Thursday, 13 December 2012

CF Gene Therapy: Small Bet/High Payout

The Cystic Fibrosis Foundation in the US does not provide any funding for the Cystic Fibrosis gene therapy programme based here in the UK (run by the UK Cystic Fibrosis Gene Therapy Consortium “UKCFGTC”).

The UKCFGTC is an innovative form of collaboration. Established in 2001 by Rosie Barnes, then CEO of the CF Trust, it brings together over 50 scientists from Edinburgh University (Centre for Molecular Medicine and Roslin Institute), Oxford University (Gene Medicine Group) and Imperial College London (Department of Gene Therapy, National Heart & Lung Institute).

The single-dose trial of the first generation (lipid-based) product was completed last year. It demonstrated safety and showed very promising results with CFTR protein expression better than VX-770 at the same stage (according to Prof. Eric Alton of Imperial College). 

Multi-dose clinical trials of the first generation product are now under way at the Royal Brompton Hospital in London and at the Western General Hospital and Royal Hospital for Sick Children in Edinburgh. These are due to complete in mid 2014. 

Since it was created in 2001, the programme has been funded to the tune of £34.3m, of which the CF Trust has provided £30m. This may sound like a lot of money but it equates to £3m per annum and this is very cheap compared to most pharmaceutical R&D projects. The hard reality is the gene therapy progamme is run on a shoestring and it relies on the incredible commitment and determination of a team of very smart scientists. It very nearly collapsed at the beginning of this year but the government did step in at the eleventh hour and, through the MRC and NIHR, provided £3.0m needed to get to the next stage with the first generation product and a further £1.3m to progress work on the second generation (lentivirus-based) product. This money was provided as a grant.

Hopefully when the results of the multi-dose clinical trial are available, the results will justify the next stage of work and the UKCFGTC will be able to raise the capital necessary to get there. The programme continues to be supported by the CF Trust and Rosie Barnes, through her new charity, Just Gene Therapy. The problem is neither of these groups has sufficient resources to fully fund the UKCFGTC. It is too early-stage for Big Pharma and so it is going to need a well-funded new partner to bridge the gap. This could be the CFF (or another "venture philanthropist") or a biotech company with a strategic interest and infrastructure that the UKCFGTC can leverage to get the product to market faster than it could do on its own. Ideally a new partner would be a combination of CFF/venture philanthropist and biotech.

The science in this programme is cutting-edge and brings together the work of specialist teams in a number of global centres as well as the three principal UK teams. The consortium is not competing against other groups; all the leading scientists in the world are working together and it is therefore the only horse in this particular race. The IP situation is complicated but it has been well thought through and does not represent a barrier to third party investment.

The discoveries and tools coming out of the programme may well be applicable to other "autosomal recessive disorders" like Cystic Fibrosis. These are inherited disorders where an affected person usually has unaffected parents who each carry a single copy of the mutated gene (and are referred to as carriers). Two unaffected people who each carry one copy of the mutated gene have a 25% chance with each pregnancy of having a child affected by the disorder. Other examples of this type of disorder are Tay-Sachs disease, sickle-cell disease, Niemann-Pick disease, spinal muscular atrophy and Roberts syndrome.

Unlike the mutation-specific small molecule drugs being developed by Vertex, Pfizer and others with the backing of the CFF - representing a total funding commitment by the CFF of $133m to date - the CF gene therapy treatment being trialled by the UKCFGTC substitutes correct DNA for faulty DNA, whatever the mutation. So it is a single product with the potential to treat the whole CF population as compared to the small molecule candidates which treat sub-populations and therefore have limited (sometimes “ultra-orphan”) markets.

