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P21 Prescribing lvacaftor in paediatric patients with cystic fibrosis (cf) in accordance with the clinical commissioning policy in an nhs trust
  1. McGeehan Eimear
  1. Bristol Royal Hospital for Children


Introduction CF is a genetic condition affecting more than 10 800 people in the UK. CF is caused by a mutation in the gene cystic fibrosis trans membrane conductance regulator (CFTR).Prior to the licensing of Ivacaftor, standard treatment for CF was to treat symptoms associated with CF but not the underlying cause. Ivacaftor targets the CFTR gene. Ivacaftor is funded by NHS England, if criteria outlined in the clinical commissioning policy is followed.1,2

Aim Ensure Ivacaftor is prescribed in adherence to guidance documented in the Clinical Commissioning Policy: Ivacaftor for CF (2012) and Clinical Commissioning Policy: Ivacaftor for children aged 2–5 years with CF, named mutations (2016).1,2

Standards 100% of Ivacaftor prescriptions will be for patients:

  • 2 years of age or older

  • Have a G55ID mutation

  • 100% of patients will receive lung function test (6 years and older) and baseline sweat test 6 months prior to commencing treatment

100% of patients will receive a follow up sweat test/lung function test (6 years and older) at:

  • Next routine appointment

  • 6 months after starting treatment

  • Annually thereafter

  • 100% of patients who don’t attain an adequate treatment response will discontinue Ivacaftor

Method Retrospective study investigated the prescribing of Ivacaftor in CF patients from March 2012 – June 2017 at an NHS trust. Ethics approval not required. List of patients prescribed Ivacaftor was obtained from the CF team. Patient age, mutation type, treatment start dates, lung function test results were obtained from medical notes. Dates and results of sweat tests were obtained from Sunquest ICE Desktop (electronic patient reporting system). Data analysed using Microsoft excel.

Results Eight patients prescribed Ivacaftor at the NHS trust between March 2012–June 2017. Baseline sweat chloride data unavailable for one patient who was previously part of a clinical trial. This patient was excluded from standard 2, however maintained for the other standards as his annual sweat data was available. One patient was excluded from standard 3(a), five patients excluded from standard 3 (b), (c) as they had not yet reached this stage of treatment. Standard 4 was not evaluated as all patients to date were responding to treatment. Overall, all standards were completely met with a result of 100%.

Discussion and conclusion Standards were completely met; highlighting a robust system ensuring all appropriate testing is adhered to, as failure to comply with the criteria in the clinical commissioning policy may contribute to pressure within the trust’s budget. Treatment response can also be appropriately determined.


  • Ensure data is inputted onto the system electronically.

  • CF pharmacist to re–audit data yearly to ensure the clinical commissioning policy is being adhered to.


  1. NHS Commissioning Board. Clinical commissioning policy: Ivacaftor for cystic fibrosis 2012. https://www. [Accessed: 18th July 2017].

  2. NHS Commissioning Policy: Ivacaftor for children aged 2 5 years with cystic fibrosis (named mutations) 2016. [Accessed: 18th July 2017].

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