Cost regulators for NHS therapies in England and Wales have now issued final guidelines endorsing the use of Roche’s Esbriet to treat patients with idiopathic pulmonary fibrosis (IPF), but only in those with moderate to severe forms of the disease.
The National Institute for Health and Care Excellence is sticking with its position that Esbriet (pirfenidone) should 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.
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.
In response to a draft guidelines outlining NICE’s stance last year, Dr Toby Maher, consultant physician at the Royal Brompton Hospital, said it was “an illogical decision to deny effective treatment in a condition that is irreversibly progressive and ultimately fatal.”
“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.”
However, the Institute said Esbriet could not be considered a cost-effective use of NHS resources for adults with mild to moderate IPF 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.
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.