Project

A registry-based trial investigating therapy rationalisation in people with cystic fibrosis (the CF STORM study) - HEP06

Full project title: A randomised registry-based open-label study to assess change in respiratory function for people with cystic fibrosis (pwCF) with one or two Phe508del variants established on triple CFTR modulator combination therapy after rationalisation of muco active aerosolised therapies (the CF STORM study).

Summary

CF STORM is a ground-breaking trial to find out if people with cystic fibrosis (CF) receiving the drug Kaftrio can safely start to reduce the number of treatments they have to manage as part of their daily healthcare routine. 

Background

Cystic fibrosis (CF) affects over 10,000 people in the UK, and the median life expectancy for a person in the UK with cystic fibrosis is around 49 years. Feedback from the cystic fibrosis community has indicated that reducing the treatment burden is considered to be their top research priority. People with cystic fibrosis spend many hours every day on multiple treatments, including aerosolised therapies delivered by nebulisers to keep their lungs stable and prevent chest infections.

CF STORM, one of the first trials of its kind, has been designed to address the question of how to safely reduce the treatment burden. The recent introduction in the UK of a highly effective new drug, Kaftrio, which is suitable for most people with cystic fibrosis, provides an ideal opportunity to robustly explore this question. 

CF STORM will investigate whether it is safe to stop so-called mucoactive nebulisers (DNase and hypertonic saline) once eligible people with cystic fibrosis are established on Kaftrio. This could save people valuable time each day that is currently spent prepping, carrying out and cleaning nebulisers, which may have a positive impact on quality of life.  

Members of the Health Economics and Prioritisation in Health and Social Care theme will be conducting an analysis of the cost-effectiveness of this strategy alongside the trial.

Project aims

The primary aim of the clinical trial is to establish whether patients established on Kaftrio can safely stop taking nebulised active drugs without a significant fall in their respiratory function.

The aim of the health economic component of the study is to establish the cost-effectiveness of this strategy.

Project activity 

The study opened recruitment in July 2021, it expected that patient follow-up will continue into late 2023.

The potential or actual impact 

At the end of the trial, a knowledge transfer activity will place the trial results in context, both nationally and internationally. The trial outcome will be published in an open-access peer-reviewed journal. In addition, the innovative approach taken to assess a 'stopping strategy' in the context of undertaking a pragmatic registry-based study following the introduction of highly effective and costly therapy will also be published.

This approach is likely to be translatable to other conditions where new transformational therapies are becoming available. It is important that the impact of new, high-cost drugs on the patient journey is assessed rigorously.

A patient-centred knowledge transfer exercise will also be undertaken in October 2023 and will immediately inform people with cystic fibrosis of the implications of the results of CF STORM for their individual patient journey. The main 'take-home messages' will be distributed through the CF Trust framework, including their social media platforms.

Recent activity 

Developing data management and data management plan, drafting a health economics analysis plan.

Next step 

Finalising the health economic analysis plan.

Related projects

HEP02 - Evidence-based valuation of patient-centred outcomes in Cystic Fibrosis

Who is involved?

Co-Principal Investigator: Prof. Kevin Southern, Professor of Child Health, University of Liverpool (K.W.Southern@liverpool.ac.uk

Dr Gwyneth Davies, Clinical Lecturer, University College London (gwyneth.davies@ucl.ac.uk)

Researchers and Institutions 

Health economics lead: Professor Jennifer Whitty, Senior Research Scientist, Evidera 

Health economist: Dr Rory Cameron, Researcher, ARC East of England Health Economics and Prioritisation (HEP) theme / University of East Anglia (Rory.Cameron@uea.ac.uk

Host organisation: Alder Hey Children’s NHS Foundation Trust

Collaborating institutions: Cardiff University

Imperial College London

Liverpool Clinical Trials Centre

Oxford University Hospitals NHS Foundation Trust

Papworth Hospital NHS Foundation Trust

Queen's University Belfast

Royal Brompton and Harefield NHS Foundation Trust

UK Cystic Fibrosis Trust 

University College London

University of East Anglia

University of Manchester

University of Nottingham

Contacts 

Trial Lead:  Dr Gwyneth Davies, Clinical Lecturer, University College London (gwyneth.davies@ucl.ac.uk)

Health Economics: Dr Rory Cameron, Researcher, ARC East of England Health Economics and Prioritisation (HEP) theme / University of East Anglia (Rory.Cameron@uea.ac.uk)