Life-saving drugs FAQs

Choose from the frequently asked questions below to find out more about our fight for access to life-saving drugs for everyone living with cystic fibrosis.

Hand drawn image of lungs

About precision medicines

  • What are precision medicines?

    People with CF have two copies of a mutation of the gene that causes cystic fibrosis (CF). An example of precision medicine in CF could be a drug that targets one or a small number of these mutations (or ‘genotypes'). Each class of mutation affects the production of the Cystic Fibrosis Transmembrane Regulator (CFTR) protein (which controls the movement of salt and water in and out of cells in your body's organs) differently. This is why the Trust ran the Genotype Matters campaign, to highlight the importance for people with CF of knowing their genotype, as knowing it could make the difference between getting access to new medicines or missing out. Take a look at our mutations video to find out more.

  • What is a combination therapy?

    A combination therapy combines more than one compound, which work together to impact the production or function of the CFTR protein, or otherwise improve lung function.

  • What is a triple therapy?

    A triple therapy combines three drugs that perform different functions, including a potentiator and a corrector. These drugs work together to impact the production or function of the CFTR protein, or otherwise improve lung function.

  • What is a ‘corrector’ and a ‘potentiator’?

    A corrector compound will help correctly fold the CFTR protein (as mutations mean the protein can’t correctly fold itself), and the potentiator helps opens the channel in CFTR to allow chloride out.

  • What is the drug pipeline?

    The ‘drug pipeline’ is a phrase used to describe future medicines in the context of the journey each drug takes through the process of development in the laboratory, then through clinical trials to the availability on the NHS. The final trial stage, Phase IV (4), takes place when the drug has been made available publicly, to monitor its long-term efficacy in the real world. Find out more about clinical trials.

  • How are drugs licensed?

    A new treatment needs to be given a licence for use before it is appraised for clinical and cost-effectiveness in any nation in the UK.

    How are drugs licensed if I live in:

    Wales / Scotland / England

    Now that we have left the European Union (EU), the body responsible for this in England, Scotland and Wales is the Medicines and Healthcare products Regulatory Agency (MHRA), the UK’s standalone medicines and medical devices regulator.

    The MHRA’s decision, called Marketing Authorisation, is made after it has analysed whether a new treatment is both safe and clinically effective. It makes no judgement on the cost of a new medicine when deciding whether or not to grant a licence. Find out more about how drugs are tested in clinical trials.

    Northern Ireland

    Currently the MHRA only approve drugs for people living in England, Wales and Scotland. The European Commission approve drugs for people living in European countries including Northern Ireland. 

  • How are drugs appraised?

    There are different appraisal bodies across the UK.

    - England – National Institute for Health & Care Excellence (NICE)

    - Wales – All Wales Medicines Strategy Group (AWMSG).

    - Northern Ireland – NICE/Department of Health and Social Care (DHSC).

    - Scotland – Scottish Medicines Consortium (SMC)

    Once a new treatment has a licence, drug appraisal bodies weigh up the price a company has asked for against the clinical trial and other relevant data. They then recommend whether the drug will be clinically- and cost-effective for the NHS, in the context of available budget.

    The NHS is legally obliged to fund drugs recommended by these bodies. However, it also works the other way. If a drug is not recommended, then the NHS is not obliged to fund it.

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Triple combination therapy (Kaftrio)

  • What is Kaftrio?

    Kaftrio is a 'triple-combination therapy' made up of three different compounds, tezacaftor and ivacaftor (which together make up Symkevi) combined with elexacaftor.

  • Why is the drug called Kaftrio?

    Trikafta is the US name for the triple combination therapy, whereas the drug has the brand name Kaftrio in Europe. The two names refer to the same drug.

  • Who is currently able to benefit from Kaftrio?

    The European Commission and the MHRA have formally licensed Kaftrio for use by people with CF who:
    - are aged over 6
    - have two copies of the F508del mutation or one copy of F508del and any other mutation.

    The various drug regulatory bodies across the world take different approaches to how they interpret evidence on the safety and clinical effectiveness of new drugs and ultimately what licence indication should be granted. In the UK, the MHRA is the regulator which makes these decisions.

    There may be other reasons why someone is not able to benefit from Kaftrio. We have a factsheet on the complex and individual experiences around Kaftrio, for those who are taking it, as well as those who are not. Download the factsheet

  • When did Kaftrio become available?

