RECOVER (Real World Clinical Outcomes with Novel Modulator Therapy Combinations in People with CF)
Cystic Fibrosis Trust
Children's Health Ireland at Crumlin
Triple combination CFTR modulator therapy (TCMT), has shown promising results on important health outcomes in clinical trials in PWCF and has been approved in the US for use in people>12 years of age with one or more copy of F508del. Approval in Europe is pending, subsequent to which the drug will be provided for use in PWCF in Ireland as part of a pre-existing agreement with the manufacturer. Clinical trials are highly monitored and tightly controlled, and regulators understand the importance of 'real world' data to ensure they help patients as they are supposed to and are safe when widely used. We have designed a real-world study involving children and adults with CF across eight paediatric and adult CF centres in the UK and Ireland in collaboration with CF registry of Ireland and the UK CF registry. We will be examining a wide range of well-established tests in these people, as well as some newer, more sensitive tests looking at how this new treatment might affect health and quality of life in PWCF. We will be looking at how PWCF feel about this new treatment, how it affects their quality of life and whether it affects their need for, or ability to take, other CF treatments. One of the key strengths of RECOVER will be the direct comparison of a 'control' group in UK centres (who do not yet have access to treatment) with an on-treatment group in Ireland. With a point of care 'registry style' database and data management by CFRI we will be in a position to produce real time data on the effectiveness of TCMT in the real world and hopefully, convince reimbursement agencies of the benefits of therapy.
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