From database to medicine cabinet: the role of the UK CF Registry in getting access to life-saving drugs

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The UK CF Registry will be taking a pivotal role in monitoring the effectiveness of Orkambi and Symkevi once they are in the hands of people with cystic fibrosis (CF) across the country. Here, Rebecca Cosgriff, Director of Data and Quality Improvement at the Cystic Fibrosis Trust, explains how the Registry will help bodies across the UK to evaluate the efficiency of these drugs, and future therapies.

It’s been an amazing two months for the cystic fibrosis (CF) community. Access arrangements for Orkambi and Symkevi have been agreed in Scotland and EnglandNorthern Ireland has committed to signing an agreement, and a deal has been agreed in principle for people in Wales. 

During the North American Cystic Fibrosis Conference (NACFC), impressive clinical trial results for Vertex Pharmaceuticals’ triple therapy, known as Trikafta in the United States, were published shortly after the drug received record-breaking early approval from the U.S. Food and Drug Administration (FDA). A submission for Trikafta to the European Medicines Agency (EMA) for a license in Europe followed hot on the heels of these results.

A solution four years in the making

There has understandably been an air of jubilation amongst people with CF, their families and CF professionals around the world. However, announcing a deal is not the same as getting drugs into the hands of people with cystic fibrosis, and we are determined to do everything possible to ensure people with CF have access to these drugs as soon as possible.

The UK CF Registry was proposed by the Trust to monitor the real-world effectiveness of CFTR modulators like Orkambi and Symkevi over four years ago. Although the CF community had to battle far too hard and for far too long to have this proposal accepted, we are delighted that a resource that has been supported by people with CF and their clinical teams for over 20 years has been central to giving decision-makers the confidence to invest in life-saving drugs for people with cystic fibrosis.

How will managed access work?

Data that is already routinely collected in the UK CF Registry – thanks to people with CF agreeing to donate their data, and to their clinical teams for entering it – will be analysed by the UK CF Registry team at the Trust to create a report that will be crucial for the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) to evaluate Orkambi and Symkevi in future.

In this way, the UK CF Registry can provide evidence on the effects of these treatments without any patient-level data being seen or accessed outside the immediate UK CF Registry team, who have expertise in handling this type of information as well as drawing on independent clinical expertise.

We have attended several meetings with NICE and the Scottish Government over the past two months to ensure that the clinical measures used are appropriate and meaningful. We are also exploring ways to ensure that quality of life can be factored into the analysis of the drugs.

Vertex will fund the resources required to produce this evidence. This will include funding to boost the Trust’s Registry Support Grant programme sufficiently to enable CF centres that choose to opt in to enter three ‘encounters’ – records of height, weight and lung function – throughout the year, so that a more robust analysis can be conducted of the impact on rate of change in lung function of these new medicines.

Backing a powerful resource

So, not only has investment of the community in the UK CF Registry helped to get our campaign for access to life-saving drugs over the line, but its involvement in regulatory processes will supercharge the amount of data held by the Registry, making it an even more powerful resource for research and quality improvement long into the future. 

The data collected as part of these access schemes will also gather vital real-world baseline data that will be fundamental to evaluating the impact of Trikafta in the future. Helping to ensure that, for future transformative medicines, life-saving drugs for all will be a reality, without a four-year battle to get us there.

You can read the full Data Collection Agreement here.

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