Meeting of the Parliament

Meeting date: Tuesday, June 12, 2018

Official Report 1191KB pdf

---

Orkambi

The final item of business is a members’ business debate on motion S5M-12545, in the name of Maurice Corry, on Orkambi. The debate will be concluded without any question being put.

Motion debated,

That the Parliament understands that cystic fibrosis affects over 900 people in Scotland, including in the West Scotland parliamentary region, and that, in 2016, half of all people who died from the genetic condition were aged under 31; believes that Orkambi, which is manufactured by Vertex Pharmaceuticals and treats the homozygous delf508 mutation, which around 50% of people in the UK with the condition have, is the second drug to be licensed for use in dealing with cystic fibrosis; notes that the main function of this treatment is to keep a healthy balance of salt and water in the organs, particularly the lungs; believes that, according to 2016 UK Cystic Fibrosis Registry Report, there are 336 people in Scotland who could benefit from having access to this, but that it is not currently available via the NHS, despite, it understands, the Scottish Medicines Consortium noting that it was a beneficial treatment and NICE recognising it as an important treatment; believes that clinical data has shown that the drug is able to slow decline in lung function, which is the main cause of death from the condition, by 42%, and notes the work of all the people and organisations, such as the Cystic Fibrosis Trust, which are campaigning to put pressure on the Scottish Government, the NHS and pharmaceutical companies to ensure that such drugs reach those who need them without delay.

17:04  

I thank the members who have supported my members’ business motion so enthusiastically over the past week or so.

Cystic fibrosis is a devastating genetic disease that has a terrible effect on patients and their families. From very early in life, children who have cystic fibrosis can exhibit multiple manifestations of the disease, including structural lung damage and abnormal lung clearance, which is a way of measuring airways’ health, and they also face nutritional impairments. The damage that they sustain to their lungs is progressive—it worsens over time and leads to increasing impairment of lung function. One consequence of that is that people with cystic fibrosis are more susceptible to life-threatening lung infections.

Due to the geographic origins of cystic fibrosis, the United Kingdom has a very high prevalence of the disease, accounting for some 12 per cent of the global population of patients. NHS Scotland has estimated that one in 24 Scots has a genetic mutation that can, if it is present in both parents, lead to a child being born with cystic fibrosis. According to the United Kingdom cystic fibrosis registry, there are approximately 900 people in Scotland with cystic fibrosis. They have a median age of just 21 and the median age of death is just 31. Only 5 per cent of cystic fibrosis sufferers will live to see their 50th birthday. I want members to think about that for a second. My 31st birthday is but a distant memory, but those with cystic fibrosis who reach that milestone have just a 5 per cent chance of getting to a 50th birthday.

I want members to think about what they would not have been able to do if they had died before one of those birthdays. I want them to think about the experiences that they would have missed and the memories that they have created with loved ones that would never have happened. That is the reality for so many people who suffer from cystic fibrosis. Surely it is incumbent on us to ensure that people with cystic fibrosis have the best chance of having as many of those experiences for as long as possible in order to create those memories. That is what Orkambi can do.

As I note in my motion, clinical data has shown that Orkambi is able to slow decline in lung function, which is the main cause of death from the condition, by 42 per cent. It has cut the number of infections that require hospitalisation by 61 per cent. According to the Cystic Fibrosis Trust, about 40 per cent of people with cystic fibrosis in Scotland would benefit from treatment with Orkambi.

The drug is different from traditional treatments for cystic fibrosis in that it is a precision medicine. Traditional treatments for cystic fibrosis aim to reduce symptoms and complications, but progressive damage still occurs, which means that symptoms and complications increase with age. However, precision medicine targets the root cause of cystic fibrosis—the dysfunctional protein that causes cystic fibrosis. Precision medicines have the potential to preserve or to restore lung function, and to slow decline and improve the life expectancy and quality of life of patients.

Other precision medicines are being developed. Within five years, about 90 per cent of people with cystic fibrosis could be treated with new medicines that could transform cystic fibrosis from being a condition that kills people to one that they can live with. However, people with cystic fibrosis are worried that they will not get access to those life-changing medicines in Scotland because of the process that the medicines need to go through to become available on the national health service here. Orkambi received its licence two years ago, but is still not available here, although it is available to all eligible patients in Austria, Denmark, Germany, Luxembourg, the Netherlands, Italy, Ireland, Greece and the United States. It is still unavailable here because the Scottish Medicines Consortium announced in 2016 that it was unable to recommend the drug due to its cost, despite acknowledging that the drug is “important and effective”.