It has taken much longer to reach this point than it would have done with a more appropriate level of funding. But through the sheer talent and tenacity of the people involved, the UKCFGTC has now got to a point where it has demonstrated the exciting potential of the science. A relatively small financial bet, at this point, would materially accelerate the programme offering any new partner the potential of a high return in regular financial terms and also in terms of world-changing impact with a fat moral dividend attached. There are no guarantees of course; it may or may not prove successful in terms of translation into a new medicine but, if not, at least we will be able to eliminate it quickly and focus on other areas. It does look highly promising though.

It may have been a better use of CFF resources to put at least some of the $58m it gave to Pfizer into other projects such as the gene therapy programme where its money would certainly not be displacing that of Big Pharma and where it would have  a transformational impact on the project.     

You can read all about the UKCFGTC here:

Click here: UK Cystic Fibrosis Gene Therapy Consortium

Kalydecogate (Part II): A Global CF Foundation?

This follows on from my post on 1 December 2012 in response to the difficulties CF patients in the UK and other non-US markets have had in accessing the new drug Kalydeco which was developed by Vertex Pharmaceuticals with financial backing from the CF Foundation: Click here to read "Kalydecogate (Part I)"

The Cystic Fibrosis Foundation (“CFF”) has total annual revenue of over $300m. It has total assets of $250m, including an investment portfolio of $160m and cash equivalents of $40m. By comparison, the UK’s CF Trust is far smaller with c. £9m in annual revenue and total assets of c. £10m. 

In the UK, the CF Trust has 9,000 patients to serve so that's $1,600 per pw/CF.

In the US, the CFF has 30,000 patients to serve so that's $10,000 per pw/CF.

Clearly this is not comparing apples with apples as the two countries have very different healthcare systems but it does point to the relative clout of the CFF and its absolute size is important in a world where new treatments are so expensive to develop. The CFF has a great deal of firepower in an ecosystem with 70,000 patients globally (less than the capacity of Old Trafford). It is essentially the global voice of people with CF when it comes to speaking to the pharmaceutical industry. It has certainly made some amazing things happen and helped CF life-expectancies rise steadily over the last 50 years or so.

The CFF has an innovative model that is now being used by other foundations as a template to facilitate the development of treatments for other diseases. But is the CFF model the most efficient one with respect to the collective interest of CF patients in the US? How about CF patients globally? 

Pharmacy Services

In the most recent CFF financial statements, CF Services Inc. (“CFS”) the specialist CF pharmacy arm of the CFF is the Foundation's largest source of income (approx. $170m last year). In addition to filling prescriptions, CFS also provides advice and assistance on re-imbursement issues for patients. The CFF says this means it can make sure people with CF get what they need at reasonable prices but it may also mean their interests are not always aligned with those of patients. It has an interest in high sales, albeit one which it must balance against other interests. Does the CFS give retail preference to drugs and treatments in which the CFF has a royalty interest over purely third party products? 

On 7 December 2012, CFS joined forces with Walgreens, the largest drug retailer in the US. Since the insurance company payers often dictate which pharmacy a patient must use for their prescriptions, patients may feel they are subject to a form of monopoly.  The CFF must have some fascinating and very complicated discussions with payers in relation to new drugs like Kalydeco where it has a royalty entitlement. They must negotiate the price alongside the pharmaceutical partner and then also try to persuade the payers to use their pharmacy services in preference to other independent pharmacy service providers. They have a lot of different interests here but it is difficult to see how this arrangement gives the CFF any negotiating power or, indeed, any incentive to keep prices down. Clearly Walgreens have no philanthropic duty towards CF patients. This complex web of relationships may or may not be in the interests of patients in the US. It is important because these pricing discussions have significant consequences for people with CF all over the world. 

Given its other roles, it looks like the CFF's role as a provider of specialst CF pharmacy services might create a conflict of interest. 

Collaborative Drug Discovery & Development

Cystic Fibrosis Foundation Therapeutics, Inc. (“CFFT”) is the CFF’s non-profit discovery and development affiliate. It provides capital to pharma/biotech companies and also access to the national patient data/genotype database and assistance in organising clinical trials and recruiting volunteers through the network of specialist CF centres. It would very difficult and perhaps impossible for a drug company to try to develop a new CF drug without the support of the CFF acting through CFFT.

