The National Institute for Health and Clinical Excellence (NICE) has called for a clinical trial to be set up to assess the clinical effectiveness of Merck, Sharp & Dohme's Daxas (roflumilast) as an add-on treatment for people with severe chronic obstructive pulmonary disease (COPD).

In draft guidance which is now out for consultation, NICE says its independent appraisal committee has concluded that there is "great uncertainty" about the effect of Daxas on reducing the rate of exacerbations in people with severe COPD associated with chronic bronchitis as an add-on to bronchodilator treatment. For that reason, the draft guidance recommends setting up a trial to assess the drug's clinical effectiveness for this indication, and also says that it should be compared with theophylline in people for whom it might be suitable.

The appraisal committee had concluded that Daxas was most likely to be used in addition to triple therapy but the members were concerned about the lack of clinical evidence available on its use in that way. As there was "great uncertainty" about how well Daxas works alongside triple therapy, the committee could not establish that it would be good value for money, said Professor Carole Longson, director of NICE's health technology evaluation centre. 

"As a result, the committee discussed whether it was appropriate to recommend the set-up of a research trial to determine the clinical effectiveness of roflumilast on top of triple or dual therapy. In this case, the committee felt that the benefits and costs of carrying out the research would be favourable to the NHS," said Prof Longson.

Before the independent appraisal committee can make an "only in research" recommendation, the following factors need to be considered: - the intervention should have a reasonable prospect of providing benefits to patients in a cost-effective way; and - the research can realistically be set up, is already planned, or is already underway.

There are currently 1 million people with COPD in England and Wales. It is estimated that almost 200,000 would be eligible for treatment with Daxas by 2015, and that likely treatment duration would be many years, says NICE.

"While we recognise it is important for people with severe COPD to have a range of options, NICE has to ensure that the NHS provides treatments which are effective and value for money," said Prof Longson. "We hope a clinical study would generate robust evidence so that we can be clear about the benefits roflumilast brings for these patients," she added.

Prof Longson also called on Merck, Sharp & Dohme, patient organisations and other relevant third parties to comment on NICE's preliminary recommendation during the public consultation period "so that they can contribute to the development of this guidance."

NICE is expected to issue final guidance to the NHS in England and Wales on the use of Daxas in the management of COPD in January 2012.