Since then, people who suffer from cystic fibrosis and organisations including the Cystic Fibrosis Trust have called for negotiations and a fair sustainable pricing deal for Orkambi. Sadly, there has been no progress, so people with cystic fibrosis are still waiting for help while their health and quality of life decline.

I know that elsewhere there has been success in negotiating prices with Governments. Vertex Pharmaceuticals, the company that makes Orkambi, also makes Kalydeco, which is another cystic fibrosis precision medication. Kalydeco is available in Scotland via the new medicines fund, following the intervention of ministers in 2013, but Orkambi has not had the same intervention. That means that inequality has been created among cystic fibrosis patients, depending on the treatment that they require.

A good example of a different way of opening up access to new medicines is the Republic of Ireland’s approach. By agreeing a portfolio approach, which is a long-term solution, new medicines for cystic fibrosis have become available for patients in that country when they are manufactured and licensed. Deals of that type mean that the overall prices of current and future medicines are capped for a set amount of time, which means that a doctor can move a patient on to new medicines if they would better address that patient’s particular type of cystic fibrosis. That approach has major benefits, and science is now moving so quickly that I worry that the old models for bringing medicines into the system no longer work.

I want to quote Mr and Mrs O’Neill of Lenzie, from my region, who wrote to me and encapsulated what I think the debate is all about. They say:

“My son is 18 months old and he is the future—he should not be denied access to precision medicines that will support him to live the life he so rightly deserves. Although not now, Conan’s health will deteriorate at some point—his life should not be shortened even further by denying Conan and the other 907 people in Scotland who live with cystic fibrosis access to the medicines that they need. It’s not just about Orkambi, it’s about what comes after Orkambi and that is why it is so important that an approach can be agreed that allows access to the pipeline of future life-giving medications and treatments. Scotland led the way with access to Kalydeco and must lead the way again.”

We must have unity throughout the United Kingdom. The National Institute for Health and Care Excellence has approved the use of Orkambi in England and Wales. We should do the same in Scotland.

Thank you, Mr Corry. I remind all members who wish to speak to press their request-to-speak buttons, please. I see that some folk have woken up.

17:12  

I congratulate Maurice Corry on obtaining the debate and for his opening remarks, which were very informative and well balanced.

One thing that Maurice Corry did not mention is that the impact of having cystic fibrosis from birth results in a decline of lung function by about 2 per cent a year, on average. That means that cystic fibrosis sufferers have lost about 20 per cent of their lung function by the time they get to the age of 10. By the time they get to the age of 20, they have lost 40 per cent, and by the time they get to the age of 30, they have lost 60 per cent of their lung function.

I emphasise that because the debate is as much about timing as it is about anything else: time is marching on for cystic fibrosis sufferers. The longer it takes to complete the process to get approval for the new drug—Orkambi—and successor drugs, the more time will be lost for cystic fibrosis sufferers. Therefore, the matter has to be treated as being very urgent.

The good news is, as Maurice Corry said, that we are on the brink of a major transformation in the treatment of cystic fibrosis because of the nature of the advanced new drugs that are coming on the market. However, as a former health secretary, I know the challenges that arise when expensive new drugs that have demonstrable impacts are being made available. When we are looking at the entire picture of the health service, we have to be mindful of the cost—not just the unit cost but the overall cost—of any new drug. That is why the SMC system was set up to take an objective and non-political look at new drugs.

However, sometimes it is necessary for Government to knock heads together. The stage that we are at just now, in particular with Orkambi, is that we need the Government to knock together the heads of the manufacturer—Vertex—the SMC and NHS National Services Scotland, which is responsible for NHS procurement on behalf of the Scottish Government. It is right that politicians are not directly involved. The role of politicians is to set the framework and, if necessary, to intervene where there is undue delay.

For the rest of my speech, I will therefore concentrate on making a suggestion to the Scottish Government and Vertex about the way ahead. The one thing on which we are all united is the need to do everything that is within our power, budget and resources to ensure that cystic fibrosis sufferers get the treatment that they need at the earliest opportunity.

I welcome the decision that became effective about two weeks ago to make Orkambi available under what used to be called the individual patient treatment request system—now, the peer approved clinical system. That is progress, but it is not enough progress, because we know that getting approval under that system is not always fast or guaranteed. That is why the quicker we get general approval for the drug, the better.

Will the member take an intervention?

Sure.

I am sorry, but Alex Neil has only 30 seconds left, which I am sure he wants to use.

