NICE consults on mannitol for cystic fibrosis
In preliminary draft recommendations published today (1 June) for consultation, NICE has not recommended mannitol dry powder (Bronchitol, Pharmaxis) for the treatment of cystic fibrosis.
The independent Appraisal Committee concluded that mannitol could not be recommended because of gaps and uncertainties in the evidence on its effectiveness compared with other treatments currently used in the UK and uncertainties about the long term effect of mannitol on lung function. The Committee also concluded that the analysis provided by the manufacturer was not a true reflection of clinical practice or the efficacy of mannitol.
Mannitol dry powder for inhalation acts by inducing an influx of water into the airway, improving hydration of airway secretions, and increasing mucus clearance by making it less thick and sticky and stimulating a cough. Mannitol dry powder is administered by inhalation with a hand-held, breath activated device.
Cystic fibrosis is one of the UK's most common life-threatening inherited diseases, and currently affects around 8,000 people. Over two million people in the UK carry the faulty gene that causes cystic fibrosis - around 1 in 25 of the population. For a baby to be born with cystic fibrosis, both parents must be carriers of the faulty gene. If two carriers have a child, the baby has a 1 in 4 chance of having cystic fibrosis. The condition affects the internal organs, especially the lungs and digestive system, by clogging them with thick, sticky secretions, making it hard to breathe and digest food. Symptoms of cystic fibrosis can include a troublesome cough, repeated chest infections, prolonged diarrhoea and poor weight maintenance. The condition can also lead to pulmonary disease as well as cystic fibrosis related diabetes (CFRD), male infertility and it can cause the blockage of small ducts in the liver. At present, there is no cure. Management of the pulmonary aspect of the condition includes a number of measures to help clear the respiratory secretions and to reduce inflammation and bacterial growth in the respiratory tract. This includes regular physiotherapy, antibiotics, and use of a nebuliser.
Dr Carole Longson, Health Technology Evaluation Centre Director at NICE said: "The independent Appraisal Committee recognises the significant effect that cystic fibrosis can have on everyday life. However, in order for NICE to recommend any drug or technology, we have to be certain that it is both clinically and cost effective. The evidence presented for mannitol did not support a positive recommendation for this condition. This draft recommendation is now available for consultation on the NICE website, and the manufacturer and other consultees have the opportunity to respond to concerns and comments made by the Appraisal Committee."
NICE has not yet issued final guidance to the NHS; these decisions may change after consultation.
Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its guidance on a technology it replaces local recommendations across the country.
Final guidance is expected to be published in August 2012.
Notes to Editors
About the guidance
1. The appraisal consultation document (ACD) can be found from Friday 1 June on the NICE website at: http://guidance.nice.org.uk/TA/Wave21/9
Embargoed copies are available on request; please contact the press office.
Closing date for comments is Tuesday 3 July.
2. After consultation the Appraisal Committee will meet again to consider the evidence, the appraisal consultation document and comments from the consultees.
3. After considering these comments, the Committee will prepare the final appraisal determination (FAD). Subject to any appeal of the FAD by consultees, NICE will produce final guidance on the use of mannitol in the NHS in England and Wales. For further details on appraisals, see the NICE website.
4. Mannitol dry powder for inhalation (Bronchitol, Pharmaxis) is a mucoactive agent that acts by inducing an influx of water into the airway lumen improving hydration of airway secretions, and increasing mucociliary clearance by reducing its viscosity and stimulating cough. Mannitol dry powder is administered by inhalation with a hand-held, breath activated device.
5. Mannitol has a marketing authorisation as an add-on therapy to best standard of care in adults with cystic fibrosis. The dose used in clinical trials was ten 40-mg capsules inhaled twice daily, giving a cumulative dose of 400 mg twice daily.
6. The list price given in the manufacturer's submission is £0.84 per 40-mg capsule, or an average cost of £16.88 per day, including the cost of the inhaler. The manufacturer's submission quoted a cost for a 14-day pack of 280 capsules and two inhalers of £236.25. These prices do not include VAT. Costs may vary in different settings because of negotiated procurement discounts.
7. The manufacturer's submission presented Incremental Cost Effectiveness Ratios (ICERs) of more than £30,000 per QALY. However, the Committee considered there was considerable uncertainty regarding the robustness of the model and concluded that using more plausible assumptions in the modelling would increase the ICERs even more.
8. A patient access scheme has not been submitted by the manufacturer.
9. Mannitol has not yet been appraised by the Scottish Medicines Consortium.
Related NICE guidance
10. There is no related guidance for this appraisal.
11. The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health.
12. NICE produces guidance in three areas of health:
- public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
- health technologies - guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
- clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.
13. NICE produces standards for patient care:
- quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
- Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients.
14. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.
This page was last updated: 01 June 2012