UK Government debate access to life saving drug for people with Cystic Fibrosis

Apr 1, 2018 · 8 min read

Cystic Fibrosis (CF) is the UKs most common life-threatening genetic condition that affects the lungs, digestive system and other organs. The gene affected by CF controls the movement of salt and water in and out of cells. People with cystic fibrosis experience a build-up of thick sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms affecting the entire body.

The build-up of mucus in the lungs causes chronic infections, meaning that people with cystic fibrosis struggle with reduced lung function and have to spend hours doing physiotherapy and taking nebulised treatments each day..

CF affects 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 carries the faulty gene that causes it, usually without knowing.

There is no cure for CF, however a drug called Orkambi has been developed by Vertex Pharmaceuticals and is the second precision medicine that tackles the underlying cause of CF rather than just managing the symptoms.

About 3,500 people with Cystic Fibrosis in the UK could benefit from Orkambi, if it was funded by the NHS.

There are many different gene mutations that cause CF. Orkambi treats the F508del mutation, which around 50% of people with CF in the UK have, about 3,500 people could benefit from the drug.

Orkambi has been proven to slow the decline in lung function — the main cause of death among people with CF — by 42%. It also reduces the number of times a patient is hospitalised because of an infection by 61%.

Despite this, in June 2016, the National Institute for Health and Care Excellence (NICE) has rejected its use by the NHS due to it’s high cost.

Orkambi is available to all patients in Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, Ireland, Greece, and the United States, but not the UK.

Since NICE’s rejection 2016, the Cystic Fibrosis community have been campaigning to continue negotiations between the NHS, the UK Government and Vertex Pharmaceuticals. With the ultimate aim of reaching an agreement on price as soon as possible, to ensure Orkambi reaches the people who need it.

The debate was well attended with an impressive 60 MPs in Westminster Hall. Many MPs commented on the extraordinary numbers of MPs that attended and told the stories of their constituents who are waiting for Orkambi.

Paul Scully MP open the debate, and made a strong case for Orkambi, based on the inequalities of the NICE appraisal system and the availability of Kalydeco for a small number of patients.

”We can talk about the Government getting involved in pushing NHS England and Vertex together to make a sensible deal in this case, but I come back to the point that this is an analogue system in a digital age. I will try to do it justice, but it is a case of inequality. About 400 patients have access to Kalydeco, which I mentioned earlier. They have a particular mutation to which Kalydeco responds. Around 3,000 patients would be eligible to access Orkambi.”

“The current evaluation process turns on an incremental cost-efficiency ratio, which is the total additional lifetime cost of a treatment divided by the additional quality-adjusted life years resulting from that treatment. For acute conditions, the additional quality-adjusted life years resulting from the shorter-term treatments moderate the efficiency ratio, even if the drug is expensive. However, because drugs for chronic and lifelong conditions have to be taken every day for life, the cost of the treatment prevents that downward moderation. Basically, it is easier, under the NICE appraisal system, for medicines for acute conditions to attain a more favourable cost-effectiveness outcome than for innovative medications for chronic conditions, like Orkambi. It is basically a one-size system.

“We then have to take into account section 13G of the National Health Service Act 2006, as amended by the Health and Social Care Act 2012, which requires NHS England to have regard to the need to reduce inequalities in health outcomes. Those two imbalances in the system need to be looked at if we are to have a system that is far fairer for people with illnesses such as cystic fibrosis.”

Ian Austin MP has campaigned on behalf of Cystic Fibrosis patients for a long time.

”Hon. Members are supposed to start their speeches in these debates by saying how pleased they are that the debate is taking place, but I am not pleased at all. I think that this is the third debate on this issue in which I have taken part over the past few years. We have presented petitions at Downing Street. We have had campaign events in Parliament. As has been said, we had 41 Members at a roundtable just a few weeks ago. And we are still here. Three years after Orkambi was approved for use and two years after NICE said that it was “important and effective”, we are still here, waiting for it to be provided for people with cystic fibrosis. So I am not pleased that we have to have another debate about this issue, but whatever I feel about that is nothing compared with the upset and worry — indeed, the terror — that people with cystic fibrosis and their families go through as they wait while their health, life expectancy and quality of life decline.”

Ian Austin MP told the story of one of his constituents, who is receiving Orkambi on compassionate grounds.