The time has come for the Scottish Government to knock heads together—those of Vertex and NSS. Two things are necessary. The Government needs to agree to enter into portfolio negotiations, which are outwith due process. It is a novel way of negotiating access to drugs, but we should not let bureaucracy and being outside due process hold us up. The Government should authorise the start of those discussions. The discussions will not be finished quickly, so in the meantime, Vertex should make the drug available at a reduced price so that, when the deal is done, both sides end up where we are trying to get to. Let us do that sooner rather than later.

17:16  

I apologise for being unable to stay for the whole debate because, as I have indicated to the Presiding Officer, I have to leave to chair a meeting of the cross-party group on cancer.

I congratulate my colleague Maurice Corry on securing the debate and I commend his excellent speech, which I whole-heartedly agree with. I very much welcome the broad cross-party support on the issue and I pay tribute to the Cystic Fibrosis Trust for its work and to constituents in my region—Lothian—who are campaigning hard to ensure that Orkambi is available on the NHS to all who need it.

One such constituent is Jenny Landers from Musselburgh, who emailed me just a few days ago about her daughter Freya, who is five years old and has cystic fibrosis. Jenny Landers said:

“Every day Freya takes up to 30 tablets, 4 nebulisers and does 45 minutes of physiotherapy just to keep well. Even with these treatments her health is slowly declining.

She has already been admitted to hospital 3 times for up to 2 weeks at a time. Currently her future is very uncertain as many people with CF are still dying in their twenties.

In one year she will be eligible for Orkambi.

This drug has the potential to slow the pace of the disease, giving her a much better chance of staying well into adulthood and having a career, living independently and having a family. Things most people take for granted. It is not right that we are denying people with CF the chance at a better life.”

None of us will disagree with Jenny Landers’s sentiments, which are shared by many parents, families and friends of people with CF across Scotland. Alex Neil laid out for us the challenge, which is how each of us as elected representatives, the Scottish Government, the manufacturers and the SMC’s processes translate that desire, and the accepted fact that the medicine is a beneficial and effective treatment, into access to the drug on the NHS at an affordable rate that is fair for everyone involved and is sustainable for our NHS.

In quite a few ways, the campaign to access Orkambi mirrors that for Perjeta, which I have been involved in recently, through which women with breast cancer want access to another life-extending drug. I am pleased that Perjeta’s manufacturers have announced that they will make a resubmission to the SMC and I hope that Vertex will do something similar for Orkambi in the shortest possible time.

I commend Vertex’s scientists, who have a large pipeline of potential CF treatments in development that could offer a great deal to many patients. We should celebrate that, and we should all look at how we ensure that the SMC meets the challenge.

I welcome recent reforms that mean that clinicians can make requests to NHS boards for individual patients to access drugs that the SMC has not yet approved, but such requests are not always successful and, understandably, campaigners want Orkambi to be available to everyone who needs it, without delays or extra processes to go through. Maurice Corry referred to the process through which Orkambi was made available in the Republic of Ireland. We need to examine that and consider whether our systems are capable of mirroring it.

I welcome the debate. It is a chance for the Parliament to focus and has brought the campaign for Orkambi here. I hope that the whole Parliament will unite in supporting our constituents whose lives could be improved by access to Orkambi, which is routinely available in many European Union countries and elsewhere.

I hope that the Minister for Public Health and Sport is willing to meet with MSPs from across the parties who are working with the CF Trust and other organisations on the issue. I also hope that, in her closing speech, she will assure us that she will do whatever she can to facilitate Vertex getting to a position where it can make an acceptable application.

Patients with cystic fibrosis in Scotland—many of whom face limited life expectancies—and their families rightly expect the Scottish Government to step up and get things moving. I and other MSPs from across the chamber will keep up the pressure on the Government. Above all, we need action on the matter so that our constituents can realise their potential.

17:21  

Like others, I congratulate Maurice Corry on securing the debate. It is important and timely and the issue has far-reaching consequences: it is literally one of life and death. Orkambi is a life-changing drug that improves the quality of life for cystic fibrosis patients. The genuine cross-party support for and nature of the debate emphasise how important the issue is. I recognise that it is not an easy one for any minister or Government and I hope that, collectively, we can find a solution that is in the best interests of patients.

It is worth noting that Orkambi is available in many countries throughout the world, including our European neighbours, Ireland and Holland. It is more than two years since the drug received its European Medicines Agency licence and almost a decade since the first clinical trial started in Scotland. However, it is still not available to Scottish patients. The reason given—I quote directly from a letter from the cabinet secretary’s office—is that

“justification for the treatment’s cost in relation to its benefits was not sufficient.”

