Cystic Fibrosis drug given green light in England

24 October 2019

A life-extending drug for cystic fibrosis will be available on the NHS in England, health bosses say.

NHS England reached a deal with Orkambi manufacturers Vertex Pharmaceuticals after months of talks.  Patients should be able to get the drug within 30 days.

The drug improves lung function and reduces breathing difficulties and can be given to children as young as two.

The firm wanted to charge £100,000 per patient per year but a compromise has been reached in a confidential deal.

It is understood to involve significantly less than the sum originally asked for.

Two other drugs made by Vertex – Symkevi and Kalydeco – will be made available as part of the deal.

These also treat cystic fibrosis symptoms.  Symkevi is restricted to over 12-year-olds, while Kalydeco can be used from 12 months.

The treatments do not work for all patients with cystic fibrosis – only those with certain mutations.

It is estimated about half of the 10,000 patients in the UK will benefit from these drugs.

Cystic fibrosis is a life-shortening genetic condition that can cause fatal lung damage.

Only about half of those with the condition live to the age of 32.

‘Best present ever’

Patients and campaigners have expressed delight at the announcement.

Christina Walker said it meant her son, Luis, eight, should be on it by Christmas, calling it the “best present ever”.

“I can’t stop smiling. I’m overwhelmingly happy. It’s absolutely wonderful,” she said.

David Louden, from Carlisle, said the decision would make a “huge difference” to his daughter, Ayda, who has cystic fibrosis.

He said the battle to get it had been “demoralising”.

“You could see this drug with all its benefits that was just hanging there in the balance, dangling like a carrot in front of you but you couldn’t access it.”

‘Good for patients and taxpayers’

More than 10,000 people in the UK have cystic fibrosis, which cause fatal lung damage

The deal comes after the Scottish government reached an agreement with the manufacturers last month.

Under the terms of the agreement both Wales and Northern Ireland can now access the drug for the same price as NHS England, but there has been no announcement over whether they will.

NHS England chief executive Simon Stevens said the deal was “good for patients and fair to British taxpayers”.

Health Secretary Matt Hancock described it as “wonderful news”.

David Ramsden, of the Cystic Fibrosis Trust, said: “This is a very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting.”

He also said there could be more good news in the pipeline as a new therapy Trikafta, which 90% of people with the condition could benefit from, was getting close to be licensed for use.

‘Life-changing’ cystic fibrosis drug deal for Scotland is welcomed

12 September 2019

SNP MP Marion Fellows with granddaughter Saoirse, who was diagnosed when she was three weeks old

Families have welcomed a deal which will make two “life-changing” cystic fibrosis drugs available to eligible patients in Scotland.

A five-year agreement has been reached between the Scottish government and pharmaceutical company Vertex over the use of Orkambi and Symkevi.

The drugs improve lung health but were rejected for NHS use last month because they were not deemed cost-effective.

The medication normally costs £100,000 per patient, per year.

The Scottish government said it had secured a “confidential discount” from the manufacturer.

Patients in other parts of the UK are still not able to access the treatment, with campaigners calling for an end to the “postcode lottery”.

‘Never let up’

SNP MP for Motherwell and Wishaw Marion Fellows, whose three-year-old granddaughter Saoirse has cystic fibrosis, said it was a “big moment” for patients and their families, who had been “relentless and inspiring” in their campaign.

Ms Fellows said: “I’m so glad that so many families across Scotland will benefit from these new drugs. Hundreds will be able to lead longer and fuller lives thanks to this decision.

“The Scottish government never let up in discussions and is leading the way in cystic fibrosis treatment.

“But cystic fibrosis knows no borders. The UK government must follow the Scottish government’s lead and make it available to sufferers in England also.”

The drugs can reduce hospital admissions by stabilising lung health

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage, and affects about 10,400 people in the UK – around 900 of them in Scotland.

Only around half of those with the condition live to the age of 40.

