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Cystic Fibrosis Trust

After a clinical trial

Here you can find out more about what should happen once you have finished taking part in a cystic fibrosis clinical trial.



What happens after I've taken part in a clinical trial?

Depending on what stage of the trial you joined, it is likely that the trial will continue even when you have stopped participating. Most participants want to know what the outcome of the trial has been - just keep in mind that it could be a significant length of time (sometimes a year or two) after your participation has ended before results are available.

How can I track the progress of a clinical trial?

At the end of the trial, a summary of the results should be made available to everyone who took part. Results will also be published so that others can use the information to help them make decisions about treatment and healthcare - this will be in a scientific journal, and possibly at a conference too.

Once results have been published, we will endeavour to make these available via our Trials Tracker.

  

Where a trial doesn’t have the predicted outcome, for example if the intervention is proved to be ineffective, or not effective enough to proceed, the trial has still been worthwhile. Without conducting trials, questions about safety and efficacy of potential new treatments would go unanswered, and potentially valuable treatments would never reach patients.

All trial results are building blocks in our search for the best treatment and care, so a disappointing outcome in one trial is still a successful study!

If you have taken part in a clinical trial, but haven’t been kept up to date on outcomes, speak to your CF team.

Will I be eligible to stay on the treatment?

Possibly, but there may be a gap between when the trial finishes and when you can continue to take the treatment. Where a treatment has been shown to be effective, research teams, pharmaceutical companies and patient organisations will all be keen to see the treatment made available as quickly as possible.

In many trials of new drugs, ‘rollover’ studies are conducted at the end of the initial trial. A rollover study is generally used to continue to observe a treatment’s effect over a longer period and can allow people to remain on a new treatment.

However, even once a treatment has been shown to be effective there can be a significant wait as the NHS, pharmaceutical companies and other organisations negotiate on the price of the treatment. In this case you will continue to be given your standard treatment.

In some situations, while negotiations are underway to bring a new treatment to patients, doctors may be able to prescribe the treatment to certain patients via an 'expanded access programme' (also known as a 'patient access scheme' (PAS) or 'compassionate use' scheme), in which the pharmaceutical company makes the medicine available on a case by case basis. Such programmes usually involve strict eligibility criteria, and the doctor prescribing the medicine would have to take full responsibility for prescribing the treatment for their patient.

In an expanded access programme, patients are usually followed up in the same way as if they were on a clinical trial. Because the medicines in question are unlicensed, and therefore there may not be detailed information available on their safety and efficacy, they are usually limited to patients for whom conventional treatment options are no longer effective, and who are often very unwell.

The Cystic Fibrosis Trust is currently campaigning to make sure that ground-breaking treatments are made available to the people with CF who are eligible for them. Find out more about our Stopping the Clock campaign.

How are new treatments licensed and approved?

Licensing

Licensing means granting legal approval for a drug to be made available to patients (also known as ‘marketing authorisation’). The process of how drugs are licensed varies depending on whether the drug is intended for use in the UK or Europe.

For a UK license only, drugs are licensed through the Medicines and Healthcare Products Regulatory Agency (MHRA). For a European license, drugs are licensed through the European Medicines Agency (EMA), legislated by the European Commission.

Approval 

Once a drug has been licensed for use, it can legally be prescribed for patients. However, this does not mean it automatically becomes available to all who could benefit. Before a drug can be made available on the NHS in England and Wales, it must be reviewed by the National Institute for Health and Care Excellence (NICE), an independent Government organisation. In Wales, the All Wales Medicine Strategy Group makes some decisions, but generally follows NICE recommendations. In Northern Ireland, the Department of Health, Social Services and Public Safety (HPSS) makes decisions on new medicines, but usually follows NICE guidance. In Scotland, the Scottish Medicines Consortium (SMC) makes the overall decisions.

NICE bases its decision on how well a treatment works, and also, whether it represents value for money. If it is felt that a treatment does not offer enough benefit to patients to warrant the cost, it may recommend against making the treatment available on the NHS on a cost-benefit basis.

When this happens, the drug manufacturers, patient organisations and the Government may need to come together for negotiations, to agree a way to make the treatment available to those who could benefit. This all means that it can take some time even after a drug is licensed, before it is made available to patients.

Taking part

Find out more about taking part in a clinical trial. We've got information on everything from trial types to eligibility and consent as well as links to loads of useful resources.

Find out more

Trials Tracker

We have created a brand new CF Trials Tracker so that you can easily access the trials that relate to you. Search by genotype or trial type, and save your favourites!

Explore trials

Clinical trials booklet

Our clinical trials booklet is full of useful information from our webpages on taking part in clinical trials.

Download the PDF