What price do we put on life? For individual families and patients, that reason will not be much comfort. I recognise the difficulty that the Government has and the difficult decisions that the SMC has to make, but we must do that in the context of a genuinely humane approach on such issues, particularly when we are talking directly about people’s lives.

Miles Briggs mentioned that Orkambi is not an isolated case. Perjeta is also a running issue. That demonstrates the challenges that we have with providing access to vital medicines. Perjeta is a vital medicine that is available to breast cancer patients in other parts of the United Kingdom but not in Scotland. That will impact on the life expectancy of those patients. The matter requires a robust response and approach from the Government.

I echo Alex Neil. He took a fair-minded approach and called on the pharmaceutical company, the Scottish Government and the SMC to put their heads together and find a solution that is in the best interests of patients and their families.

I will offer a few other quick reflections. First, I will comment on the letters that we received from the cabinet secretary. I was really disheartened by the letter that came in on 31 March, addressed to Jackie Baillie and Alex Neil, because one of those members is a former health secretary and they were taking a genuinely cross-party approach, but the letter was not a personal response from the cabinet secretary. That was ill judged on her part. I am pleased that she has followed that up today with another letter to Mr Neil and Ms Baillie, but I say gently that it should not take the front page of a national newspaper or a debate in the Parliament for the cabinet secretary to respond directly to fellow parliamentarians.

I will also comment on the individual patient treatment request process and the ease of access to it. It is welcome that our discussions about Perjeta led to a change in approach: requests will be judged not on money but on clinical efficacy. However, alongside that, we have to recognise that that will impact on health boards’ budgets, so we need the Government to back up those budgets to make sure that money will be available to make the drugs available if the IPTRs are approved.

Although these debates may be more far reaching than the SMC review, its recommendations could have dealt broadly with the challenges that we face on Orkambi and Perjeta, which are the ability to negotiate and to make a drug available while those negotiations take place in the interim accepted period. I urge the Government to knock heads together on the Orkambi and Perjeta issues and to get on and implement the SMC review recommendations so that we can stop or limit such situations from happening in the future.

I say gently to members of the public—I am about to tell you off—that we do not permit applause from the public gallery. I know why you are doing it, and it happens regularly, but I am afraid that it is not permitted.

17:26  

I congratulate Maurice Corry on bringing the debate to the chamber. Members will have noticed that my colleague Alison Johnstone waited until the last minute, but she has to chair a meeting tonight. She is our Green health spokesperson, so I will refer to her work along with my personal reflections on the issue.

I have great admiration for the achievements of the pharmaceutical industry—but there is a “however” coming: public health should not be in the hands of profit makers. I am uncomfortable with what I see as the wheeling and dealing that is openly talked about, as we are talking about individuals. My attention has been drawn to the just treatment campaign and the Crown use licence, which Alison Johnstone has written about. Although I accept that it is not a quick solution to anything, there is a feeling that the scrutiny and public concern that might be directed at companies as a result of that intended approach—which is a legal mechanism that allows patent law to be overridden and another producer to be put in place—might result in reduced prices.

Alison wrote to the Cabinet Secretary for Health and Sport to say that she was concerned about a

“lack of clarity about the basis on which the Scottish Government intervenes directly in decisions about drug approval and purchasing.”

On the specific issue under consideration, she wrote:

“Understandably, many cystic fibrosis patients feel that it would be inequitable for the Scottish Government to intervene in order to make cystic fibrosis treatments which act on one genetic stratification available, but not ... so for others.”

I am not a legal or health person, but I understand about the Montgomery review, which the Scottish Government is committed to implementing. It is incredibly important that decisions are medically led. There is a role for parliamentarians and politicians, but people will be concerned about what is being seen as the politicisation of access to medicines.

Alison touched on that point in her letter to the cabinet secretary, when she wrote that we have an

“opportunity to develop a robust, transparent and equitable approval process which is open to scrutiny. One of the review’s recommendations is that a comparative review of arrangements for the introduction of medicines in other countries should be undertaken.”

She went on to commend

“New Zealand’s Pharmac model, and the potential to adapt aspects of that model”

in the Scottish context.

Members have alluded to some of the existing drugs, and I suspect that there will be another one next week and another in future months. We must get the process right—that is most important. I am very uncomfortable when we talk in terms of sums of money when we are talking about people’s lives.