NHS Scotland estimates that one in 24 Scots have a CFTR mutation which, if carried by both parents, would lead to a child being born with cystic fibrosis.

Glasgow mum Gail Gilmour, whose son has cystic fibrosis, tweeted that there were “no words adequate” to thank those who helped secure the deal for Scottish patients.

Others have said that they hoped it would lead to progress in negotiations for England, Wales and Northern Ireland.

Earlier this year campaigners appealed to the UK government to use its powers to break a deadlock in making Orkambi available on the NHS in England.

Vertex refused a £500m offer for the drug over five years – which was described as the “largest commitment” NHS England had ever made.

Those affected want other drug firms to be asked to make a cheaper version.

The UK government’s Department of Health said its approach was still to urge Vertex to accept NHS England’s “generous offer”.

‘Landmark moment’

The Scottish Medicines Consortium (SMC) had rejected the routine use of the drug in August, saying there were uncertainties about the long-term health benefits of Orkambi and Symkevi in relation to their costs.

David Ramsden, chief executive of the Cystic Fibrosis Trust, said the deal was a “landmark moment” for the hundreds of people with CF and their families across Scotland.

“This breakthrough is a victory for their perseverance and enduring hope. It means 350 eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.”

He said the trust would continue to campaign for access to the drugs in England, Wales and Northern Ireland, adding: “Scotland’s success must now be replicated across the UK without further damaging delay.”

What are Orkambi and Symkevi?

Since 2015, the drug Orkambi has been licensed to treat cystic fibrosis in patients from two-year-olds to adults, who have a specific genetic mutation known as F508del.

It was not available on the NHS, except for certain people on compassionate grounds.

Symkevi is used to treat the same mutation in patients age 12 and older.

The mutation causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

Orkambi has been shown in clinical trials to improve lung function and respiratory symptoms in people with cystic fibrosis.

It is the first of a string of drugs that have been developed, with newer ones expected to be even more effective.

Scotland’s Health Secretary Jeane Freeman said the “fantastic news” would allow cystic fibrosis patients to “live fuller lives for longer”.

“The agreement has been reached after extensive discussions between the Scottish government and Vertex Pharmaceuticals and means the medicines will now be made available to patients on the NHS in Scotland, subject to a confidential discount,” she added.

Vertex has also committed to collecting real-time data on the medicines to support any future submissions to the SMC.

Ludovic Fenaux, Vertex senior vice president, said: “We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution.

“It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi and Symkevi.”

Some Scottish patients have previously had access to Orkambi and Symkevi through the Peer Approved Clinical System Tier 2 (PACS Tier 2), which allows doctors to apply for access on behalf of individual patients.

A spokesperson for SMC said the pharmaceutical industry had responsibility for bringing their medicines to market at a fair price.

“The SMC members make these difficult decisions based on extensive clinical experience and taking all of the available evidence into account, including that provided by patient groups. We look forward to receiving new evidence from Vertex on the impact of these medicines following the agreement they have reached with the Scottish government.”

A time for the Cystic Fibrosis community to celebrate…

Today I’ve had news that NHS England has reached a deal with Orkambi manufacturers Vertex Pharmaceuticals after months of talks.
This was brought to my attention through the Cystic Fibrosis (CF) clinic I attend after it was announced on BBC News (see link).
CF is a genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections.
The effects of CF reduce the quality of life of patients as they age and whilst over the years the quality of life and average life expectancy has improved due to advancing treatments, the research into new treatments will always be ongoing that will eventually see CF being cured.
Orkambi is currently the closest to a cure for CF. Though until now has been offered to patients on compassionate grounds. Either as a result of a drop in lung function or as a result of volunteering to take part in an Orkambi related drug study.
I’ve been taking Orkambi since January 2017 on compassionate grounds after taking part in a drug study.
Within a week commencing the drug, I found I was able to breathe easier and the first month saw a vast improvement with my endurance. Now my endurance is back to how it was 5 years ago.
You can say today’s news has been a huge welcome and something worth celebrating about.