Clearly some form of collaboration makes a great deal of sense. The CFF has a body of knowledge and infrastructure in place and it is far more efficient for the drug company to “rent” these things than try to build them from scratch. The economics of orphan drug development are bad enough without taking on unnecessary costs.

If the collective goal is to bring effective new disease-modifying and symptomatic treatments to market as quickly as possible, there must also be a role for the CFF to make sure different drug companies are not deploying resources on the same work, to encourage the timely and efficient sharing of information and to facilitate the development of “general purpose” tools and platforms that can be accessed centrally. There must be a role in overseeing a highly-targeted global research programme - linking with centres of excellence around the world – coupled with pro-active global engagement with scientists, drug companies, politicians, government officials, clinical trial participants, patients, donors and other stakeholders. 

With each new drug costing approximately £1 billion to bring to market, the major challenge is for the pharmaceutical industry to find ways to design experiments so things that don’t work are identified and eliminated far more quickly and far more cheaply. This problem is particularly acute for rare or orphan diseases like CF and the new collaborative models are helping to address that.  

Science is a series of failures, need to fail fast and get them out the way.”
Michael J. Fox, speaking at the 2012 Forbes Healthcare Summit

Autism Speaks has followed the CFF’s model and recently set up a non-profit affiliate called DELSIA to collaborate with pharmaceutical companies and other for-profit players to move new scientific discoveries from the lab into the hands of patients in need. The Crohn’s and Colitis Foundation of Canada is doing something similar as is Scott Johnson with his incredible Myelin Repair Foundation. The Michael J. Fox Foundation for Parkinson’s Research has developed a remarkable model to develop new treatments for PD. This is a brilliant role model for others. It has leveraged global scientific resources and re-configured the PD community to achieve significant impacts and change the paradigm with a laser focus on translating scientific breakthroughs into treatments available to patients. Michael J. Fox has shown that sometimes it is not the science that limits the treatment options for a given disease but the development model. The so-called “action-tank” FasterCures, backed by the Milken Institute, is acting as an effective and open knowledge-sharing platform and a catalyst to help spread these new collaborative models to other under-treated diseases. 

So, in principle, collaboration makes sense in the context of many diseases. However, the practical details of these emerging collaborations are less intuitive.

Collaborative Models: Practical Question No.1

What kind of work should the foundations fund? There are two aspects to this. First, they should only fund work that is important. That is, they should have an overall scientific strategy (and ideally some notion of a critical path) and they should only enable work that is progressive in the context of that strategy. Second, it is axiomatic that they should not simply fund work that the market was going to fund in any event. They should only fund projects that are too experimental or early-stage for the pharmaceutical industry to do by themselves. They can create the maximum impact by bridging this gap (making sure they always know how big the gap is and don’t end up building half a bridge) and then making it a priority to move projects forward to the stage where pharmaceutical companies can take them on and release the foundation to focus financial resources on other early stage projects.

In this way, the foundations can expand the pool of available capital to fund a greater universe of projects rather than simply change the nature of the capital going into a given universe of projects. Foundation capital should complement not substitute for private capital. These points have been well made and developed very effectively by John LaMattina on his blog at

There may also be a “moral hazard” point here though. If a pharmaceutical company feels it can get a foundation to fund their research at a cost of capital which is lower than the market would charge or its own internal rate, then it may reconsider its commitments more generally. For example, if a pharma receives money from a foundation for a project it was planning to fund itself, then the pharma may well look at planned spending on other projects and seek to tap the same or a different foundation for those projects too. So if they are not careful the foundations as a whole may see a negative compounding effect i.e. they may find a lot more gaps appearing in places they do not expect. Big Pharma will treat them like suckers if they act like suckers!      

