A new drug has been approved for use in England and Wales in patients with severe chronic obstructive pulmonary disease (COPD) who continue to suffer with exacerbations.
The National Institute for Health and Care Excellence has this week recommended treatment with roflumilast (Daxas) as an add-on to bronchodilator therapy under certain circumstances.
It has been recommended as an option for treating severe COPD in adults with chronic bronchitis, but only under certain circumstances.
It can only be used if the disease is severe – defined as a forced expiratory volume in 1 second (FEV1) after a bronchodilator of less than 50% of predicted normal – and the patient has had two or more exacerbations in the previous 12 months, despite triple inhaled therapy with a long-acting muscarinic antagonist, a long-acting beta-2 agonist and an inhaled corticosteroid.
The clinical regulator estimates that around 122,000 adults in England will be eligible to receive roflumilast.
NICE also noted that patients having treatment outside its new recommendations may continue without change, until they and their NHS clinician consider it appropriate to stop.
Roflumilast, which is made by AstraZeneca, is an orally administered long-acting selective phosphodiesterase-4 enzyme inhibitor.
It targets cells and mediators believed to be important in COPD, according to NICE’s technology appraisal guidance on the drug, which was published on Wednesday.
The recommended dose is 500 micrograms (one tablet) of roflumilast once daily. It costs £37.71 for 30 tablets and £113.14 for 90 tablets.
The most common adverse reactions associated with roflumilast include diarrhoea, weight loss, nausea, abdominal pain and headache, according to AstraZeneca.
The NICE appraisal committee considered evidence for roflumilast submitted by the drug’s firm using pooled analyses from two trials, one called REACT and one called RE2SPOND.
In the REACT trial, 973 patients were randomly assigned to roflumilast and 972 to a placebo group. The rate of moderate-to-severe COPD exacerbations was 13.2% lower in the roflumilast group than for the placebo group. Compared with placebo, roflumilast treatment led to a 24.3% reduction in severe events and 23.9% reduction in exacerbations requiring hospital admission.
Meanwhile, in the RE2SPOND trial, the rate of moderate-to-severe exacerbations per patient per year was reduced by 8.5% with roflumilast versus placebo, but was not statistically significant. The time to first exacerbation event was also no different between the two groups.
However, in a post hoc analysis in participants with more than three exacerbations in the prior year, the rate of moderate or severe exacerbations was reduced by 39% with roflumilast versus placebo. In addition, in those with one or more prior severe exacerbations, the rate of moderate or severe COPD exacerbations was reduced by 25% with roflumilast versus placebo.