Vertex: Not So 'NICE' After All?
Written by Marc Cotterill
Introduction
In a recent article titled ‘Worry: A Guide for the Cystic Fibrosis Community’, I wrote the following message of hope for new parents:
If there was ever a point in history to live with CF, or to have a child born with the condition, that time is now. Today, we have unmatched levels of understanding, treatments, and community support that far outweigh what my parents were ever privy to back in 1982 when I was born. For that reason alone, we have much to be thankful for…
The sentiment was largely based on the relatively recent introduction of CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapies that have—in many people’s eyes—changed the game for those living with CF.
And given the majority of people who consume my content are from the UK, it was delivered with confidence in the knowledge that we are part of the lucky few who can gain access to these ‘wonder drugs’ at almost no cost to ourselves.
Unfortunately, that confidence was rocked this week, following a post released on Instagram by the Cystic Fibrosis Trust (CF Trust), a UK-based charity dedicated to fighting for the rights and interests of individuals with CF:
But what does it mean?
Here are some of the key facts:
NICE is the authoritative body in the UK that decides which new medicines and treatments the NHS should fund.
NICE makes its decisions by evaluating the impact of treatments on the patient's health and quality of life, alongside the cost it will incur to deliver them.
This approach helps in determining whether the investment in a particular treatment is justified, ensuring that the NHS funds are allocated towards treatments that provide good value for the amount of money spent.
On 3rd November, NICE released a preliminary recommendation not to fund the key Cystic Fibrosis treatments Ivacaftor–Tezacaftor–Elexacaftor (aka Trikafta), Tezacaftor–Ivacaftor (aka Symdeko/Symkevi), and Lumacaftor–Ivacaftor (aka Orkambi) on the NHS, due to concerns about their cost compared to the benefits they provide.
It is important to note that this decision is not final yet and it may change after further deliberation.
However, if the decision does become final, it could affect the accessibility of these treatments through the NHS for young children with CF who might need them in the future.
Patients already on treatment or those who commence treatment before a final decision from NICE will continue to receive funding. This is confirmed.
It is at present unclear if a negative decision from NICE would prevent kids on earlier modulators (for example, Orkambi) from ‘upgrading’ to Trikafta when they reach the eligible age. [UPDATE 08/11/23: it is now confirmed that children on earlier modulators can move through the portfolio of modulators: find out more here.]
Based on all of the above, our collective concern within the CF community is with future generations who might not have access if a negative decision is reached before they need treatment.
I have provided reference links at the end of this article.
What happens next?
In light of the draft recommendation by NICE, the process now enters a further consultation phase. Here are the next steps outlined:
Further Consultation: NICE is engaging in additional consultations as the current recommendations are in the 'Draft' stage. They are open to revisions, but that would only be based on more ‘comprehensive analyses’, which were not presented earlier.
Community Input: Families and others in the community can help by sharing insights based on their own experiences:
Treatment-specific utility benefit:
This term refers to the unique benefits a particular treatment offers to patients. In a healthcare evaluation context, it's about identifying the specific advantages a treatment has over others or no treatment, which could include faster response to treatment, fewer side effects, or other unique benefits.
Providing data and personal experiences on how modulators significantly benefit people with CF, could aid in the cost-benefit analysis. It is worth noting that anecdotal evidence will likely not be enough here, data-driven evidence is what would make the difference.
Severity modifier:
NICE has a "severity modifier" that increases the value of QALYs (Quality-Adjusted Life Years) provided by medicine for patients with severe diseases. This means that NICE is prepared to pay more for a medicine that treats patients with severe diseases, allowing for a higher cost-effectiveness threshold for these treatments.
The ‘severity modifier’ considers how severe CF is, therefore sharing information that could help in adjusting analyses based on the severity of CF could influence NICE's willingness to pay more for modulators, thereby allowing for a higher cost-effectiveness threshold for these treatments.
Extending the carer impact with EQ-5D surveys:
Another angle is the impact CF has on caregivers (aka carer impact). It is an important aspect of evaluating the broader effects of the disease and treatments. EQ-5D surveys are standardised tools used to measure health-related quality of life, which can be extended to measure the impact on caregivers too. By supporting further research through EQ-5D surveys, more comprehensive data on caregiver impact can be gathered, potentially influencing NICE's evaluation.
