Price cut and real world data persuades NICE to recommend lupus drug Benlysta


Benlysta, GlaxoSmithKline's treatment for autoimmune disease lupus has been recommended by NICE, after the company agreed to a price cut and a real world data study.

The decision comes after years of rejection from England's cost effectiveness watchdog, which first reviewed the drug in 2011.

Systemic lupus erythematosus (SLE) is a severe life-shortening condition which mainly affects women of child-bearing age, and Benlysta is the only drug on the market aimed at halting the progression of the disease.

Once tipped to be a blockbuster, Benlysta (belimumab) has been slow to build sales it was launched in 2011. In 2015, GSK posted sales of £230 million, up 25% on the previous year, but the vast majority of these revenues were from the US market.

Benlysta's poor commercial performance is largely due to weak efficacy data, plus concerns about a long list of side-effects, including most commonly nausea, vomiting and diarrhoea.

GSK contends that its drug can extend the lives of patients from an average estimated survival of 32.8 years to 35 years. But NICE's committee says the trial data suggests the contrary, with a trend towards higher mortality in the Benlysta arm compared to patients receiving standard care.

Negotiations to lower the price, real world data collection and limiting treatment to patients with the most severe disease have all helped to finally produce a positive NICE final appraisal determination.

It recommends Benlysta as an option for add-on treatment for patients with the most severe active autoantibody-positive SLE, and treatment will only continue beyond 24 weeks if patients show a measurable improvement.

Added to this will be the price discount (which remains confidential). However Benlysta's list price is relatively modest compared to many other, newer rare disease treatments. The list price is £2,308.50 for the first four weeks, and then £769.50 every four weeks, but this will now be substantially discounted.

GSK's offer of a real world study to back up Benlysta's cost and clinical effectiveness is a sign of the times, with pharma companies increasingly having to offer such studies to gain NICE approval.

NICE's committee says it efficacy, safety and quality-of-life data should be collected using the British Isles Lupus Assessment Group (BILAG), an existing registry set up by GSK.

The company told NICE that the data would help clear up "many areas of uncertainty" identified by the committee, including which sub-groups of patients would benefit most, how long treatment should be continued for, and how the drug could help reduce the need for steroids and reduce the damage caused to organs by the disease.

GSK has said around 9–11 patients receiving belimumab would enrol in the registry each month, with an estimated total of 300 patients after three years.

NICE's decision is also remarkable because it says it still can't be sure how cost effective the drug is. The committee said that "considerable uncertainty" remaining in the economic modelling meant it could not place the drug within its incremental cost-effectiveness ratio (ICER) framework compared to standard care.

Read the NICE documents here 

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Andrew McConaghie

11 May, 2016