NICE Against NHS Funding of Pirfenidone in Pulmonary Fibrosis
National Institute for Health and Clinical Excellence issued preliminary guidelines against prescribing pirfenidone (Esbriet) to patients with idiopathic pulmonary fibrosis.
Although the drug had a modest measurable effect on improving lung function, NICE found that the scarce evidence for its long-term efficacy, as well as concerns over safety of the drug, did not warrant NHS funding. Esbriet is currently available in 29 European countries, Japan and South Korea. Health Canada approved Ebsbriet in early October and the drug is expected to be available in January 2013.
Idiopathic pulmonary fibrosis, with over 5000 new cases identified each year in the UK alone, is a disease where progressive damage to the alveoli leads to breathing difficulties, and eventually respiratory failure. Median survival from diagnosis is 3.5 years, and only one in five patients survives until five years later. The condition is most commonly treated with steroids, and occasionally with a lung transplant.
Pirfenidone, manufactured by InterMune, was approved by the European Medicine Agency in March last year based on evidence from trials that showed improvement in disease symptoms in adults with mild-to-moderate condition, but had to be terminated before the scheduled end-date either due to ethical considerations or concerns over data handling. Nevertheless, a meta-analysis of six randomized placebo-controlled trials published in PLoS One earlier this year showed that patients on Esbriet were more likely to discontinue the therapy compared to the controls treated with placebo (RR=1.85), and had a greater risk of gastrointestinal (RR=2.11), neurological (RR=1.68) and dermatological (RR=2.88) side-effects.
Additionally, the treatment was not found cost-effective, with spendings exceeding £30,000/QUALY, the standard threshold of cost-effectiveness used by NICE.
“Given these uncertainties and when compared with best supportive care, our committee concluded that treatment with pirfenidone would not represent a cost-effective treatment option for the NHS,” Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE said in the press release.
NICE initiated public consultations and those wishing to comment have until 5pm on Wednesday 19 December to do so.
“We encourage healthcare professionals, people with idiopathic pulmonary fibrosis and other relevant third parties to comment on our committee's provisional recommendations,” the statement said. The committee will meet again in January, and the final recommendations for England and Wales is expected to be released in April 2013. Guidelines for Scotland are expected two months earlier.