The National Institute for Health and Care Excellence (NICE) it remaining steadfast in its position that Roche’s Esbriet should only be funded by the NHS for patients with moderate forms of the fatal lung scarring disease idiopathic pulmonary fibrosis (IPF).
Essentially, this means that patients will have to wait until their disease has worsened - and lung function has been lost - before getting routine NHS access to the drug.
“This is an illogical decision, to deny effective treatment in a condition that is irreversibly progressive and ultimately fatal,” said Dr Toby Maher, consultant physician at the Royal Brompton Hospital.
“I would like to treat patients with a drug that has shown clinical efficacy, rather than wait for them to decline until they are considered by NICE sick enough to warrant treatment. England continues to be one of the few countries in Europe where IPF patients don’t have access to pirfenidone or an alternative treatment for early IPF.”
IPF is a fatal disease caused by irreversible, progressive scarring of the lungs, which makes breathing difficult and prevents the heart, muscles and vital organs from receiving enough oxygen to work properly.
The condition affects 15,000 people in the UK and, without treatment, patients only live for around two to five years from diagnosis on average, highlighting the urgent need for new options.
NICE first recommended Esbriet (pirfenidone) for patients with moderate IPF in 2013. Evidence submitted to the Institute from the Phase III ASCEND trial, which included patients with early IPF, estimated survival was increased by up to three years with Esbriet therapy.
Following an appeal by Roche, the Institute has now published a second final appraisal determination in which it stipulates that Esbriet will only be funded by the NHS if the person has a forced vital capacity (FVC) between 50 percent and 80 percent predicted and Roche provides the drug with the discount agreed in the patient access scheme.
The Institute says Esbreit could not be considered a cost-effective use of NHS resources for the population specified in the marketing authorisation - that is, adults with mild to moderate idiopathic pulmonary fibrosis - because the most plausible incremental cost-effectiveness ratio (ICER) lay somewhere between about £25,700 and £28,900 per quality-adjusted life years (QALY) gained compared with best supportive care, and was associated with uncertainties that had the potential to substantially increase this.
“As a company, Roche invested more than £460m in UK R&D in 2016[ to bring personalised, innovative treatments to patients,” commented Dr Rav Seeruthun, medical director, Roche UK.
“We consider it vital that we work with NICE to find solutions today for the innovation of tomorrow. But we do not believe that in this instance the decision of the committee is in the interest of early IPF patients and their families.”