Collaborative Models: Practical Question No.2

How should the deals be structured? Specifically, what should the foundations receive in return for providing these resources to the drug companies? Obviously one kind of “return” would simply be effective outcomes for patients i.e. the foundation might try to establish precisely-defined milestones and controls to ensure each dollar spent is tied to an effective step forward on the path to an effective overall outcome. In addition, foundations may seek to build in pricing guarantees or other relevant commitments from the partner.

While much of the CFFT transaction detail is not disclosed, one can comment on those aspects which are disclosed. The CFFT’s adopted model includes negotiating royalties from its pharma/biotech partners on drug sales in return for its commitment of capital and other resources. The principle is CFFT receives these royalties and recycles them into more R&D for CF especially targeting the most common mutation delta F508 (c.80% of CF cases). This looks like a great model for people with CF. The two most notable examples to date of deals based on this model are the $75m to Vertex and the $58m to Pfizer both of which are aimed at developing new genotype-specific small molecule treatments such as Kalydeco (formerly known as VX-770) and principally targeting delta F508.

It stands to reason that the CFF's royalty position may influence its attitude to pricing. Since it stands to make more money the higher the price, the CFF may push for prices higher than it otherwise would. Clearly the prices of new orphan drugs have to be relatively high as the cost of development is largely the same irrespective of the number of patients with the disease. This cold equation may well mean the difference between a completely new kind of drug being put in the hands of patients in need, or not.

With the CFF and the relevant pharma/biotech partner having an interest in high prices and the US insurance companies tending not to push back too hard, the result is of course high list prices. The point about the CFF royalty is that it may lead to prices that are higher than they would be if the CFF did not have a royalty since, with no royalty rights, it might naturally seek to negotiate prices down (in order to maximise the number of people who can benefit) or procure commitments for future R&D work in return for its financial support and access to its unique IP and infrastructure.   

On the one hand, the adopted model means relatively large royalties to CFF (through CFFT) which should help to speed the development of other treatments. At least most patients in the US who would benefit will get the drug as the health system is relatively well-funded. On the other, it means people with CF in other countries with less well-funded health services may not be able to get the new drug. Around 60% of people currently diagnosed with CF live outside the US so it is an important consideration.

As has been well documented elsewhere, Kalydeco is a transformative treatment that is still not generally available to eligible patients with the G551D mutation in the United Kingdom, the Republic of Ireland, Canada and Australia. In the UK, the NHS is resisting paying the $294,000 per patient per annum list price on the basis of cost-effectiveness. This is an on-going travesty causing unnecessary suffering to many young people and one can only hope it is resolved without further delay and that procedures are changed to ensure nothing like it happens again.

One can argue that health services in these countries ought simply to pay the price asked and that the problem is not limited resources but misallocation of resources in systems that fully-fund certain elective treatments or treatments with only marginal, or even unproven effects, while not prioritising highly effective treatments that only impact on a small number of people. It is a political issue but also an issue of the effective management of resources. It seems painfully obvious that health services should spend less on things that don’t work and more on things that do. Others such as David Grainger on his DrugBaron blog and @sciencescanner Twitter feed have argued the case for limiting the scope of services offered by the NHS and I referred to this in my Kalydeco post. Ben Goldacre continues to eviscerate the powers that be in his relentless evidence-based campaign based on in his excellent book (“Bad Pharma”) and his accompanying multi-media assault. The corollary, of course, is more Good Pharma such as Kalydeco. These kinds of changes will have to happen but they will take a long time. It is not academic. It is time many people with CF and other rare diseases simply do not have.   

Be that as it may, it does firmly underline the need for people with rare diseases to organise themselves more effectively and get their message across to the right people at the right time with more effective lobbying.

It also remains valid to ask whether the CFF/Pharma/Payer “complex” is efficient in terms of the collective interest of the global CF community or whether it may lead to prices higher than they need be and a lower portion of eligible patients treated.