    Kaftrio was first made available to UK patients in August 2020. Since then, the license has expanded to include younger age groups and a wider range of Genotypes.

    NHS England announced on 30 June 2020 that a deal had been agreed for Kaftrio. This was followed by similar deals in Wales on 22 July, in Northern Ireland on 30 July and in Scotland on 4 August, this enabled the drug to be prescribed as soon as it was licensed in the UK on 21 August 2020 for patients aged 12 and older with one F508del mutation and one minimal function mutation, or two F508del mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. 

    The deal with NHS England included 'tag along rights' for the other devolved nations that paved the way for similar deals across the UK and the Crown Dependencies of Jersey, Guernsey and the Isle of Man.

    The Trust has worked closely with the UK CF Medical Association to support roll-out and ensure everyone can start Kaftrio as fast as clinically possible. 

    On 11 January 2022, the European Commission and MHRA approved Kaftrio for eligible children aged 6-11 in England, Scotland, Wales and Northern Ireland.

  • What effect does Kaftrio have?

    In clinical trials for Kaftrio, people with two copies of the F508del mutation had a 10% increase in lung function compared to treatment with Symkevi, and people with a single copy of F508del and a minimal function mutation had more than a 14% increase in lung function compared to treatment with the placebo.

    Clinical trial data show that many people taking Kaftrio will have improvements in gastrointestinal (GI) issues and overall quality of life. Further studies are underway to better understand the long-term effects Kaftrio will have on the overall health of people with CF, including infections, inflammation, mucus clearance, Gl health, blood sugar, growth, and liver function.

    Kaftrio has shown to be safe and effective, with potentially fewer negative side effects than previous licensed modulators.

  • What is the Trust doing to support access to Kaftrio?

    We are working with all key stakeholders to support access to everyone in the UK who could benefit.

    We are working with the UK Cystic Fibrosis Medical Association to support the roll-out to ensure everyone can start Kaftrio as fast as clinically possible.

    We also host and manage the UK CF Registry, which will continue its valuable work in monitoring the effectiveness of this drug, among many others.

  • Are there other therapies like Kaftrio being developed?

    It is difficult knowing that not everyone will stand to benefit from Kaftrio or the other currently licensed modulators.

    AbbVie and Vertex Pharmaceuticals are developing other potential triple combination therapies.

    Through our Clinical Trials Accelerator Platform, we are actively working to support the ongoing HIT-CF Europe research project which aims to provide better treatments for people with rare CF mutations who do not benefit from Kaftrio.

    For some people with CF, transplant remains the only option to prolong their lives. We are working hard to make sure more people with CF are able to have a successful transplant, and to improve support offered to those following on from their transplant.

    The Trust is fully committed to finding effective treatment for everyone with cystic fibrosis. You can find out more about our research that leaves no one behind and donate to support this vital work.

  • Will I be able to stop my other medications and treatments?

    Do not stop any of your medications or treatments without first speaking to your CF team. Studies are underway and in development to explore whether these drugs can reduce the number of other treatments people with CF take.

  • Will I still have to worry about cross-infection?

    People with cystic fibrosis who have been prescribed CFTR modulators such as Orkambi, Symkevi, Kalydeco and Kaftrio are still susceptible to infection and should be aware of and take precautions against cross-infection. These drugs are able to reduce infections that would require hospitalisation, but there is currently no evidence to suggest that they can prevent them altogether and therefore there is still a significant risk of cross-infection between people with CF and close contact should be avoided. You should speak with your CF team if you have any questions or concerns.

  • Can I take Kaftrio if I have received a lung transplant or other organ transplant?

    Currently Kaftrio, and other modulator therapies, are not recommended for most people who have received a transplant. This is because most of the benefits from the treatments are seen in CF lungs, and are not seen in new transplanted lungs. 

    Another reason they are not recommended is that they can come with side effects, and can also interfere with the immunosuppressant medications that people have to take after lung transplant. 

    However, if you have severe CF symptoms outside of your lungs after transplant, then it would be worth talking to both your CF team and your lung transplant team, as sometimes (although very rarely) it could be worth considering. There are reports of people, mainly from America, who have done so, and the majority (but not all) stopped the treatment because it caused side effects or didn’t help.

