Cystic fibrosis and access to medicines

Cystic fibrosis (CF) is a genetic condition affecting more than 10,400 people in the UK. You are born with CF and cannot catch it later in life, but one in 25 of us, often unknowingly carries the faulty gene that causes it.

CF has changed radically since its pathology was first described in the 1940s, when surviving infancy was extremely unlikely – through to the foundation of the Cystic Fibrosis Trust in 1964 where surviving to secondary school age was remarkable – to today where more than half of people living with cystic fibrosis are adults, of whom nearly 70% are in education or employment.

This progress is a result of incremental improvements mixed with some fundamental step changes that have included shifts in both clinical practice and drug therapy. However, the median age of death in 2017 was still just 31 years old.

As people with CF and their families work so hard to carve out the space to live their lives as they would want, CF still steals health, happiness, independence and opportunity. Loss, heartbreak, pain and struggle are a constant shadow to everyone affected by the condition.

The mystery of the genetic underpinnings of CF disease began to be unravelled in the late 1980s and the hope that we could one day treat the disease at its root cause – rather than continue to build a heavyweight and burdensome arsenal of symptom-fighting interventions – became a realistic prospect.

It would take another 20 years but – jumping to 2013 – people with CF with a rare CF-causing mutation, known as G551D, were the first to benefit from a disease-modifying therapy for cystic fibrosis on the NHS. Kalydeco (ivacaftor) produced by Vertex Pharmaceuticals, unlocked the cellular block that leads to the thick, sticky mucus that wreaks havoc on the internal organs – particularly the lungs – of people with CF.

Then in 2015, jumping from the 5% of people with CF who could benefit from Kalydeco, the same company received a European license for a new disease-modifying therapy, known as Orkambi (lumacaftor-ivacaftor), which had been proven effective in people with two copies of the most common CF-causing mutation, raising the treatable population to more than 55%.

2018 has seen the licensing of a new drug to treat a similar population – Symkevi (tezacaftor-ivacaftor, Vertex) and early and remarkable data from a phase III trial of a so-called triple combination therapy that looks certain now to fundamentally transform the disease for 90% of people with CF in the UK.

2018 has also seen a desperate stalemate on pricing negotiations spill into acrimonious public pronouncements, statements, ultimatums and thinly-veiled threats. There is no access to the drugs beyond Kalydeco and the small minority of people with CF who can benefit from it.

Since Orkambi received a negative recommendation from NICE in 2016, there has been substantially more strategic and tactical posturing than any substantive negotiation from all the parties involved: Vertex, NICE, NHS England and the UK Government.

In time, through the UK CF Registry, we will demonstrate, in the starkest of terms, what loss this failure to reach agreement has meant to the lives of people with CF and their families.

As the transformative benefit of triple therapy combination therapy piles even greater pressure on the parties to find the compromise that will allow people in the UK to access these medicines, the UK will be starting from a lower baseline of health for its population, in comparison to the dozen other European countries that have granted access to Orkambi and Symkevi, including Germany, Holland and Ireland. The data we hold will bear that out.

With the Health and Social Care Committee in Westminster due to publish private negotiations between the parties involved, people with cystic fibrosis may be interested to know whether representatives of the NHS and Government have worked tirelessly to find a solution and explore every option they have available. They may be interested to know whether Vertex have poured their time and effort into bringing every shred of evidence forward to justify the price they wish to charge.

I expect they will be deeply disappointed. Both parties have divided loyalties. While the goal of improving the lives of people with cystic fibrosis is shared, protecting investors and shareholders or the public purse and precedent appears to have won out – for now.

The Cystic Fibrosis Trust is committed to supporting all parties to appropriately evaluate the value and impact medicines can have on the lives of people with cystic fibrosis and we are confident that we have an important role to play in finding the way forward towards a deal.

For now, thousands of families share the feeling expressed by these parents, who wrote to the Health and Social Care Committee last month:

‘We write to you as parents, our seven-year-old daughter has cystic fibrosis. She has the genetic mutation that Vertex’s drug, Orkambi, targets and would improve her quality and length of life.

We cannot explain, let alone expect you to understand, the sheer dread and helplessness it is to know that we are likely to outlive our daughter. The anguish in knowing that a drug exists that can change that and spare her from unnecessary suffering and decline in health, but that she is denied it, is unbearable. To refuse her, and many others, a drug, that is made in the UK and sold worldwide from the UK, is inhumane.

The Department of Health and Social Care, NHS England, NICE and Vertex have all had a part to play in this inexcusable situation, and we are very grateful that you have opened this inquiry.