Need for a Global Foundation

Perhaps this points to the need for a new global foundation to act in the interests of everyone with CF regardless of where they happen to live. In addition to the points about new drug pricing and deal structures, a global foundation would have greater political clout and may help to address the lobbying issue when it comes to re-imbursement. 

On a more technical note, it is unhelpful that North American standards of diagnosis for CF are slightly different from those in Europe.

This kind of misalignment is not conducive to the efficient sharing of research in the common pursuit of a cure. A global foundation could address some of these technical issues and otherwise facilitate the efficient discovery and development of new drugs and treatments.

The limiting condition for rare diseases should be the pace of science and not organisational friction or the geographic location of capital. 

Cure the model. Cure the disease.

Related Links:

Monday, 10 December 2012


This day is sad but I will strive,
to channel the feelings burning inside,
into some good things, she will select;
And through her spirit she'll protect,
So when I do, do these good things,
her graceful echo transmits and sings;
And through my acts she'll resonate,
in this world and in my fate.

I am depleted but not defeated,
Now through my acts I'll be completed;
There'll be rocks on which I stumble;
But I bounce right back no time to tumble;
She'll drive me on to greater heights,
Give me the strength to win my fights.

I’m a lightning rod in a barrage of bolts,
Galvanised by the stream of volts;
I'll take this chance to change the pace,
and make the world a better place;
Instinct says survive and thrive,
I'll use this force myself to drive.

I'll say what I mean and mean what I say,
Do deeds that matter, and count every day;
Bites of life I'll sweetly savour,
In all its texture, all its flavour;
I'll paint pictures like Matisse,
I'll make my life a masterpiece;
Keep things simple every day,
Eat, sleep, love and maybe pray;
I'll care for me and the ones I love,
I'll nail this mission from above.

Through such things I'll be alive,
But first I pause so to revive;
Til good and ready, I will rest,
then steel myself for this great test.

That is all, I must now sleep;
May the sunrise remind me this promise to keep.

Saturday, 1 December 2012

Kalydecogate (Part I)

As I sit in the Cystic Fibrosis ward of my hospital doing my IV antibiotics this morning, I feel increasingly angry about the on-going inability of the Government and Vertex Pharmaceuticals, Inc. to get Kalydeco into the hands of patients who desperately need it. This drug works, it is cheap to make and it should not be this difficult.

Kalydeco, Vertex's new drug for Cystic Fibrosis, was approved by regulators in the EU in July 2012. While treatments to date have sought to control symptoms, Kalydeco is able to target the underlying cause of the disease. It is not gene therapy but it does correct the action of the defective CFTR protein which is what causes the symptoms. Kalydeco is a kind of “small molecule” drug; these are small enough to get into the right parts of cells and then bind to and correct specific proteins. For patients, it means significantly improved lung function, weight gain and improved sweat chloride levels. It would be difficult for a physician to make a CF diagnosis on a patient taking Kalydeco without doing a DNA test.

Unfortunately Kalydeco only works on people with a certain mutation of CF; people with at least one copy of the G551D gene. This is approximately 4% of the 70,000 people in the world who have been diagnosed with CF. For these people, it has the potential to transform their daily lives and give them a fundamentally different future. In some cases, it will save their lives. 

The Cystic Fibrosis Foundation in the US has provided $75m to back the “small molecule” drug development programme which produced Kalydeco and this on-going programme may well lead to similar drugs for the other 1,500+ CF mutations identified to date including delta F508 which accounts for approximately 80% of the global CF population.

If you want to hear some of the personal accounts of the impact Kalydeco can have, follow these quick links:

Vertex is currently seeking to sell Kalydeco fully into the major EU markets but finding that “payers” or reimbursement authorities such as the NHS in the UK are resisting the $294,000 per patient per annum list price. The drug companies set the list price based on what they know the US health insurance companies will pay. The problem is that this sets the bar at a level which is not necessarily appropriate for other markets.

