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Access to medicines FAQs

Choose from the frequently asked questions below to find out more about precision medicines and the work we're doing to stop the clock on cystic fibrosis.

About precision medicines

What are precision medicines?

People with CF have two copies of a mutation of the gene that causes cystic fibrosis. 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 bodies 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 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.

How are drugs appraised by the EMA?

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

The European Medicines Agency (EMA) grants this licence, which is called Marketing Authorisation, 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.

Orkambi and Symkevi

When will Orkambi and Symkevi be available to people with CF?

NHS bodies across the UK have reached managed access agreements making Orkambi, Symkevi and Kalydeco available on the NHS to eligible adults and children with cystic fibrosis. 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 OrkambiSymkevi or Kalydeco. 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.

I am still waiting to hear when I can start Orkambi and Symkevi – why is this?

Cystic fibrosis teams can now prescribe Orkambi and Symkevi, but this does not mean you will necessarily be started on it straight away. All CF centres are working to get eligible patients on to these drugs as soon as clinically possible. If you are concerned about timescales you should contact them for an update.

What is the Trust doing to speed up the roll out?

CF centres are doing everything they can to roll out Orkambi and Symkevias fast as clinically possible. Centres need to undertake testing and monitoring when placing a patient on Orkambi or Symkevi and this can slow the rollout.

The Trust has worked closely with the NHS and the UK CF Medical Association to support CF teams with information.

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 OrkambiSymkevi and Kalydeco 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?

It is currently not recommended that any CFTR modulators are taken by anyone who has had a transplant, as the effects of these drugs for transplant patients has not been studied. Studies are in development to explore potential use of these drugs in people who have had an organ transplant.

When will Symkevi be expanded to younger age groups?

Vertex announced positive clinical trial results in early 2019 for the use of Symkevi in people with CF aged six to 11. They intend to use these results in an application to the EMA to expand the eligibility of Symkevi for people in the EU. The EMA will have to approve the age expansion before Symkevi becomes available to people aged six to 11 in the UK.

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 whilst further data is collected on their efficacy.

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 for 18 months, prior to a NICE appraisal that is expected to conclude in September 2021 to inform a new agreement between the NHS and Vertex.

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 appraisal. Following the NICE appraisal, a new deal will be negotiated between NHS England and Vertex based on the added real-world data collected as part of this initial deal.

What happens if I am already in receipt of Orkambi or Symkevi through compassionate use or as part of a clinical trial?

All individuals in England currently receiving one of these drugs, either through compassionate use, or as part of a clinical trial, will continue to do so under the new agreement with Vertex Pharmaceuticals.

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.

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 will be eligible for Kaftrio?

The European Medicines Agency (EMA) have announced a positive opinion for Kaftrio for use by people with CF who:

  • are aged over 12
  • have two copies of the F508del mutation or one copy of F508del and one copy of a 'minimal function’ mutation. The details of which precise genotypes the latter group covers is to be confirmed.

This decision by the EMA is different to the one made by the FDA in America, who allow the drug to be prescribed to people over 12 who have at least one copy of the F508del 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.

However, the NHS England deal allows clinicians to prescribe Vertex drugs for certain patients with rare mutations, that are covered by the U.S. Food & Drug Administration’s licensing decisions. We are still awaiting the fine details of the deal to understand exactly who is eligible and will update our website and social media pages when this is clear.

What will be included in the minimal functions list?

We will not know this detail until the licence is agreed by the EMA. We are working hard behind the scenes to let relevant bodies know your concerns and advocate for the CF community. We know how hard and frustrating this uncertainty will be for some people and we assure you we will let you know when we have any updates. We’re aware there are some lists of mutations circulating but these are not official/definitive lists. We know there are a lot of questions around the recent deal in England for Kaftrio and we recognise how important it is that the CF community has clarity on which ‘minimal function mutations’ are included in the agreement.

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.

When will Kaftrio be available?

Following the EMA’s positive opinion on 26 June 2020, there is now a period of up to 67 days for scrutiny before the drug can be granted a licence and made available in Europe.

In England, NHS England announced a deal on 30 June 2020 with Vertex to facilitate access from the first day the European licence is granted. The deal with NHS England includes 'tag along rights' for the other devolved nations and we hope it will pave the way for similar deals across the UK. It is great to see NHS England supporting their colleagues in the devolved administrations, and we are seeking more information and updates about negotiations and access in Wales, Scotland, Northern Ireland, and the crown dependencies of Jersey, Guernsey, and the Isle of Man.

When CF teams can prescribe Kaftrio, it will take time for teams to organise and roll-out Kaftrio. The Trust will work closely with the UK CF Medical Association to support roll-out and ensure everyone can start Kaftrio as fast as clinically possible. 

What is the Trust doing to support access to Kaftrio?

We are working with all key stakeholders to support access for everyone in the UK who could benefit. In the first instance, that is seeking more information and updates about negotiations and access in Wales, Scotland, Northern Ireland, and the crown dependencies of Jersey, Guernsey, and the Isle of Man. We are also working to understand the mutations covered in the NHS England deal and what flexibility clinicians will have in prescribing Kaftrio, Symkevi, and Orkambi for people with rare or other mutations.

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

We also support 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.

At least three companies, including AbbVie, Proteostasis Therapeutics 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 are ineligible for Kaftrio.

We are here for everyone with cystic fibrosis and we know there is more to do to ensure everyone with CF has access to life-saving drugs. Read more about the Trust’s commitment to no-one left behind.

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?

It is currently not recommended that any CFTR modulators are taken by anyone who has had a transplant, as the effects of these drugs for transplant patients has not been studied. Studies are in development to explore potential use of these drugs in people who have had an organ transplant.

When will Kaftrio be expanded to younger age groups?

Phase III clinical trials testing the effects and safety of Kaftrio in 6–11 year olds are currently at different stages of the process, with one expected to complete in August of this year. We are not in a position to predict the timings for submitting trial data for regulatory approval, but Vertex have stated they intend to submit an extension application to the FDA later this year, followed by regulatory submissions in other countries. It is likely that a submission for an extension to the EMA will be after the FDA, with a decision being made in 2021.

In addition to Kaftrio, Vertex announced positive clinical trial results in early 2019 for the use of Symkevi in people with CF aged six to 11. They have since submitted an extension request to the EMA and is under active review, with a decision expected in the coming months.

What about expanding to further genetic combinations?

There is a currently an ongoing phase III clinical trial testing the safety and effectiveness of Kaftrio for people with one copy of F508del and one copy of either a residual function or gating mutation. The trial is expected to complete in October of this year and a regulatory submission to the EMA will follow to provide a licence extension to these genetic combinations. We expect the EMA to make a decision in 2021.

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.

When will the triple combination therapy be available on the NHS?

The triple combination therapy (elexacaftor, tezacaftor and ivacaftor, called Trikafta in the United States) is not currently licensed for use in Europe, and is currently being reviewed by the European Medicines Agency for safety. Our top priority is working with Vertex and the NHS to ensure the triple combination therapy is made available as soon as possible after licensing to people with cystic fibrosis.

What is the Cystic Fibrosis Trust doing?

The Cystic Fibrosis Trust is working closely with clinicians and Vertex to ensure that the compassionate use scheme for triple combination therapy is running efficiently and smoothly. Please speak to your CF clinical team for advice about eligibility for the scheme.



Stopping the Clock

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