RT Journal Article
SR Electronic
T1 Projecting the impact of triple CFTR modulator therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials
JF Thorax
JO Thorax
FD BMJ Publishing Group Ltd and British Thoracic Society
SP 873
OP 881
DO 10.1136/thoraxjnl-2020-216265
VO 77
IS 9
A1 Ruth H Keogh
A1 Rebecca Cosgriff
A1 Eleni-Rosalina Andrinopoulou
A1 Keith G Brownlee
A1 Siobhán B Carr
A1 Karla Diaz-Ordaz
A1 Emily Granger
A1 Nicholas P Jewell
A1 Alex Lewin
A1 Clemence Leyrat
A1 Daniela K Schlüter
A1 Maarten van Smeden
A1 Rhonda D Szczesniak
A1 Gary J Connett
YR 2022
UL http://thorax.bmj.com/content/77/9/873.abstract
AB Background Cystic fibrosis (CF) is a life-threatening genetic disease, affecting around 10 500 people in the UK. Precision medicines have been developed to treat specific CF-gene mutations. The newest, elexacaftor/tezacaftor/ivacaftor (ELEX/TEZ/IVA), has been found to be highly effective in randomised controlled trials (RCTs) and became available to a large proportion of UK CF patients in 2020. Understanding the potential health economic impacts of ELEX/TEZ/IVA is vital to planning service provision.Methods We combined observational UK CF Registry data with RCT results to project the impact of ELEX/TEZ/IVA on total days of intravenous (IV) antibiotic treatment at a population level. Registry data from 2015 to 2017 were used to develop prediction models for IV days over a 1-year period using several predictors, and to estimate 1-year population total IV days based on standards of care pre-ELEX/TEZ/IVA. We considered two approaches to imposing the impact of ELEX/TEZ/IVA on projected outcomes using effect estimates from RCTs: approach 1 based on effect estimates on FEV1% and approach 2 based on effect estimates on exacerbation rate.Results ELEX/TEZ/IVA is expected to result in significant reductions in population-level requirements for IV antibiotics of 16.1% (~17 800 days) using approach 1 and 43.6% (~39 500 days) using approach 2. The two approaches require different assumptions. Increased understanding of the mechanisms through which ELEX/TEZ/IVA acts on these outcomes would enable further refinements to our projections.Conclusions This work contributes to increased understanding of the changing healthcare needs of people with CF and illustrates how Registry data can be used in combination with RCT evidence to estimate population-level treatment impacts.Data may be obtained from a third party and are not publicly available. This work used anonymised data from the UK Cystic Fibrosis Registry, which has Research Ethics Approval (REC Ref: 07/Q0104/2). The use of the data was approved by the Registry Research Committee (Data Request Reference 382). Data are available following application to the Registry Research Committee (https://www.cysticfibrosis.org.uk/the-work-we-do/uk-cf-registry/apply-for-data-from-the-uk-cf-registry).