NICE OKs Pharmaxis and Novartis drugs but not Forest's colistimethate in CF patients
This article was originally published in Scrip
In a wave of developments for cystic fibrosis (CF), NICE, the health technology appraisal institute for England and Wales, has recommended Pharmaxis's Bronchitol (mannitol dry powder) as a treatment option and separately, Novartis's Tobi Podhaler (tobramycin dry powder for inhalation; DPI) for Pseudomonas lung infection in people with the disease.
However, Forest Laboratories failed to convince the Institute over its Colobreathe (colistimethate sodium DPI) similarly, for chronic pulmonary infection caused by P aeruginosa in this patient set.
Pharmaxis's Bronchitol
Bronchitol is the first and only cystic fibrosis product to have received a positive recommendation from NICE, Pharmaxis noted.
In fact, the news provides a big boost to the company as the Institute, in June, rejected the drug outright, saying there were too many gaps and uncertainties relating to the evidence on the drug's relative effectiveness and about the long-term effect of the product on lung function (scripintelligence.com 1 June 2012).
NICE now said: “Following the helpful responses received during consultation on the draft guidance, the Committee concluded that mannitol was a good use of NHS resources when treatment was offered to those who cannot use hypertonic saline or rhDNase and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.”
Despite the recommendation, NICE's latest draft guidance stipulates that Bronchitol is to be used as a treatment option only in adults who do not use inhaled mucolytics (because they cannot take rhDNase) and for whom other osmotic agents like hypertonic saline are not appropriate. Additionally only cystic fibrosis patients with rapidly declining lung function (FEV
Consultees have until 9 November to appeal the new decision on Bronchitol with the final appraisal determination document expected to be published next month itself.
Tobi Podhaler and Colobreathe
In separately published draft guidance, NICE has recommended Novartis's Tobi Podhaler as a treatment option when nebulised tobramycin is considered an appropriate treatment, that is, when nebulised colistimethate is contraindicated, not tolerated or has not produced an adequate clinical response. The decision is also contingent upon Tobi Podhaler being provided at a discount agreed with Novartis as part of a patient access scheme to primary, secondary and tertiary care in the NHS.
NICE's Committee outlined that the economic analysis for Tobi Podhaler versus nebulised tobramycin did show that Tobi Podhaler was cheaper and slightly more effective, albeit with some 'uncertainty'.
“Despite the limitations of all the data and the uncertainty in the model, the Committee agreed that it was reasonable to conclude that tobramycin DPI was a cost effective use of NHS resources in people with cystic fibrosis who would otherwise have been treated with nebulised tobramycin,” it concluded.
But in the case of Colobreathe, the Institute stated that “there was no economic analysis which compared colistimethate sodium DPI with nebulised colistimethate, the preferred comparator”. Therefore, it said, “The results of the Committee’s preferred economic analysis showed that colistimethate sodium DPI was less effective and less costly than nebulised tobramycin. The Committee concluded that although the analysis showed potential for cost savings, the clinical evidence for colistimethate sodium DPI was not robust enough to conclusively show that it is a cost-effective use of NHS resources.”
The closing date for comments on this appraisal consultation document is Tuesday 13 November, in time for the second appraisal committee meeting on Tuesday 27 November. The final guidance is expected in March 2013.