New medicinesNICE’s decision to refuse licencing for pirfenidone (Esbriet) for the treatment of early idiopathic pulmonary fibrosis (IPF) leaves patients with no treatment options that would help to slow the progress of the disease, pharmaceutical company Roche has said.

The decision means the UK is one of the only countries in Europe where patients do not have access to pirfenidone or an alternative treatment for early IPF. Pirfenidone remains available for patients who have lost more lung function.

In 2013, NICE recommended pirfenidone for patients with moderate disease based on positive Phase II and Phase III data. The Phase III ASCEND trial included more patients with the early form of the disease and provided further evidence that after one year of treatment with pirfenidone, the risk of death from IPF is nearly halved. In ASCEND, the mean decline from baseline in forced vital capacity was 235ml in the pirfenidone group and 428ml in the placebo group.

Without treatment, people with IPF on average only live for around 2–5 years from diagnosis, but the evidence submitted to NICE estimates that survival is increased by 2-3 years when these people are treated with pirfenidone.  

Dr James Mawby, Medical Lead for Rare Diseases at Roche UK, said: “We fundamentally disagree with NICE’s recommendation and believe it does a great disservice to patients with early IPF because there is still no funded treatment available for them. 

“At Roche we have been calling for a review of the system for evaluating innovative medicines in the UK as it is not set up to respond positively to treatments like pirfenidone. New clinical data for pirfenidone from the ASCEND trial shows that the benefits to IPF patients are even greater than earlier trials had demonstrated. The decision-making process used by NICE is not flexible enough when assessing treatments for rare diseases, where it is difficult to prove clinical benefit in different stages of the disease, and it doesn’t take into consideration the particular set of circumstances that patients with early IPF face. This is not the end of the road for us; we are exploring all options with all partners, and we will continue to press for access to effective treatments for early IPF.”

The most commonly reported adverse reactions with pirfenidone observed in clinical trials include nausea, rash, fatigue, diarrhoea, dyspepsia (heartburn) and skin photosensitivity. Adverse events tend to appear early in the course of treatment and are typically manageable, reversible and self-limiting. 

Dr Toby Maher, Consultant Physician at the Royal Brompton Hospital, added: “It is deeply disappointing that patients with less severe IPF are denied access to treatment. IPF is an irreversible disease and a growing body of evidence shows that the earlier the disease is treated the more lung function can be preserved thus providing greater long-term patient benefit. From my clinical perspective, I would like to have this medicine as an option for all my patients rather than wait for them to get irreversibly worse before I can treat them.”