I have a particular interest in the matter because of my constituent Hannah McDiarmid. I was delighted when she and her mother came to the Parliament as my guests in January. Many members signed a motion about her. She was a University of the Highlands and Islands student of the year who achieved a BA honours degree in Gaelic language and culture at Sabhal Mòr Ostaig, for which she studied latterly via distance learning. Hannah was diagnosed with cystic fibrosis at 17 weeks, and her health has deteriorated in the time that I have known her. Most recently, she has been coughing up blood due to a condition that I am unable to pronounce, and she has just managed to get her lung function back up to 50 per cent after it declined to 45 per cent recently. She spends two hours a day undergoing physiotherapy to clear mucus from her chest and lungs. Hannah has given me a list of her condition’s symptoms. She is a charming young woman, and she is very grateful for the assistance that she gets from her nurse, Lesley Blaikie. Her mother is also deeply affected by the condition.

There are other drugs, but the situation regarding this drug is very important and we must get the process right. As someone has said, what price a life?

17:30  

I congratulate Maurice Corry on securing the debate.

A few weeks ago, with Alex Neil—who, of course, is the former health minister who made Kalydeco available for cystic fibrosis sufferers in Scotland—I organised a cross-party meeting of MSPs. The meeting was attended by a substantial number of MSPs from every party in this Parliament, many of whom are here tonight. It was hugely encouraging that, for things that matter, we can put aside our differences and join together to fight in a common cause.

I organised the meeting because of my constituent Kelli Gallacher, who has cystic fibrosis. She is a bright, intelligent and happy young woman. She recently bought a home with her boyfriend, much to the delight of her parents, because it meant that she left the house. She works for the local council and has dedicated her spare time to raising awareness of cystic fibrosis and raising thousands of pounds to improve the day-to-day lives of those who have the illness.

The debate is happening—[Interruption.] I promised that I would not cry. The debate is happening because Kelli and hundreds of others cannot get access to Orkambi—a drug that is available to CF sufferers in Ireland, America and the Netherlands and that would enhance Kelli’s life.

It does not stop at Orkambi. The next generation of medicines that will effectively ensure that those with CF live to a ripe old age are just round the corner. We have an historic opportunity literally to save lives. As Alex Neil described, instead of one drug at a time, the pharmaceutical company is offering a portfolio deal for all the drugs—something that already exists in Ireland. The company is in discussion with the NHS in England and Wales, but it is not discussing the same terms in Scotland. Instead of talking about one drug, which is all that the Scottish NHS seems to want to do, we should be talking about having the opportunity to access them all.

On 25 April, Alex Neil and I wrote to the Cabinet Secretary for Health and Sport, asking her to meet Kelli Gallacher and a cross-party group of MSPs. I wrote again on 30 May, following our cross-party meeting, with the same request. I received a response on 31 May, which said that the cabinet secretary’s diary was too full for her to meet MSPs but that she would meet Kelli. That was welcome.

I received a further email from the cabinet secretary’s office yesterday, simply noting the second letter. When I contacted her office immediately to ask whether that was it, I was told that she had nothing to add. I was genuinely disappointed, but I was happy to receive a letter about 2 hours ago—just before the debate—outlining the peer-approved clinical system tier 2 process, which enables individual patients to apply for drugs, and the method for appealing decisions. As Alex Neil said, that process is helpful but it takes time, and time is something that CF sufferers do not have. Orkambi was licensed two years ago and is still not available. The cabinet secretary has now agreed to meet Kelli, which is great, and Alex Neil and I have been included in that meeting, but only at Kelli’s request.

I thank the Daily Record for its moving and robust campaign in support of Kelli Gallacher and all the cystic fibrosis sufferers in Scotland. Today, the Daily Record highlighted Kelli’s pleas to the First Minister, and I encourage members to read Kelli’s letter to the First Minister. The Daily Record has also previously covered comments from Gordon MacGregor, who is Kelli’s consultant. I absolutely understand John Finnie’s approach, which is that the clinicians need to be on board. Gordon MacGregor briefly spoke out about access to medicines. He has had to stand by helplessly while

“a young man is dying in a hospital bed while the drug which will save his life sits, untouched, along the corridor.”

Presiding Officer, when Alex Neil and I agree, you know that something extraordinary is happening. We need the Scottish Government to get things moving, because a portfolio deal plus interim access now is what is needed. I will finish with Kelli Gallacher’s words:

“We shouldn’t have to fight for drugs that could save our lives. Some of us don’t have time to wait.”

