LONDON, Feb 19 (APM) - NICE on Thursday issued a green light to National Health Service funding of Norgine’s Targaxan (rifaximin) in hepatic encephalopathy after being swayed by third party real-world data which the company could not produce.
In a statement outlining its second-draft guidance, the cost-effectiveness body said the drug should be an option for treating the liver failure-related brain condition after being presented with real-world data by liver specialists showing potentially large savings in secondary care spend.
Speaking to APM, Norgine chief operating officer, Peter Martin said it was data collated by liver units in the UK, rather than anything the company could produce, which led to NICE changing its initial rejection in first draft guidance (APMHE 36397
) to a positive recommendation.
Martin said the data showed a 50% fall in hospital admission in patients on Targaxan versus those not on the agent and a reduction in duration of hospital stay, including a fall from 28 days stay per patient admitted to 9.4 days in one UK hospital.
This data, which was pivotal to NICE’s findings was not company funded but came about as NHS clinicians sought to quantify their observations of benefits for his company’s drug, Martin added.
“We did not have this data, it’s not our data” and without this the Dutch company may not have been able to change NICE’s thinking, he added.
Oblique reference from NICE to potential cost savings
Unusually for NICE, it did not provide a cost per quality adjusted life year (QALY) gained for Targaxan in a statement outlining its decision.
Neither did it directly refer to any potential savings from use of the drug but did note the hospital burden on patients with the illness.
“This serious brain condition has far-reaching effects on people with the condition and their families and carers. It can be serious, even fatal. People affected may have to go into hospital regularly. Rifaximin can prevent these life-threatening episodes and improve people’s quality of life,” it said.
NICE listed the average price at 1,690 pounds (2,281 euros) for six months treatment - less any locally negotiated discounts.
Martin said clinicians were gaining experience of use of Targaxan but so far there was little indication of patients being taken off the drug unless they died or had a liver transplant.
With value-based pricing scrapped and value-based assessment of drugs stalled, any immediate changes to NICE’s process appeared on hold although all parties - pharma, patients, government and the institute itself - now appear aligned on the potential benefits for reform, at least in oncology.
UK pharma has welcomed even oblique observations on the wider potential savings to the NHS in terms of reduced hospital bed-days, out patients or anywhere else which can be used to mediate the cost of new therapies under NICE's calculations.
As second draft guidance, NICE’s ‘yes’ will become binding on the NHS in an unchanged form unless it is appealed against - something almost unheard of when the institute issues a recommendation for state funding.