NICE recommends new drug for adults with cystic fibrosis in final draft guidance
In final draft recommendations published today (26 October), NICE has recommended mannitol dry powder (Bronchitol, Pharmaxis) as an option for treating cystic fibrosis in some adults. The draft guidance, which is now with consultees who have the opportunity to appeal against it, recommends mannitol as an option for treating cystic fibrosis in adults who:
- do not use inhaled mucolyticsi because they cannot take rhDNaseii and for whom other osmotic agents like hypertonic saline are not considered appropriate and,
- whose lung function is rapidly declining (forced expiratory volume in 1 second [FEV1] decline greater than 2%
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.
The aim of treatment in adults with cystic fibrosis is to clear the respiratory secretions in order to maintain lung function, as well as to reduce inflammation and bacterial infection in the respiratory tract. Treatment includes regular physiotherapy, antibiotics, and use of a nebuliser.
Mannitol dry powder is inhaled with a hand-held, breath activated device. It 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.
Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: “The Committee heard from the patient expert and clinical specialists that cystic fibrosis has a major impact on the quality of life of patients and their carers, and that mannitol could ease some of this burden because it is a dry powder for inhalation, is associated with fewer unpleasant effects, needs less costly equipment and needs less time to administer than nebulised treatments. However, the previous draft guidance 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.
Professor Longson continued: “Following the helpful responses received during consultation on the draft guidance, the Committee concluded that mannitol was a good use of NHS resources when treatment was offered to those who cannot use hypertonic saline or rhDNase and whose lung function is rapidly declining.”
The draft guidance is now with consultees, who have the opportunity to appeal against it. 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 November 2012.
Notes to editors
References and explanation of terms
i. Inhaled mucolytics are types of medicines which break up, dissolve and liquefy mucus.
ii. rhDNase or daily recombinant human deoxyribonuclease is a treatment that decrease the thickness and density of sputum and aids secretion clearance.
About the guidance
1. The final appraisal determination (FAD) can be found from Friday 26 October on the NICE website. Closing date for appeals is Friday 9 November.
2. 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.
3. 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.
4. Mannitol is available as a 40 mg powder capsule for inhalation. The list price for a 14-day pack of 280 capsules and 2 inhalers is £231.66 (excluding VAT; ‘Monthly Index of Medical Specialities' [MIMS] September 2012). This equates to £0.83 per 40 mg capsule, or an average cost of £16.55 per day, including the cost of the inhaler. These prices do not include VAT. Costs may vary in different settings because of negotiated procurement discounts.
5. The Committee noted that if mannitol treatment was offered only to patients with a rapid decline in lung function, the ICER would most likely be lower than in the whole population because of this group's lower quality of life and lung function, and a greater potential to improve. The Committee concluded that the ICER would be less than £30,000 per QALY gained for patients who cannot use hypertonic saline or rhDNase and whose lung function is rapidly declining, bringing the estimate of cost effectiveness within a range that could be considered an acceptable use of NHS resources.
6. A patient access scheme has not been submitted by the manufacturer.
7. Mannitol has not yet been appraised by the Scottish Medicines Consortium.
Related NICE guidance
8. There is no related guidance for this appraisal.
9. The National Institute for Health and Clinical 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.
10. 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.
11. 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.
12. 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: 26 October 2012