    We are also aware of reports where it seemed to cause a drop in lung function that only improved when Kaftrio was stopped. If you would still like to consider it, please talk to both your lung transplant team and your CF team, who will also need to talk to each other. Suitability will also still depend on your CF genotype. 

    If you have received another organ transplant and still have your CF lungs, then it would be worth talking to your transplant and CF teams about whether these treatments are available for you and whether the benefits to you outweigh the risks. Suitability will again depend on your CF genotype. 

  • When will Kaftrio be expanded to younger age groups?

    In November 2021, the Committee for Medical Products for Human Use (CHMP) gave consent for the next step in extending the rollout of Kaftrio for 6-11 year olds. This was an extension of the age group of the original application, and therefore did not have to go through the assessment process again.

    In January 2022,  the European Commission (EC) and Medicines and Health Regulatory Authority (MHRA) approved Kaftrio for eligible children aged 6-11 in England, Scotland, Wales and Northern Ireland. 

    A small clinical trial for Kaftrio in ages 2–5-year-olds is currently taking place. You can find out more about the trial on our Clinical Trials Tracker. 

    A licence extension has been granted for SYMKEVI in children with CF aged 6–11. As part of the deals negotiated by the NHS across the UK, this licence extension will automatically apply to the UK.

    We’ll continue to work hard so that people with CF of all ages and mutations have access to the best medicines.

  • What about expanding to further genetic combinations?

    The European Commission and MHRA has now formally licensed Kaftrio for use by people with CF who are:

    - 6 years and older who have at least one copy of the F508del mutation regardless of the other mutation type

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Orkambi and Symkevi

  • Are Orkambi and Symkevi available on the NHS?

    In 2019, NHS bodies across the UK reached managed access agreements making OrkambiSymkevi and Kalydeco available on the NHS to eligible adults and children with cystic fibrosis. Speak to your CF team for more information. These managed access agreements were updated in the summer of 2020 to include the triple combination therapy, Kaftrio, to the deals. Speak to your CF team for more information.

  • What must happen before I can start taking Orkambi and Symkevi?

    You may be required to take a few tests before beginning a CFTR modulator therapy. These tests may include checking your eye and liver health. The tests required will depend on your age and health, and therefore will be decided by your CF team.

  • Will I be able to stop my other medications and treatments?

    Do not stop any of your medications or treatments without first speaking to your CF team. Studies are underway and in development to explore whether these drugs can reduce the number of other treatments people with CF take.

  • Will I still have to worry about cross-infection?

    People with cystic fibrosis who have been prescribed CFTR modulators such as OrkambiSymkeviKalydeco and Kaftrio are still susceptible to infection and should be aware of and take precautions against cross-infection. These drugs are able to reduce infections that would require hospitalisation, but there is currently no evidence to suggest that they can prevent them altogether and therefore there is still a significant risk of cross-infection between people with CF and close contact should be avoided. You should speak with your CF team if you have any questions or concerns.

  • Can I take Orkambi or Symkevi if I have received a lung transplant or other organ transplant?

    Currently Orkambi and Symkevi, and other modulator therapies, are not recommended for most people who have received a transplant. This is because most of the benefits from the treatments are seen in CF lungs, and are not seen in new transplanted lungs. 

    Another reason they are not recommended is that they can come with side effects, and can also interfere with the immunosuppressant medications that people have to take after lung transplant. 

    However, if you have severe CF symptoms outside of your lungs after transplant, then it would be worth talking to both your CF team and your transplant team, as sometimes (although very rarely) it could be worth considering. There are reports of people, mainly from America, who have done so, and the majority (but not all) stopped the treatment because it caused side effects or didn’t help.

    We are also aware of reports where it seemed to cause a drop in lung function that only improved when the treatment was stopped. If you would still like to consider it, please talk to both your lung transplant team and your CF team, who will also need to talk to each other. Suitability will also still depend on your CF genotype. 

    If you have received another organ transplant and still have your CF lungs, then it would be worth talking to your transplant and CF teams about whether these treatments are available for you and whether the benefits to you outweigh the risks. Suitability will again depend on your CF genotype.

  • When will Symkevi be expanded to younger age groups?

    Symkevi is available for use in the UK for people with CF aged 12 and over with two copies of F508del or one F508del and a residual function mutation. On 18 September 2020, the EMA announced they had adopted a positive opinion to expand the eligibility of Symkevi to children from the age of six. The European Commission granted the licence extension on 27 November 2020, meaning that clinicians will now be able to prescribe Symkevi according to this expansion.

