Patients in England and Wales with the lung scarring disease idiopathic pulmonary fibrosis will have to wait until their disease has worsened before getting routine NHS access to Roche's Esbriet.
The National Institute for Health and Care Excellence has published a Final Appraisal Determination rejecting the use of Esbriet (pirfenidone) for the treatment of early IPF, which Roche says leaves patients with no treatment option to slow the progression of their disease until lung function has been lost.
In 2013, the Institute recommended Esbriet for patients with moderate disease based on positive Phase II and Phase III data. Findings from the Phase III ASCEND trial - which included more patients with the early form of the disease - showed that, after one year of treatment with drug, the risk of death from IPF nearly halved, with a 48 percent relative reduction in mortality at 52 weeks compared to placebo.
Also, the mean decline from baseline in forced vital capacity (FVC) was 235 ml in the Esbruet group and 428 ml in the placebo group, Roche said.
"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," said Dr Toby Maher, Consultant Physician at the Royal Brompton Hospital. "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."
Dr James Mawby, Medical Lead for Rare Diseases at Roche UK, said NICE's rejection "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. 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."
Roche also stressed that the UK is one of the only countries in Europe where patients do not have access to Esbriet or an alternative treatment for early IPF.
However, explaining the decision, a spokesperson for NICE said: "Roche presented new evidence to our committee to propose expanding the use of Esbriet and they also wanted the NHS to pay a higher price for the drug. The NICE committee concluded that pirfenidone was not cost effective at the higher price. It also concluded that the new evidence was not robust and, and even at the current price paid by the NHS, Esbriet could not be considered cost-effective to a wider group than was already recommended."
IPF affects 15,000 people in the UK. Without treatment, patients only live for around two to five years from diagnosis on average.