World Mental Health Day

Thursday, 10 October 2019

Today is World Mental Health Day. Some will know what it means to raise the importance of talking about what is on your mind, which is one way of dealing with mental health. Another is doing things that make you happy such as being in a job you love, being around people who appreciate who you are, going the gym to train and fight through the effects through cardio and strength, hobbies, etc.

I have the odd bout of fearing not my future, but my demise. Why? Some will already know one reason why. Unlike some, I am not in denial of my mental health. I’m rather open about it and deal with it by talking or doing something that helps. My mental health issue is something that comes and goes. What helps is keeping busy with things like work, weekends at the cottage and the gym. Sometimes I can be motivated. Sometimes not and just don’t want to do anything.

With the gym, a lot of motivation comes from my PT, who helps by encouraging me through a good cardio and strength workout. The last few sessions have been amazing compared to a year ago. My breathing control and technique have improved vastly. My lung function reaching nearly the same score I was at 5 years ago.

I’ll be blunt, but had it not been for the much needed improved treatment for CF, I question whether I’d still be here, or whether my health would be as good as it was 10 years ago, which is why I take my meds and have the fitness lifestyle I have.

The meds and healthy lifestyle have maintained my health well, so the thing that baffles me is despite how well I am and how well I’m doing, I still have the odd mental fall.

It sort of pisses me off more than anything because I shouldn’t be feeling like this with how well I’m doing. Though I’m positive that I’m not the only one who is like this.

One thing for sure is I talk about how I feel rather than shut myself off and not talk at all about it. I’d much rather have people around me than push people out my life as some have done.

There is great importance to have people around you as had they not been there, I don’t think I would be here.

Though sadly not everyone is in the same way of thinking.

Life with Cystic Fibrosis

Originally published on Tuesday, 28 February 2017

When I was young (I’m a 1980’s child), management of CF was primitive compared to how it is now.

Diet & Treatment
From what I remember, Creon wasn’t on the market until around 1985. However, there was another tablet that aided digestion, which wasn’t as powerful as Creon. As a result, fatty foods such as cake, crisps, biscuits, etc were foods to avoid in the CF diet. So the introduction of Creon came as a shock to me when I was allowed these types of food and although I am able to have these foods, I don’t really eat them due to not having much of a sweet tooth.

Over the years treatment has improved vastly, aiding a better quality of life and CF people living longer too.

Not only has treatment improved over the years, but also equipment used to administer them. With advances in technology, nebuliser equipment has become more compact and treatment taken through these have become quicker to take too.

Medical advances seem to be happening fast at the moment with all these promising drug trials.

OK not all trials work out as we hope, lessons from them are learnt for the next drug trial, so all is not lost.

It was once believed exercise was bad for the lungs of CF patients based on the coughing fits caused as a result.

It was later realised coughing during exercise was down to the airways opening up as a result of deep breathes, which in turn improved the lung function.

Mix and match
Before segregation came into force in 1991, CF patients were allowed to mix no matter the strain or bugs they had. Little was known about cross-infection.

Looking at how segregation works now, people new to CF and use to the segregation rules will not believe that there was a time when CF patients went on coach trips together. One trip seeing the patients camping at Sherwood Forest, with 6 patients of different bugs and strains sharing a tent.

Lessons learnt about CF for new parents of children with CF
From my own experience, one thing I’ve said to parents new to CF is to encourage exercise with their CF child from an early age. Find something the child will enjoy and would take up as a hobby.

I am lucky to have a mild form of CF and have coped with it really well.

However, not all has been rosy with CF. There are times when I get upset, stressed or anxious about things. In particular when I’m not well.

However once treatment for a chest infection shows signs of working, the psychological strain this has on me is lifted.

CF will have a psychological effect on not just patients, but also their parents, siblings and partners. This side of CF happens to everyone at some level.

CF people do try to remain positive and tend to use a lot of humour as a coping mechanism, which for me works.