Technical Arguments: Families or individuals familiar with NICE methodology are encouraged to form technical comments to refine the recommendations. Collaborations are underway between CF Voices and Quest for a CF Cure in Scotland to consult with NICE on effectively making technical arguments. I will link the associated Facebook groups below for you to follow.
Deadline: The deadline for this process is November 24th, providing some time to formulate impactful arguments to influence the final recommendation.
For a detailed understanding, I will link to the full guidance below.
Root cause: Vertex Pharmaceuticals
Vertex Pharmaceuticals’ values underscore its mission and operational ethos; fearlessness, diversity, collaboration, commitment to patients, excellence, innovation, and integrity (links below).
The company states that these values drive its innovation process and corporate responsibility efforts. But there is a problem, because the pricing of their CF treatments seems to challenge these values.
It’s stated that Vertex has already made over $35 billion in global sales of these drugs, which—based on a recent presentation by Jonathon Guo at the North American CF Conference 2023—is said to be far more than their costs of research (total R&D expenditure since 2001: $12 billion) and production (estimated minimum cost of production is $5,700 - links below).
Last year, Vertex spent $3 billion buying back their shares. There are a few reasons why this is important to note:
Shareholders First: Share buybacks are a way for companies to return capital to shareholders, and they often signal that a company believes its shares are undervalued.
Financial Priorities: It showcases the financial priorities of Vertex. While it has the financial capacity to spend a substantial amount on share buybacks, the pricing of its CF drugs remains exorbitantly high, which has led to significant global accessibility issues.
Corporate Responsibility: It raises questions about Vertex's corporate responsibility. The high cost of CF drugs versus the company's willingness to spend on share buybacks may be seen as a lack of commitment to making treatments affordable and accessible.
Public Perception: Such financial decisions undoubtedly affect public perception, potentially seen as prioritising shareholder value over patient access to life-improving medications.
Ultimately, these practices highlight the stark contrast to the values Vertex claims to uphold. They stand behind a message of ‘innovation’ and ‘corporate responsibility’, but their financial decisions seem to tell a completely different story.
It is a landscape of conflicting interests, where the pursuit of profit overshadows the altruistic goal of enhancing patient wellbeing. It raises ethical questions about the responsibility of Vertex, a pharmaceutical giant, in balancing its commercial interests with the larger societal good, especially when they have already amassed significant revenue from a community grappling with a life-threatening condition.
Vertex and its shareholders have profited immensely from the CF community—a community that now calls for a balance between the company's profit-driven incentives and its moral obligations to provide affordable, accessible treatments.
The plot thickens as we review some of the key facts.
Key facts
Over 10,000 people are living with CF in the UK.
Vertex was demanding a price of £100,000 per person per year for their treatments.
If the NHS accepted this price, this would mean a £1 billion cost to treat everyone with CF, per year, for life. The entire budget for drugs in the UK is around £16 billion per year.
If the decision from NICE is that they choose not to fund the drugs, newly diagnosed patients may not be able to access a drug which could increase their life expectancy by 40 years.
Between 2015 and 2019, Vertex refused to lower the prices for their treatments for Cystic Fibrosis. During this time, hundreds of children in the UK died without access to effective treatment.
Around the same time, Vertex destroyed stocks of CF drugs for the UK, after the NHS refused to agree prices back in 2019. Sarah Boseley covered this in the Guardian (link below).
Just before the last elections in 2019, NHS agreed on a confidential deal with Vertex to allow access to the treatments, which was adopted across the UK, however, this did not involve any assessment of value through NICE at the time (link below).
Now that NICE have fully evaluated the drugs they are questioning why the NHS should continue to pay high prices (although a flexible commercial agreement assures continued access for patients already receiving any of the licensed treatments - link below)
Back in 2019, a ‘Buyers Club’ was developed to bring these drugs in from Argentina as generics, for a fraction of the price (link below)
Evaluations of cost-effectiveness have been run in several countries already. The conclusion is that prices need to fall by 70-90% for the Vertex drugs to be considered cost-effective. Therefore a price for Trikafta in the region of £30,000 per year would be more realistic.
Vertex Pharmaceuticals has been strategically securing patents in many countries, which is a standard practice to protect their inventions and maintain market exclusivity.
