The National Institute for Health and Care Excellence (NICE) has published a stream of final guidances approving Forxiga for diabetes, Betmiga for overactive bladder and Xarelto for pulmonary embolism, but turning down Jakavi for enlarged spleen and Krystexxa for gout.

The cost regulator has OK'd funding for the NHS use of Bristol-Myers Squibb/AstraZeneca's first-in-class diabetes type II therapy Forxiga (dapagliflozin) in some patients.

Specifically, the drug has been recommended for use as a dual therapy in combination with metformin (if it is used as described for DPP4-inhibitors) or in combination with insulin.

Forxiga's unique mode of action blocks the reabsorption of glucose in the kidneys and promoted the excretion of excess glucose in the urine. Given that this also removes calories from the body, the drug could offer patents secondary benefits in terms of weight loss.

NICE has also recommended Astella's Betmiga (mirabegron) as an NHS option for treating the symptoms of overactive bladder (OAB). 

The first in a new class of drugs, Betmiga works by stimulating _3 receptors to relax the bladder muscle during filling and thereby improve storage capacity without inhibiting bladder voiding.

"Offering mirabegron as an option for treatment, with clear advice about its potential benefits and side effects will help improve quality of life for people living with this distressing condition," said Professor Carole Longson, the Institute's health technology evaluation centre director.

In draft guidance, it was noted that Betmiga fell “well below” NICE’s £20,000 QALY limit when compared against a current standard therapy - Pfizer’s Detrusitol (tolterodine), and thereby represents value for money for the NHS.

Elsewhere, Bayer and Janssen’s Xarelto (rivaroxaban) pill has been given a green light for treating pulmonary embolism (PE) and preventing recurrent deep vein thrombosis (DVT) and PE after acute PE in adults.

Treatment with the veteran standard of care, warfarin, requires regular monitoring and dose adjustments that can be costly and inconvenient, and the medicine is linked with many drug and diet interactions.

"Rivaroxaban therefore represents a significant potential benefit for people with PE and DVT because it avoids the need for initiation with heparin and the subsequent transition to warfarin," Longson noted.

With the committee’s preferred assumptions, the incremental cost for long-term treatment with the drug was under £19,400 per QALY gained, and so within the normal cost-effectiveness threshold.


On the downside, NICE has rejected the use of Novartis' Jakavi (ruxolitinib) on the NHS for treating enlarged spleen in patients with types of the rare blood cancer myelofibrosis.

The independent committee concluded that while Jakavi was indeed clinically effective in reducing spleen size and symptoms such as itch and fatigue, it could not be considered a cost-effective use of NHS resources compared with best available therapy.

Furthermore, although it was plausible that the drug could offer an overall survival benefit, the reason for this "remained unclear because of uncertainties in the evidence", NICE said.

Savient's Krystexxa (peglioticase) was also turned down for NHS use in patients with chronic gout.

The committee agreed that the drug effectively lowers the level of uric acid in the blood for a significant proportion of patients with the condition, but noted the risk of severe adverse reactions and uncertainties about its long-term efficacy and safety.

The cost per QALY was estimated to be over £54,000, and thereby well above £20,000-30,000 range normally considered cost-effective.