Patients at various centres in the UK took part in Vertex’s clinical trial programme earlier in 2012. I am aware of one case at my centre where a young patient with G551D who is very poorly and ineligible for a lung transplant (due to the particular bugs colonizing his lungs) took part in the trial and saw some big improvements. At the end of the trial, the drug was withdrawn and now he is going downhill again since nothing else comes close to Kalydeco in treating his CF. Despite Vertex’s “compassionate use policy”, there are other such stories. I was not aware that a drug company could withdraw treatment in this way after clinical trials. I had naively assumed that if a patient took the risk of participating in a clinical trial (partly to help a drug company prove the value of a new drug) and it worked, the drug company would have a clear ethical obligation to continue supply post-trial. It raises some major ethical questions.   

Discussions with reimbursement authorities are on-going, including with the NHS Clinical Priorities Advisory Group (CPAG), a group established to provide reimbursement advice to the four specialist commissioning groups (SCGs) in England. These SCGs will make the final decision on whether the drug should be funded in the UK. The result of the last CPAG meeting on 22 October was a request for further analysis of the cost-effectiveness of Kalydeco.

Following completion of that analysis, more discussions and negotiations have taken place between NHS representatives and Vertex. A final decision had been expected to be announced by mid-December but this week we learned that it has now been delayed until January 2013. The implication is that Vertex is being urged to come back to the negotiating table offering Kalydeco to the NHS at a reduced price and that the additional time will give both parties a chance to reach an agreement.

It is bad enough to see many young lives being put at risk, and in some cases deteriorating with each day that passes, when there is an effective treatment available. However, a look into Vertex as a company puts an even more tawdry complexion on things. 

Vertex is quoted on NASDAQ and has a market capitalisation of $9 billion. The company recently announced that it had overstated May 2012 results from clinical trials involving a combination therapy (Kalydeco combined with another “small molecule” known as VX-809) aimed at people with the delta F508 mutation of CF.

Before the company released the original data in May 2012, the share price was just over $37. This announcement showed significant improvements in lung function among adults with delta F508. It claimed that 46% of patients experienced at least 5% absolute improvement in lung function (FEV1) from baseline and that 30% of patients experienced at least 10% absolute improvement. The share price jumped 55% on that day closing at $58.

A couple of days later the share price reached $66 and Vertex executives (namely Chief Commercial Officer Nancy Wysenski, Chief Scientific Officer Peter Mueller, and Senior Vice Presidents Lisa Kelly-Croswell and Amit Sachdev) sold shares valued at over $20 million. They sold more shares in the next weeks and cleared a total of $38m between them. Nothing irregular so far.

Then on May 29, Vertex announced “amended” results from the Kalydeco/VX-809 combination trial for delta F508. The amended release stated 35% of patients (down from 46%) experienced at least 5% absolute improvement in lung function and 19% of patients (down from 30%) experienced at least 10% absolute improvement.

The amended trial results were released the same week the Food and Drug Administration highlighted violations by Vertex relating to an advertisement that was deemed “misleading because it overstates the efficacy, omits material facts, and minimizes important risk information about the drug product.”

About a week later, Vertex announced the retirement of 54 year old Chief Commercial Officer, Nancy Wysenski. Of the $38m proceeds from the mid-May sale of shares by executives, Nancy Wysenski took away $22m. In the announcement the company said “after more than 30 years in health care, she has chosen to move on to the next chapter of her life, and we wish her all the best in retirement.” 54 is obviously a relatively early retirement age.

Around this time, Senator Chuck Grassley approached the US Securities and Exchange Commission asking them to look into the executives’ share sales. By selling when they did the executives made about $13m more than they would have done had they sold after the amended announcement.

Since then the share price has fallen back to $40 on the back of questions over this episode as well as weak trading results and important progress made by competitors such as Gilead in the company’s other main market, Hepatitis C.

Several groups in the US have now commenced litigation in relation to the way trial data was announced and insider share sales.