17:35  

I, too, thank Maurice Corry for bringing this important debate to the chamber this evening. I remind the chamber that I am a registered nurse and convener of the cross-party group on lung health. We will meet this evening—I am sure that we will be late starting—and cystic fibrosis and Orkambi are on the agenda for us to discuss. I have sought permission from the Presiding Officer to exit the debate before the final speeches, as I have that long-standing engagement to attend to.

I acknowledge the work of the Cystic Fibrosis Trust. That work needs to be at the forefront, and the trust has done a lot of important work to raise awareness of the issues.

Cystic fibrosis affects 70,000 people across the world, including 30,000 in the United States and 900 here in Scotland, which seems like a wee drop in the ocean. Our ability to give people access to the medications that can support them to have a healthier, more prolonged life out of hospital and our ability to support those people’s families need to be considered. There are new disease-modifying drugs such as Orkambi and others that will present soon.

I will not go into detail about cystic fibrosis, but I will say that it is a condition that affects not just the lungs but the pancreas and the ability to digest protein, carbs and fats. The amount of medication that people have to take every day can be high—as Miles Briggs mentioned, it can be up to 30mg. Patients need to demonstrate a real commitment to adhere to therapy—both the meds and the adjunctive care of physiotherapy and exercise—in order to manage their health. It is important to consider that when we look after those patients.

When I spoke to my sister Phyllis, who is a respiratory nurse consultant, and her colleague Stuart Little, they assured me that they were working hard with all their patients and colleagues. Phyllis said that for drugs such as Symdeko, which will be available, and Orkambi, which could become available, the research is progressing and evidence is increasing—the research is looking really good. It is showing that those medications have a direct ability to support the way that protein is activated, or pushed, in order to make salt and water transfer across cell membranes. When we engage with health professionals and clinical consultants, such as Gordon MacGregor, and hear about the work that they are doing, it is clear that we need to make sure that they are part of the process.

When I heard Alex Neil and Jackie Baillie speak about the drugs being managed in Ireland with a portfolio approach, my first thought was, “How can we introduce a portfolio of drugs when some of them have not even been presented yet?” I was concerned about the safety aspects of introducing a portfolio of meds. However, when I met the Vertex representative, I was assured that each drug would still be presented as an individual drug, even though a portfolio option could be approved. I think that it would be worth considering such an approach as an interim approach while negotiations are taking place.

I would be interested to know how the Scottish Government will support and engage with the drug company, the NSS and the Scottish Medicines Consortium. It is the year of young people and we want to promote the extension of young people’s lives—my sister says that some people are living to the age of 51—so it would be great if we could support more folk with cystic fibrosis in Scotland to have healthier, out-of-hospital lives.

17:40  

I thank Maurice Corry for bringing the debate to the chamber today and I recognise the work of the Cystic Fibrosis Trust.

I also thank Robert Barker, who is a constituent of mine, for allowing me to tell his remarkable story of receiving Orkambi at what he thought would be near the end of his life. His father is in the public gallery today, along with many others. They are close family friends, so I have seen the progress of Robert’s improvement, as well as the other side of it.

Robert said:

“I started Orkambi in January 2017. Prior to this, my health had been deteriorating over the years due to my cystic fibrosis. I was having more regular hospital stays, more chest infections and I was now experiencing many other problems associated with the condition.”

He went on to tell of the progressive challenges that he had faced. He said:

“In 2016, I spent nearly 100 nights in hospital receiving strong IV drugs to treat the symptoms. On the few occasions I was well enough to stay at home, I was hooked up to oxygen 24/7 and I could not even get around my own home without being severely out of breath. I could no longer look after my family, go to work, drive or walk anywhere. On top of this, I had to spend most of my day doing my daily treatments, taking nebulisers, doing physio and taking a long list of other medications. By the end of 2016, my weight had dropped to 50kg and my lung function had dropped to a very serious 17 per cent, despite all the medication and care I was receiving. I knew that I didn’t have long left, and that my wife and I would soon have to explain this to my six-year-old daughter. Fortunately, this all changed in January 2017 when I was prescribed Orkambi on compassionate grounds, as a lung transplant was not an option for me. After three to four weeks on Orkambi, my lung function had risen above 30 per cent and I was able to come off oxygen. After three months on the drug, it had climbed to 45 per cent and I was able to return to work full time. By the summer, I felt great and I was able to take my family on holiday—I had my life back. A year on from first taking Orkambi, my lung function is nearly 60 per cent and my weight is up to 65kg. I am able to do everything I need to do physically. I have had no chest infections, no hospital stays and no sick days from work. On top of this, my doctor has reduced several other drugs that I had to take even before I got really sick. Orkambi is an excellent drug—it may not be effective for everyone, but it should be available to all CF sufferers who may benefit from it, as many of them are running out of time like I was.”