”Orkambi has changed my life. I quickly became well enough to start to live a more normal life again as a working mum. My health has remained stable. I only need one or two courses of IV’s per year instead of four. Hospital visits have massively reduced and admissions are non-existent.”

Friends and family of people with Cystic Fibrosis have been protesting hard at Parliament Square.

Many MPs repeatedly called on Vertex to be reasonable with their prices, NHS England to meet with Vertex and continue negotiations and for the Government to get involved and take responsibility to finding a resolution.

Marion Fellows MP, made a very moving and personal speech.

”I must declare a personal interest. My 16-month-old granddaughter Saoirse was diagnosed with cystic fibrosis shortly after she was born. On hearing about the debate, my daughter-in-law enlisted all of her friends and family to sign the petition, even though she knows that Orkambi will not help her daughter. Vertex is developing new treatments all the time, and the use of Orkambi can only help that research.”

”Orkambi could be just the start of a whole line of drugs that will benefit CF suffers. No one can cure the disease at the moment, but such drugs will lead to so many more better and productive lives. Yes, there are issues around pricing new drugs. Pharmaceutical companies are not there for charitable purposes, and we all know and understand how expensive it is to create new drugs, but there must be a better way of pricing the cost of new drugs at their introduction across years of their use rather than huge up-front costs.”

Steve Brine MP, Minister for Health, ended the debate with a response from the Government.

”I pay tribute to the Cystic Fibrosis Trust, which does a tremendous job on behalf of all our constituents. I know we are not meant to address the Gallery. I will not do so, but if I did I would thank those who have made the trip in the snow and ice, probably not all from SW1, to come to Westminster today. It is a credit to them that they have made time to do that.”

”The negotiations must remain constructive and be undertaken with the utmost urgency, for all the reasons we have heard — I will not repeat them all — or, as my hon. Friend the Member for Mid Dorset and North Poole (Michael Tomlinson) said, people living with cystic fibrosis will suffer.”

”It seems to me that the main ask I have heard from pretty much every hon. Member speaking today is, “Get involved.” I am not the Minister directly responsible. That falls to Lord O’Shaughnessy, the Parliamentary Under-Secretary who sits in the other place, but I can say on his behalf and on mine that we are involved, and you can bet your life that we will continue to be involved. I do not think I can be clearer.”

”The Government wholeheartedly support efforts to ensure that the precision medicines we have heard about are made widely available to CF patients and other patients. It is true that high-cost precision medicines represent a challenge to the NHS, but they are also a tremendous opportunity to deliver high-quality outcomes through highly specialised treatment. I hope I can go some way to ensuring that people with comparatively rarer conditions such as CF get the same quality, safety and efficacy in medicines as those who have more common conditions, and to doing so in a way that is sustainable for the NHS.”

”I know I speak for my colleague, Lord O’Shaughnessy, when I say that we are both impatient for a breakthrough and are watching the matter like hawks. I get the message loud and clear: the House has said, “Get involved.” The House has that assurance from me.”

Campaigning hard during the Beast from the East

Friends and family of people with Cystic Fibrosis have been protesting hard at Parliament Square.

They battled #Snowmageddon from the #BeastFromTheEast to protest on 28th February. During the 3 hour debate, friends and family again battled the snow and ice from #MiniBeastFromTheEast. The protesters have vowed to continue until their voice is heard and Orkambi and future CF drugs are available for everyone who needs them.

As there is the potential of cross contamination of harmful bacteria between people with Cystic Fibrosis, many could not join the protest in person. So they made their voices heard via an online campaign, sharing their stories on social media with the hashtag #OrkambiNow.

We continue to ask that;

  • The UK Government must take responsibility for negotiating an agreement as soon as possible
  • Drug company Vertex must ensure fair and responsible pricing for a deal to be agreed
  • The UK Government must acknowledge the importance of finding a solution that guarantees we are never put in this position again for the future pipeline of treatments for cystic fibrosis

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Orkambi is a precision medicine that tackles the underlying…


Orkambi is a precision medicine that tackles the underlying cause of Cystic Fibrosis. However it is denied to people in the UK.


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Orkambi is a precision medicine that tackles the underlying cause of Cystic Fibrosis. However it is denied to people in the UK.


Orkambi is a precision medicine that tackles the underlying cause of Cystic Fibrosis. However it is denied to people in the UK.

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