In many countries, despite holding patents, Vertex doesn't offer the drugs for sale. This move effectively blocks other pharmaceutical companies from stepping in to create generic versions of the drugs, which could be sold at a much lower price.
Generic manufacturers have demonstrated that they can produce these drugs at a fraction of the cost, but Vertex's patent holdings create a legal barrier preventing this cheaper, generic production.
This strategy not only maintains Vertex's monopoly on drugs but also leaves many countries without any access to them at all, exacerbating the global accessibility issue.
A potential way forward is for governments to take action against pharmaceutical companies which charge excessive prices for their drugs. Options include importing these drugs from other countries where generics are available, with support for ‘Buyers Clubs’, or by placing windfall taxes on Vertex.
What can we do?
Sign the Global Access Petition
The global access petition was set up by organisations Just Treatment and Vertex Save Us who are on a mission to fight for global access to modulator therapies for all.
Recent developments have reawakened many in the UK to the issue of access to modulator therapies and several prominent CF creators have since shared the petition resulting in a rapid increase in signups of almost ten thousand throughout the weekend.
However, it is important to recognise that the petition goes beyond the UK cause.
The issue of access has been a global concern for many years. Most countries have never had access to Vertex medications and won't unless the company revises their pricing. This issue has been at the forefront of the efforts of Just Treatment and Vertex Save Us for several years, and UK access is now once again part of this collective mission.
Everyone involved wants the same thing, and if Vertex’s values mean anything it shouldn’t be too much to ask: access to CFTR modulator therapies at a fair price.
Just Treatment and Vertex Save Us have made lots of progress over the last few years with many influential individuals and organisations behind them. Your support and the support of your family and friends adds depth and breadth to the global access campaign, which is important, as it will take a united worldwide effort to fight against a company as huge as Vertex.
Our petition is a different kind of petition - it’s a demonstration of worldwide unity, the number of countries with issues (which is most of them), and if when signing you agree to allow Just Treatment and Vertex Save Us to send email updates to you, it allows huge united actions to take place.
In short, this petition is the place to be if you're in the fight for access. Please sign it and pass it on.
Groups and Pages to Follow:
As we keep fighting for access both in the UK and globally, these are the groups to follow to collaborate in finding solutions and taking action:
Just Treatment:
Website: https://justtreatment.org/
Instagram: https://www.instagram.com/justtreatment/
Facebook: https://www.facebook.com/justtreatment
X (Twitter): https://twitter.com/JustTreatment
Vertex Save Us:
Instagram: https://www.instagram.com/vertexsaveus/
Facebook: https://www.facebook.com/VertexSaveUs/
X (Twitter): https://twitter.com/VertexSaveUs/
Strawfie: Prominent UK and Global Campaigner:
CF Voices:
Facebook: https://www.facebook.com/CFVoices
UK Kaftrio Group:
Summary
I understand the nervousness and worry that this week's announcement has caused within the UK CF community. However, it's important to understand that this is not only a UK issue, it is a global issue.
My intent with this article has been to give you a clear understanding of the past and present truths and to direct you to communities online where we can unite in the hope of finding solutions.
The root cause of the issue is not NICE, it’s Vertex Pharmaceuticals' exorbitant pricing, which presents a very real challenge to healthcare authorities worldwide, including NICE. The high cost of CFTR modulators makes it impossible for healthcare authorities to find them a cost-effective treatment within their calculations.
This in no way diminishes the value of lives that could be improved with the drug; it's simply a matter of affordability and sustainability over time.
For true global access, Vertex needs to agree to either a significant price reduction or they need to allow for generic versions of the drug to be manufactured. The pressure to force this change can only come from one place: us.
On one hand, some of us may see Vertex as our saviours—after all, they created a drug that has the potential to add decades to our lives. But ask yourself this, do they really care, or was CF simply an undeniable business opportunity to make obscene amounts of money?
The answer to that question reveals one simple conclusion: Vertex and its leaders are perhaps not so ‘nice’ after all.
References and further reading:
NICE full guidance: read the full guidance.
Web links for "Severity modifier" explainers:
Vertex Mission and Values.
Vertex full-year sales forecast.
Vertex Sales: SEC Filings.
Vertex spent $3 billion buying back their shares.
Vertex destroyed modulator stocks instead of negotiating a better price.
Trikafta: Minimum cost of production.