Every day will be a learning curve for anyone, whether it be for a CF person, relative or even the CF team at the specialist clinic.

One thing for sure, despite the effects CF can have, the treatment of today means we get to live a better quality of life and live longer and longer.

Two years ago a man well known at my hospital passed away at the age of 75, which for me is the oldest known patient.

If he can live so long especially when at the time he was born little was known about CF, then there is hope for us all.

Cystic Fibrosis, Orkambi & Warrington Borough Transport

Originally published on Monday, 20 February 2017

On my way home from Pure gym, I was delighted to see Warrington Borough Transport (WBT) display a poster raising awareness about Cystic Fibrosis (CF) on the bus stop on Manchester Road opposite the Esso garage.

Had I not been driving, or the roads were busy at the time of travelling, I would have pulled over to take a photo of this as it really made my day to see such publicity in aid of a life illness that I and many others suffer from.

I thought it would be nice to give WBT an insight into what I’ve been through with the new exciting breakthroughs in regards to better treatments for CF, and also update the Warrington Guardian with how things have been since I did the QBW251 trial in October 2015, a drug that was to be the closest to a cure for CF.

Although the outcome of the QBW251 drug trial concluded with the drug not being as effective as predicted in lab tests, there have been other similar medicines that have made it onto the market and used by CF people who meet criteria due to high costs for the drug.

One drug, which was on the news in recent months, is called Orkambi. This is a pink tablet whereby 2 are taken twice a day with food.

Orkambi is on the market in America but is yet to come on the market in the UK due to high costs whereby for 1 CF person, costs mount up to £100k per year.

However, all is not lost.

There have been continual drug trials happening over the last couple of years to find a drug that either work in the same way as Orkambi or will work alongside to make Orkambi more effective.

I am currently doing a drug trial whereby I have to take Orkambi, which is being tested alongside a trial drug that’s inhaled to see the effects these combined drugs have on CF.

At present, I am on week 4 of the trial whereby I am taking Orkambi before I start the trial drug next Tuesday (28 February 2017).

The drug trial program is as followed:

  • Month 1 – take Orkambi only
  • Month 2 – take Orkambi and trial drug
  • Month 3 – take Orkambi only
  • Month 4 – take Orkambi, trial drug and Hypertonic Saline (a nebulised salt solution that helps clear the lungs)

At the end of the trial, as a reward for my volunteered help, I will be allowed to continue Orkambi.

The question you’ve probably been asking throughout this email is, “Have I noticed any effects of Orkambi?”

The simple answer is, yes.

“What effects have I experienced?”

I’ve been able to breath better and when I cough, the feeling I have in my lungs is as though nothing is there.

My coughing that occurs in the morning after a nights sleep has reduced dramatically to almost non-existent.

When I’m at the gym I am able to work that little bit harder on the exercise bike and my heart rate at its peak has dropped from 150 beats per minute to 120.

I am yet to know how Orkambi has affected my lung function. At the beginning of the trial, my lung function was 44%, so it will be interesting to see how this changes over the coming months.

Had it not been for the likes of Warrington Borough Transport and many others who raise awareness of CF, the donations raised for the CF Trust would be almost non-existent and vital drug trials, an example described above, will not happen.

It may have been one poster on the side of a bus stop that I have seen on my travels through Warrington, but if I have spotted the poster, there’s a chance other commuters and bus users will have seen this too.

I am pretty sure if every single person donated £1 after seeing the poster, this would help fund further investigations into vital treatments leading to a possible cure for CF.

So thanks again Warrington Borough Transport for your contribution in raising awareness of CF, a life-threatening illness.

New Online Prescription Service

Originally published on Monday, 13 February 2017

Lloyds Pharmacy informed me Holes Lane Surgery have been chosen for a pilot online patient service, which has already been rolled out across the Halton and Liverpool areas.

The service is called “SystmOnline“.