Perhaps the Cystic Fibrosis Foundation has realised that the shit is about to hit the fan. Last week the CFF announced a commitment of $58 million to support Pfizer’s small molecule research programme with a particular focus on finding a drug candidate for delta F508.  

As John LaMattina highlights (Should the Cystic Fibrosis Foundation Invest $58 million in Pfizer Research?, 28 November 2012, big pharmaceutical companies have traditionally avoided research and development for rare diseases but, with recent commercial successes, that has started to change. Pfizer underlined its new commitment to rare diseases with the 2010 acquisition of FoldRx, a small company focused on developing treatments for diseases such as CF caused by the improper folding of proteins. In acquiring FoldRx, Pfizer inherited its partnership with the CFF to find drug candidates for CF. When Pfizer decided to take this step, without any commitment of additional funds by the CFF, it must have made an assessment of the commercial potential and signed off on the investment that would be required to work the FoldRx portfolio. As LaMattina points out, it is therefore surprising that the CFF has now agreed to fund the next stage of the FoldRx work to find new CF drug candidates. Surely, Pfizer would have done this research anyway without the CFF funding. It raises questions over the deployment of the CFF’s funds.

As LaMattina says “by requiring the CFF to pay for it, Pfizer is diverting precious research funds that the CFF could be using to fund numerous other programs in universities, research institutes, and small start-up companies. In the life sciences eco-system, $58 million can go a long way. Should the world’s largest pharmaceutical company have its hand out to the CFF? Couldn’t Pfizer have found the less than $10 million/year needed to fund this research?”

So what is going on here? Vertex is a tiny company compared to Pfizer and it has nothing like the same level of resources. Kalydeco would surely not exist if the CFF had not provided financial backing. However, the Pfizer deal is very different and doesn’t seem to add up. It looks like the CFF are providing capital that Pfizer were prepared to invest themselves. While the CFF typically seeks to leverage off private capital to enhance impacts, it looks here as though the CFF capital is simply allowing Pfizer to avoid using its own capital. Perhaps the CFF are so concerned about all the issues at Vertex that they felt the need to back, and be seen to back, another horse. That way, when Kalydecogate breaks things will not look as bad as they might have done if all CFF's eggs had still been in the Vertex basket.

Whatever the outcome of the Vertex litigation and any longer term fall-out for the company, the $38 million received by Nancy Wysenski and the other three Vertex executives from their well-timed share sales, would pay for 130 kids to get Kalydeco for a year. If these shares had been sold at $40 (i.e. the market price prior to the “later-amended” announcement of the clinical data) rather than the elevated price, the executives would have cleared only about $25 million. The extra $13 million they did in fact receive, arguably made possible by the “later-amended” announcement, would pay for 43 kids to get Kalydeco for a year.

Some of these kids are going to die without Kalydeco and every day that passes without action puts them in greater jeopardy.   

Call to Action:

Read why leading UK medical scientist David Grainger and thousands of others have signed the petition to get the NHS to agree a deal with Vertex and, while you are there, sign it yourself.

Read & Sign: Campaign for Kalydeco Petition - Comments by David Grainger

David Grainger is a Venture Partner at Index Ventures; former Director of the Translational Research Unit of the Papworth Hospital NHS Foundation Trust; and former Principal Investigator in the Department of Medicine at Cambridge University. He is also part of TCP Innovations Ltd, a leading life sciences consultancy; founder of a number biotech companies; and author of the widely-read DrugBaron blog on pharmaceutical and biotech industry news and @sciencescanner twitter feed.

Click here to read more of David's views on Kalydeco and pricing of new drugs for orphan diseases

Please also read my follow-up post on the lessons we can learn from Kaydeco and the bigger picture:

Spit It Out!: The Case for a Global CF Foundation?

Should the Cystic Fibrosis Foundation Invest $58M in Pfizer Research?, John LaMattina,, 28 Nov 2012