Quality statement 4: Choice of mucoactive agent

Quality statement

People with cystic fibrosis who have clinical evidence of lung disease are prescribed rhDNase[2] as the first choice of mucoactive agent.

Rationale

Sticky mucus accumulates in the lungs in people with cystic fibrosis, making them more prone to infection. Repeated infection can cause permanent damage to the lungs. Airway clearance techniques are used to loosen and remove excess sticky mucus. The mucoactive agent rhDNase thins the mucus, making it easier to clear from the lungs. This helps to maintain lung function and prevent infection.

Quality measures

Structure

Evidence of the availability of devices for people with cystic fibrosis to take inhaled rhDNase.

Data source: Local data collection, for example from local protocols. NHS England service specifications for cystic fibrosis require a comprehensive nebuliser service to provide devices that deliver drugs in a fast and efficient manner.

Process

Proportion of people with cystic fibrosis and clinical evidence of lung disease who are prescribed rhDNase as the first choice of mucoactive agent.

Numerator – the number in the denominator prescribed rhDNase as the first choice of mucoactive agent.

Denominator – the number of people with cystic fibrosis and clinical evidence of lung disease.

Data source: Local data collection, for example, local audit of patient records. The UK Cystic Fibrosis Registry annual data report presents the proportion of patients with cystic fibrosis on DNase.

Outcome

Lung function (forced expiratory volume in 1 second [FEV1]) of people with cystic fibrosis.

Data source: Local data collection, for example local audit of patient records. Appendix 2 in the UK Cystic Fibrosis Registry annual data report includes FEV1 converted from absolute litres of air blown out into a predicted percentage. The predicted percentage is based on the FEV1 expected for a person without cystic fibrosis of the same age, gender, height and ethnicity.

What the quality statement means for different audiences

Service providers (such as cystic fibrosis centres) ensure that specialist cystic fibrosis multidisciplinary teams have systems in place, such as prescribing protocols or prescribing decision support systems, to offer rhDNase as the first-choice mucoactive agent and to monitor the effects of the treatment.

Healthcare professionals (such as cystic fibrosis specialist paediatricians or adult physicians) offer rhDNase as the first-choice mucoactive agent to people with cystic fibrosis who have lung disease and identify an appropriate delivery device. They discuss the treatment and the importance of adherence with the child, young person or adult with cystic fibrosis and their family or carer. They assess the effects of, and tolerance to, the treatment once it has started.

Commissioners (NHS England) ensure that service specifications require rhDNase to be prescribed as the first-choice mucoactive agent to people with cystic fibrosis who have clinical evidence of lung disease.

People with cystic fibrosis who have lung disease have a medicine called rhDNase, or dornase alfa, as their first treatment to help with breathing. The medicine is inhaled (breathed in). It makes mucus in the lungs less thick and sticky, so it is easier to cough out. This medicine also makes lung infections less likely.

Source guidance

Cystic fibrosis: diagnosis and management (2017) NICE guideline NG78, recommendations 1.6.17 and 1.6.18

Definitions of terms used in this quality statement

Clinical evidence of lung disease

Evidence of lung disease based on radiological imaging or lung function testing.

[NICE's guideline on cystic fibrosis: diagnosis and management, full guideline]

rhDNase

Recombinant human deoxyribonuclease; dornase alfa[2]. A mucoactive agent.

[NICE's guideline on cystic fibrosis: diagnosis and management, full guideline]

Mucoactive agent

A drug that affects the viscosity of mucus, usually given to make the removal of mucus through coughing easier.

[NICE's guideline on cystic fibrosis: diagnosis and management, glossary in full guideline]

Equality and diversity considerations

At the time of publication, rhDNase did not have a UK marketing authorisation for use in children under 5 years with cystic fibrosis for this indication. If this medicine is considered to be appropriate for a child under 5 years, the prescriber should follow relevant professional guidance, taking full responsibility for the decision. Informed consent should be obtained and documented. See the General Medical Council's Prescribing guidance: prescribing unlicensed medicines for further information.



[2] At the time of publication (May 2018), rhDNase did not have a UK marketing authorisation for use in children under 5 years with cystic fibrosis for this indication. The prescriber should follow relevant professional guidance, taking full responsibility for the decision. Informed consent should be obtained and documented. See the General Medical Council's Prescribing guidance: prescribing unlicensed medicines for further information.