It is very easy to register. All you need to do is take 2 forms of ID (1 picture ID and a utility bill with your home address on).

I was able to use my driving licence as a picture and address ID, which saved time having to dig out a utility bill.

I was given a letter with a website address for the surgery on, my username and also a temporary password, which I changed once I logged into the service for the first time.

This online service allows you to do the following:

  • Book appointments
  • Check future appointments
  • Check previous appointments

Pros: you’re not having to phone the GP surgery on a 0845 number and wait for an answer, saving your phone bill.

  • Order repeat prescription
  • Check if you have any uncollected prescription
  • Register or change which pharmacy you would like to collect prescriptions from.

Pros: this saves you having to travel to your local pharmacy or GP surgery to delivery a repeat prescription form.

Although you can visit the pharmacy/surgery in person to place an order, this saves you time on travel.

Cons: Not all people will have computer access or know how to use one. If they’re able to travel to the GP surgery to place a prescription order this won’t be an issue. If not, they will be able to have a patient representative to do this for them.

For more information about this online service, click on this link.

JANUARY 2017: 40 years of Cystic Fibrosis Awareness

Originally published on Tuesday, 17 January 2017

On 15 October 1977 my older brother, Paul, then aged 6 years old, sadly passed away after being overpowered by the deadly genetic disorder, Cystic Fibrosis (CF).

On 24 August 1979, I was born with a much milder strain of CF compared to what Paul had.

Today would’ve been his 46th birthday and this October will be 40 years since my parents lost their first son and on 24 August 2019 time will be my 40th birthday.

As a way to say thanks to the care us CFers receive, I, with your help, would like to raise funds for the Manchester Adult CF Centre at Wythenshawe Hospital as had it not been for their efforts and help with the care of myself and others with CF, I don’t know where we’d be right now.

The money raised will go towards equipment needed to help improve the quality of life of patients who endure CF and stay as inpatients for what can be days, weeks or sometimes months whilst being treated for their illness. Some of whom like my brother sadly spend their last moments there.

So please, dip your hands in your pockets, purses, wallets and back of sofas, and donate what you can no matter how big or small, and make my 40th something special to remember.

To donate your gift, please visit Manchester Adult Cystic Fibrosis Centre and mark your donation with “Peter Trengove”.

Annual Health Checks

Originally published on Thursday, 20 March 2014

On an annual basis, I have an annual review on how I’m doing with my Cystic Fibrosis.

Yesterday (Wednesday, 19 March 2014) I was given the results of my annual review, which was done in December 2013.

Usually, I’m given goals on how I can improve my health with things I do, such as:

  • how to get into the habit of doing exercise,
  • improve taking nebulised medication,
  • improve nutrition intake to maintain weight,
  • deal with my mental wellbeing, which can be affected by CF i.e. anger, depression, being self-conscious as far as body image is concerned, etc.

The Doctor said for this coming year, they had found it very hard to come up with goals for me to follow.

He felt there was nothing I could do to help improve my condition.  Simply because the usual goals set as described above I’m already doing. Over the last year:

  • I’ve got into an exercise I truly adore.
  • I’ve been put on inhalers that replace my nebulisers and do the same job.
  • I’ve maintained a healthy diet that has maintained a healthy weight.
  • My mental wellbeing has improved vastly as a result of the above.

I was shown chest x-rays from previous annual reviews from 2010 to 2012. It was explained the white shadows on these x-rays were sputum. The whiter the shadow the more the sputum.

The x-ray from 2013 was almost black, which indicated that due to the new medication and my workouts I’ve radically improved the function of my lungs, gaining an extra capacity of 0.75 litres (up to 4.75 from 4 litres in previous years).

Various other checks such as blood levels, liver function, etc were normal.

Last year when I saw the positive effects the new inhalers had, I wanted to know what exercise would do.

Now I know how good a combination of taking my treatment properly and exercising regularly, I want to know what more I can do.

It just goes to show that as long as you look after your body, your body will look after you.