I am sure that everyone in the chamber will agree that Robert’s story is both heart rending and filled with hope for the future. He can now spend time with his family and lead a normal life, which, before Orkambi, would simply not have been possible.

As Robert and many others in this chamber and beyond have stressed, Orkambi might be expensive and does not work with all patients, but the price of the drug should not prevent it from being available on the NHS. It is important to face the fact that this is about young children, how they develop and how they need a life, as Maurice Corry said. It is about preventing progressive deterioration.

It is disappointing that the strong group of MSPs from across the parties was denied a meeting with the cabinet secretary in the response from her private secretary. In my short six years at the Scottish Parliament, I have never been to an evening meeting with MSPs that was so well attended and passionate.

I, for one, whole-heartedly condone approving Orkambi for all those who might have their lives saved, and I urge members, including those not in the chamber, the cabinet secretary, in particular, and the minister who is here today to listen to calls from across the chamber and beyond. I urge them to ensure that Robert’s pleas—and those of many others—for Orkambi to be made available are recognised.

17:45  

As other members have done, I commend Maurice Corry for securing the debate and for speaking with passion and authority, and I commend all the other members who have contributed.

The debate is timely, because it precedes cystic fibrosis week. I pay tribute to the Cystic Fibrosis Trust for its significant campaigning efforts across Scotland and beyond.

It has been a difficult and emotional debate. Who can fail to have been moved by the stories of the lived experience of people being articulated so powerfully by MSPs across the chamber?

Maurice Corry spoke about Mr and Mrs O’Neill and their 18-month-old boy, and their hopes and concerns for him as he grows up with cystic fibrosis. Similarly, Miles spoke about Freya Landers, and we also heard about the remarkable young women Hannah McDiarmid and Kelli Gallacher, who will be meeting the Cabinet Secretary for Health and Sport soon.

I am also pleased to see Ralph Barker in the gallery. I also know him and how close Claudia is to Ralph and his boy. I thank Claudia for saying what was quite a distressing thing for her to have to say.

I am sorry, minister. I did not want to intervene, but we must stop using first names in the chamber. You have been doing it throughout.

I apologise, Presiding Officer. Like everyone else, I probably got caught up in the emotion. I did not intend to disrespect Parliament, but I wanted to make sure that I put on the record my acknowledgement of Claudia Beamish’s sincere contribution about somebody she knows and who is dear to her and her family.

The tributes and testimonies that we have heard this evening highlight the debilitating impact that cystic fibrosis can have, the limitations that it puts on life, and the need for us to think clearly about how to help in the best way that we can. Much of the consideration of how best to help people who are living with cystic fibrosis has been based on the availability of the appropriate medicines. The Government absolutely shares members’ desire to increase the availability of medicines that patients in Scotland need. That is why we have sought to reform the systems that are in place and to introduce changes that enable us to get medicines to the people who need them. We want to build on those positive changes.

Between 2011 and 2013, the combined acceptance rate for orphan and cancer medicines was 48 per cent. However, from 2014 to the end of 2017, under the new approach that we brought in, the Scottish Medicines Consortium approved 79 per cent of such medicines. The SMC provides a clear and consistent process for consideration of medicines that are being appraised. From that appraisal, the SMC determines whether a medicine should be accepted for routine use in the NHS. That decision is—rightly—independent of ministers and Parliament.

Although such positive changes are all well and good, I know that the members here tonight and, more important, people who have cystic fibrosis and whom we have heard about in the debate are interested in the fact that Orkambi is not routinely available on the NHS anywhere in the United Kingdom. To clarify the position that I think Maurice Corry suggested, I note that the National Institute for Health and Care Excellence has not accepted Orkambi. That is why, last year, the cabinet secretary strongly encouraged Vertex to enter discussions with NHS NSS. We are pleased that that has happened. Those confidential talks are on-going and we hope that, as part of the discussions, Vertex will make its best offer on price and indicate that it will resubmit to the SMC as soon as possible.

Like many other members, I have been contacted by constituents who take a close interest in the issue. In my case, they are the grandparents of a five-year-old girl who has cystic fibrosis. One of the main features of today’s debate has been discussion of how we can reduce the amount of time between the drug being given the green light—if it is given the green light—and being available for use. I ask for the minister’s assurance that that will be addressed by reducing the timescale as much as possible.

