Pharmaceutical company Pharmaxis (PXS) announced that the National Institute for Health and Clinical Excellence (NICE) in the UK has issued a preliminary opinion that Bronchitol (inhaled dry powder mannitol) could not yet be considered a cost‐effective use of NHS resources for the treatment of cystic fibrosis in England and Wales. This opinion is not NICE’s final guidance on Bronchitol and the recommendations may change following consultation and receipt of stakeholder comment.
The Committee’s view at this stage is that the calculation of cost effectiveness is associated with considerable uncertainty and dependent on a variety of imputed assumptions. Pharmaxis CEO Dr Alan Robertson said, “We believe that the cost effectiveness ratios in our submission are correctly positioned to gain a positive recommendation from NICE. The Committee has clearly identified a number of areas of uncertainty and we will be working with NICE and other patient and rofessional stakeholders over coming weeks to supply further analyses and clarification. We look forward to further progress and the forthcoming Committee meeting, which will consider the comments received during the consultation period.
Bronchitol has been introduced in the United Kingdom at a retail price of £16.55 per day and notification has been received from the UK Department of Health that this price is acceptable.
“No CF drugs currently available in the UK other than Bronchitol have ever been reviewed by NICE. The decision to fund Bronchitol remains in the hands of the local health authorities with whom we have been liaising over recent months. Bronchitol is now available in pharmacies and we will be pursuing the negotiations with local payors to fund treatment for CF patients in their area,” Dr Robertson said.
Pharmaxis officially published the retail price of Bronchitol in Germany at €35.65 per day. Germany’s Federal Joint Committee (G‐BA) has a modified procedure for orphan drug assessments such that only products with annual sales in Germany of more than €50 million will be subject to an early benefit assessment from the Institute for Quality and Efficiency in Health Care (IQWiG).
Dr Robertson said, “Bronchitol has demonstrated significant reductions in the incidence of exacerbations when used on top of standard therapy in addition to its effects on improving and maintaining lung function.
This retail price is in line with guidance we have provided in relation to the German market and pricing research conducted by Pharmaxis amongst German physicians and insurance funds. “We now look forward to introducing Bronchitol to UK and German CF clinics following the formal European launch, which will take place at the European CF symposium in Dublin from June 6th – 9th 2012.”