Novartis and patients in England and Wales with advanced breast cancer will be disappointed with news that cost regulators have decided to bar access to Afinitor on the National Health Service.

The National Institute for Health and Clinical Excellence has published provisional draft guidance turning down the drug as an NHS-funded treatment for HER2 negative, hormone-receptor-positive advanced breast cancer.

Currently, there is no cure for the disease and patients can expect to live just 18-36 months after being diagnosed. According to Novartis, Afinitor (everolimus) is the first new licensed therapeutic approach in fifteen years offering substantial impact on hormone responsive advanced breast cancer, which affects over 30,000 women in the UK.

The gold standard of care is endocrine therapy, such as aromatase inhibitors, but unfortunately most patients will eventually develop resistance to treatment, allowing the disease to progress, highlighting the need for new approaches to address this problem.

"New and effective treatment options such as everolimus can make a real difference as they circumvent this resistance, slow down cancer cell growth and ultimately extend and enhance patients’ quality of life," noted Professor Peter Barrett-Lee, Consultant Clinical Oncologist and Medical Director at the Velindre NHS Trust, Cardiff.

Indeed, data from the Phase III, 724-patient BOLERO-2 trial showed that treatment with Novartis' drug plus exemestane more than doubled median progression-free survival to 7.8 months, compared with 3.2 months with exemestane alone, thereby offering patients a further treatment option.

But while NICE's Appraisal Committee acknowledged that Afinitor "may offer a step change in treatment by restoring sensitivity of the tumour to hormone therapy", there were uncertainties relating to how much the treatment extends overall survival, said chief executive Andrew Dillon. 

"Using the most appropriate estimates, the committee concluded that everolimus is not a cost-effective treatment option for the NHS," he explained.

Unsurprisingly, Novartis said it is "extremely disappointed" with the decision. But the firm remains committed to "providing as much information as we can to demonstrate the value of everolimus to patients and the NHS", said to Ibrahim ElHoussieny, Oncology Medical Director at Novartis UK & Ireland.

Also voicing disappointment over the decision, Baroness Delyth Morgan, Chief Executive of Breast Cancer Campaign, stressed that women with secondary breast cancer "currently have limited treatment options available to them".  

But while approval has not been given by NICE, access may be possible through the Cancer Drugs Fund, she said, urging "clinicians to use this avenue for patients who they feel may benefit from this drug".

Revolade and Esbriet in

Elsewhere, and on a more positive note, the Institute has published draft guidance recommending GlaxoSmithKline's Revolade (eltrombopag) for the treatment of chronic immune (idiopathic) thrombocytopenic purpora.  

As per the guidance, NICE has recommended the drug as an option for patients who have had a splenectomy, and whose condition is refractory to other treatments, as well as a second-line treatment in adults who have not had a splenectomy because surgery is contraindicated.

However, it has also stipulated that the drug should only be used if patients have severe disease and a high risk of bleeding that needs frequent courses of rescue therapies.

Originally, the cost watchdog ruled out Revolade's use in 2010 after calculating that its projected cost was £104,100 per QALY for splenectomised patients and £116,750 per QALY for those without, making it far too expensive to be funded on the health service. But a confidential discount since offered by GSK via a patient access scheme has allowed NICE to change its stance.

In another u-turn, NICE has also this morning (Thursday) recommended the use of InterMune's Esbriet (pirfenidone) as an option for certain patients with the chronic lung condition, idiopathic pulmonary fibrosis, after provisionally rejecting its use last year.

Specifically, the Institute has OK'd the drug's use for patients who have a forced vital capacity - the amount of air that can be forcibly exhaled from the lungs - between 50% and 80% predicted, agreeing that the incremental cost-effectiveness ratio (ICER) for Esbriet was likely to be around £24,000 per QALY for this subset of patients.

NICE’s positive draft recommendation comes after InterMune submitted additional evidence to demonstrate the drug's potential benefits and revised the terms of its patient access scheme, details of which are being kept under wraps.