be educated. But in the absence of such disclosure, it is inevitable that many will feel disquiet at the possibility that profiteering could result in patients being denied access to this amazing drug.

 

[Tsuper]

Of course, Vertex should make profits! Reasonable returns for money spent on development, manufacture etc. - no one denies that. There is a question though to be answered ...Why then was it that phase 1 and 2 of the trials were not conducted in the US? Perhaps Vertex, in common with many other Pharmaceutical companies, conduct research in the UK because it is so much less expensive. They have access to excellent centres, facilities and staff with expertise and at a fraction of the cost. Unfortunately, the savings made in the reduced costs of Trials does not translate into equivalent pricing of the the drug, Kalydeco, in Britain. The price being quoted is £182000 per annum, per patient! So our CF Patients with G551d mutation continue to suffer organ damage, some will undoubtedly lose their lives, while we wait to see if Vertex will set the price for Britain at a realistic cost, reflecting the fact that the clinical trials took place in this country. In time, it is my belief that this drug will be refined, changed, to treat other conditions. - already trials are under way on other mutations. Need not greed, Vertex!

 

[PeggySue]

VERTEX, KALYDECO / IVACAFTOR, all synonymous, and in our thoughts constantly. We need

We need Answers....What IS the price? If it is as reported, why so high?
We are totally agreed that Vertex have to make a profit. We know that Charitable groups in the USA, invested millions of dollars in the research. Some might have loaned, rather than invested? Do the CF Foundation or Bill Gates Charity (2 of those known) realise that the high price being asked for in UK and Ireland, is causing such a delay in funding? Resulting in a delay in prescribing, causing even more damage to the organs of our G551Ds, losing them days of precious life?
Should we make these philanthropic charities aware of the situation, in the hope that they might bring some pressure to bear on Vertex?
Ivacaftor is hailed as the miracle drug. It has gained Vertex the accolade of winning the Wall Street Journal's 2012 Award, in the past week. Commendable? Yes! Share price increase? The profits will follow.
All the accolades in the world are meaningless while every day, Vertex, with their extortionate price, deny access to Kalydeco for those sufferers who deperately need it!

 

[MaddiesFamily]

We need Answers....What IS the price? If it is as reported, why so high?
... Do the CF Foundation ... realise that the high price being asked for in UK and Ireland, is causing such a delay in funding? Resulting in a delay in prescribing, causing even more damage to the organs of our G551Ds, losing them days of precious life?
Should we make these philanthropic charities aware of the situation, in the hope that they might bring some pressure to bear on Vertex?

Err: according to its financial statements, in May this year the CFF commuted/sold part of its future royalty revenue in Kalydeco to Vertex for $150,000,000 (see page 16). The financial statements do not reveal the value/extent of the CFF's surviving right to royalties.
So, PeggySue, I suspect that the CFF does know about the high price being demanded by Vertex. This may also help explain (at least partly)why the price is so high, with the resulting possibility that some eligible patients might not get access to this important drug. Obviously royalties for IP rights consequent upon funding can be a valuable source of income for charities, and can be ploughed back into research for similarly radical treatments which might benefit the other 95-96% of those with CF. But I am sure everyone (the CFF included) would be disappointed if the level of those royalties was a factor in some patients being denied access to Kalydeco.
What difficult issues ...