I will go on to talk more about some of the improvements that we seek to make. Again, that is why it is important that members from across the chamber send a clear message to Vertex to ensure that it resubmits to the SMC as soon as possible.

The cabinet secretary also recently updated the Health and Sport Committee on the further work that we are undertaking to deliver on the recommendations from Dr Brian Montgomery’s review of access to new medicines, in order to maximise the benefits to patients. In doing so, we continue to work closely with our partner organisations, stakeholders, patient representatives and the pharmaceutical industry. I acknowledge the call from MSPs for us to do that.

The use of real-world evidence that captures the outcomes of medicines is also becoming an increasingly important element of our work, and was one of a number of data-related recommendations in the review. That is particularly relevant in this debate, as there can often be uncertainty about the robustness of the clinical evidence where the clinical trial data is limited due to small population sizes, so we look to build our use of data to support that.

Can the minister clarify by what date we will have full implementation of the review findings?

I undertake to ensure that we get information to Anas Sarwar about timetables for implementation, if he would find that useful.

We will continue to support access to new medicines through our new medicines fund. Officials are also actively examining an improved negotiating function that seeks to ensure that NHS Scotland pays the same effective price for medicines as the rest of the UK.

We recognise that the current appraisal pathway is less well suited to medicines for very rare conditions, so we are seeking to include a wider assessment of lived experience, including quality-of-life issues. That is an important element, given the need to have a holistic picture of how a condition impacts upon the life of the individual. I know that, despite the progress that I have outlined and the plans that we have in place to improve that further, that provides limited comfort to patients who need access to medicines that are not yet available. However, it is important to recognise that an SMC “not recommended” decision does not mean the end of the journey for patients. There is, as members have said, a new process in place that enables doctors to request medicines on an individual patient basis for medicines such as Orkambi.

The new peer approved clinical system—PACS—tier 2 process was introduced at the start of this month, and replaces the old individual patient treatment requests. It introduces refreshed national decision-making criteria, standardised processes and a new national review panel to enhance consistency of decision making right across the country. The new system requires doctors to present an evidence-based case to demonstrate their opinion that the patient can achieve a clinical benefit that is comparable to or better than the benefit to the population that was previously considered by the SMC. Importantly, the guidance is explicit that the cost of the medicine must not form any part of the decision-making process, and that arrangements should be only for exceptional cases.

That is why we again urge Vertex to do everything it can in its discussions with NHS NSS to find a solution at a fair price, and to do so as quickly as possible, in order that the SMC can consider a new submission.

The crucial issue, and the one that seems to be preventing real discussions between Vertex and NSS, is that Vertex is saying that the Scottish Government will not approve portfolio discussions because that is “outside due process”. Can we get clarification? Will the Scottish Government, in preparation for the meeting next week between Vertex and the chief pharmaceutical officer, instruct the pharmaceutical officer to open discussions based on the portfolio discussion? That seems to me to be the sensible thing to do, and it would open up the way for an interim arrangement. It is absolutely crucial that that happens.

I clarify that confidential talks between Vertex and procurement officials are on-going. I know that Alex Neil remembers from his stint in office that there are always considerations that the Government has to make in order to seek to do the best we can for the people whom we serve. I know that the portfolio approach sounds appealing, but we must recognise that that approach involves the NHS potentially entering agreements to purchase unlicensed medicines, the safety of which remain unproven. Despite assurances, the risk exists and it is real, so our Government must consider that. That approach also risks stopping the NHS from getting access to future medicines that may be better and offer better value. I will ask officials to look at the issue, but we need to recognise that there is a risk in purchasing unlicensed medicines and in reducing the NHS’s ability to get access to future medicines that might offer better value. We need to be mindful of those things, but we will ensure that—

Will the minister take an intervention?

I have taken a number of interventions, so I would like to bring my remarks to a conclusion. I will certainly ensure that Jackie Baillie also gets the information that I have promised to Mr Neil on portfolio discussions.

The debate is not easy to respond to. The stories and testimonies that we have heard are powerful and real, and I pay tribute to the individuals who have campaigned to make a difference, because they are inspiring.

However, I hope that the Government’s determination is clear to create a system that is fair and consistent, but which has within in, where needed, the agility to respond to exceptional clinical need, and greater cognisance of lived experience. Those measures illustrate progress.

On behalf of the Government, I look forward to continuing to work with members across the chamber on this important issue, as members have called for. Where I have pledged to get back to members with additional information, I will do so as quickly as I can.

I commend members for their contributions.

Meeting closed at 17:55.