    Managed access agreements to make Symkevi available on the NHS have been agreed in England, Scotland, Wales and Northern Ireland. To find out whether you or your child might be eligible for a precision medicine, please speak to your CF team.

  • Are the deals the same in each nation?

    All the nations have equivalent agreements with Vertex Pharmaceuticals. This means that people with CF will have access to Orkambi and Symkevi while further data is collected on their efficacy The terms of the UK deals also mean that as many as 300 patients with some rarer genetic mutations, which fall outside of the scope of the European licensing body’s considerations, will now be able to benefit from Symkevi and Kalydeco.

  • I’ve read that these deals are only interim, why is that and how long are they for?

    NHS bodies across the UK have reached managed access agreements that are between two and five years. During this period, data will be collected to monitor the effectiveness of the drugs. This data will be submitted again to NICE for appraisal. This supports the process NHS England uses to decide which drugs are clinically and cost effective, while giving the opportunity for real-world data on the efficacy of the drugs to be part of the appraisal. The real-world data collection agreement will last until 2022, with a final report being prepared in June 2023 based on the previous three years of data. We expect NICE to make a recommendation based on Vertex’s evidence submission no earlier than 2023. 

    Chief Executive of NHS England, Simon Stevens, wrote a letter to Westminster’s Health and Social Care Committee setting out some details of the deal.

  • What will happen when the interim period runs out?

    People with CF will continue to have access to these drugs. As part of the interim access deals, Vertex Pharmaceuticals also agreed to a flexible contractual mechanism, which means that in all circumstances all three drugs will continue to be available following completion of the NICE and SMC appraisals. Following appraisal, new deals will be negotiated between the NHS and Vertex based on the added real-world data collected as part of this initial deal.

  • How are long-term safety and side effects being monitored?

    The UK CF Registry will be collecting real-world data from consenting people with CF as part of the agreements in the UK for Orkambi and Symkevi, to monitor effectiveness. You can read more about the Data Collection Agreement here. Any safety concerns or side effects should be reported to and discussed with your CF team.

  • Can I take part in clinical trials if I am on Orkambi, Symkevi or Kalydeco?

    Someone taking a CFTR modulator such as Orkambi, Symkevi or Kalydeco will be excluded from another CFTR modulator clinical trial, unless they are willing to stop taking their CFTR modulator for four to 12 weeks prior to entering and throughout the duration of that trial. However, they wouldn’t necessarily be excluded from other clinical trials that are trialling non-modulator therapies. The entry criteria for every trial is unique, and you should speak to your CF team if you are interested in taking part in a clinical trial.

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Compassionate use

  • What is compassionate use?

    Compassionate use provides access to medicines that are not otherwise available on the NHS to people in critical need, where attempts to treat them with licensed medicines have been exhausted or there is no appropriate licensed treatment available. Compassionate use is sometimes also called ‘expanded access’ or ‘early access’. Please speak to your CF team to find out about compassionate use eligibility.

  • Can I still be considered for compassionate use therapy through compassionate if I am already on Orkambi or Symkevi?

    If you are already receiving a CFTR modulator therapy but your health state is critical and you feel you may benefit from a different treatment, our advice is to speak to your CF team.

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Other FAQs

  • What opportunities are there to join clinical trials for CFTR modulators?

    You can find an array of information about taking part in clinical trials, as well as our very own CF Trials Tracker, a CF clinical trials database to help you find suitable clinical trial opportunities.

  • What is the Trust doing for people who will not benefit from current CFTR modulators?

    The Trust continues to fund and support ground-breaking research into innovative new medicines for all people with CF, whatever their genotype. This includes research into a range of personalised medicine approaches to treat the underlying causes of cystic fibrosis. The Trust will also support, where possible, pharmaceutical companies that aim to develop medicines that are applicable to rare CF mutations, and strive to support people with rare CF mutations to become involved in any relevant future clinical trials, via our Clinical Trials Accelerator Platform.

    More details on activities of the Trust to support research for all people with CF can be found here.

Fighting for life-saving drugs

Find out more about our campaign to ensure that people with CF across the UK can have access to innovative precision medicines.

Campaign timeline

Find out what's been happening in our campaign for life-saving drugs since it began in 2015.

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