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Cystic Fibrosis Trust

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.

Drug appraisal, assessment and licensing 

Precision medicines

Stopping the Clock – lobbying for access to medicine

Drug appraisal, assessment and licensing  

How are drugs appraised?

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 license, 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 license. Find out more about how drugs are tested in clinical trials.

How are drugs assessed for funding?

Once a new treatment has a license, drug appraisal bodies such as the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) weigh up the price a company has asked for against the clinical trial data. They then recommend whether the drug will be cost-effective for the NHS and the taxpayer.

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 does drug appraisal work where I live?

There are different appraisal bodies across the UK

  • England - National Institute for Health & Care Excellence (NICE)
  • Wales - All Wales Medicines Strategy Group (AWMSG). The AWMSG follows all NICE advice but if NICE is not due to assess a medication within 12 months of it getting a licence, AWMSG may run its own appraisal.
  • Northern Ireland – NICE/Department of Health and Social Care (DHSC). The DHSC endorses NICE recommendations following local review by Northern Irish commissioners.
  • Scotland - Scottish Medicines Consortium (SMC)

What was the Accelerated Access Review (AAR) and how does it affect access to medicines?

The AAR was an influential government report that suggested changes to the way new drugs are appraised and negotiations over pricing conducted. The AAR was the subject of a debate held on behalf of the Trust at Westminster in December 2016, and the Government issued its response to the Review in November 2017. One of the main outcomes was to establish a new Strategic Commercial Unit within NHS England, which will aim to negotiate for fair and sustainable prices for new medicines. Take a look at our campaign timeline for more information on the work we have been doing over the last few years to promote access to medicines for people with cystic fibrosis.

Precision medicines

What is precision medicine?

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 Kalydeco?

At the moment, ivacaftor (Kalydeco) is the only precision medicine available on the NHS in the UK. It is available for everyone over the age of two with one of nine rare gating mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D). Find out more about Kalydeco.

What does Kalydeco do?

Kalydeco helps open the ‘gate’ in the cells of people with CF, allowing chloride molecules to move in and out of the cells, and keep the balance of salt and water in the lungs. Drug companies sometimes refer to Kalydeco as a ‘potentiator’, for more information on what this means, see below. 

What is Orkambi?

Orkambi is what’s known as a double-combination therapy – it combines ivacaftor with another compound called lumacaftor. Orkambi works for people with two copies of the F508del mutation – around 50% of people with CF in the UK – and is licenced for those age six years and over. 

Orkambi is licenced by the EMA but it is not available on the NHS, and is only available on compassionate grounds, where the manufacturer provides it to individuals on a case-by-case basis, who fulfil a number of criteria. Find out more about Orkambi.

What does Orkambi do?

Orkambi has been shown to slow lung decline – the main cause of death in CF – by 42%, and to reduce the number of nasty chest infections which require inpatient hospital care to treat.

What is tezacaftor?

Tezacaftor is a compound that is being tested in combination with ivacaftor in a double-combination therapy.

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.

triple therapy from Vertex began Phase III clinical trials in March 2018.

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.

Stopping the Clock – lobbying for access to medicine

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.

Why do you call your campaign ‘Stopping the Clock’?

Cystic fibrosis is a life-shortening condition – the clock is always ticking for those born with it. New treatments in CF, which work at the root cause of the condition, may help slow and, in the future, put a stop to the progress of the disease. We see every step forward as a step towards stopping the clock on cystic fibrosis.

Why are we still waiting for access to Orkambi?

It’s been over two years since the EMA gave a licence to Orkambi for people age 12 and over with two copies of the F508del gene. The drug’s manufacturer, Vertex, has yet to agree a deal with health decision makers and politicians over what constitutes value for money in terms of all the other things that the NHS has to pay for.


Stopping the Clock

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

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