The independent appraisal committee concluded that the cost of Orkambi was considerably higher than the current standard of care and it could not be considered a cost effective use of NHS resources.
Cystic fibrosis is an inherited condition where a gene defect causes a person’s lungs and digestive system to become clogged with sticky mucus. This leads to blockages that damage the body’s tubes and passages. Symptoms include persistent cough, recurring infections and malnutrition.
There is currently no cure for cystic fibrosis and current treatments aim to manage individual symptoms.
Orkambi is licensed to treat people who have a specific genetic defect known as the F508del mutation. There are around 2,750 people in England with this genotype. It costs £104,000 per patient for every year of treatment. Most people suitable for Orkambi would also still remain on the current standard of care whilst taking it.
The committee acknowledged that Orkambi does offer benefit because it reduces instances where people experience a sudden worsening of symptoms that requires hospitalisation. However, the benefits to lung function – the test to see how people with cystic fibrosis are improving overall – were modest.
Commenting on the draft guidance, Professor Carole Longson, director of the NICE Centre for Health Technology Evaluation said: Orkambi is a new treatment option and it is disappointing that we are not able to recommend it. However our independent committee found that when compared to the current standard of care, the benefit it offered was modest and comes at a considerable cost. We have to recognise that the NHS is a finite resource and we can only recommend treatments for routine funding that are both clinically effective and represent good value for money.”
Consultees, healthcare professionals and members of the public, are now able to comment on the preliminary recommendations which are available for public consultation until Friday 15 April 2016. Comments received during this consultation will be considered by the committee at a further appraisal meeting before the next draft guidance is issued.
Until final guidance is issued, NHS organisations should make decisions locally on the funding of specific treatments.
Filed Under: Drug Discovery