NICE has failed to recommend Xolair (omalizumab) for the treatment of severe persistent allergic asthma in adults and children in draft guidance after reviewing new evidence.

The guidance reviewed two separate appraisals in 2007 – one which recommended Xolair for adults and one which did not for use in children.

Sir Andrew Dillon, Chief Executive of NICE, said that new data showed that the treatment is “not as clinically or cost-effective as was first thought.”

Xolair has a UK marketing authorisation as an add-on therapy to standard care to improve control of asthma in adults and youngsters aged 6 and above with severe persistent allergic asthma.

However, after new evidence was made available, particularly on mortality data, NICE’s independent Appraisal Committee decided that the draft guidance should not recommend the treatment for either children or adults.

In addition, the Committee also took into account changes to the dosing schedule and the effect this would have on the cost effectiveness of the treatment. This, when combined with uncertainties over the new clinical data, did not support a positive recommendation.

The Committee did recognise that Xolair was an effective therapy for asthma sufferers but said evidence submitted as part of the appraisal included people whose condition was less severe than those currently being treated in the UK.

“The Committee is aware that severe, persistent allergic asthma can have a detrimental effect on a person’s life and that omalizumab is an effective therapy for children, adolescents and adults with severe persistent allergic asthma,” said Sir Andrew Dillon.

“Unfortunately, the Committee was unable to continue to recommend omalizumab for use in the NHS.”

Patients currently taking Xolair should be able to continue treatment until their clinician considers it appropriate to stop.

Final guidance is now expected in April 2013.

NICE has launched a second consultation on the use of Xgeva (denosumab) for the treatment of cancer patients with bone metastases after again recommending its use on the NHS.

An independent Appraisal Committee heard evidence from clinical experts during the original consultation on the draft guidance on current UK clinical practice concerning its comparison to existing options.

As a result, the latest draft guidance now recommends Xgeva for adults with bone metastases from breast cancer and those with solid tumours other than breast and prostate only if certain other bisphosphonates would otherwise be prescribed.

The updated guidance also states the treatment must be supplied under the terms of an agreed Patient Access Scheme, and that Xgeva is not recommended for preventing skeletal-related events in those with bone metastases from prostate cancer.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, said feedback from clinical experts “shed new light on the original recommendations”.

Earlier draft guidance recommended the treatment in people with bone metastasis from breast cancer; people with painful bone metastasis from hormone-refractory prostate cancer when treatment has failed; and for those with bone metastasis from other solid tumours for whom zoledronic acid is indicated.

The appraisal is considering the treatment as an alternative to bisphosphonates and as an alternative to best supporting care where they are not used.

Comments are now being invited by NICE and its Appraisal Committee on the updated draft guidance.

NICE has launched a second consultation on the use of Xgeva (denosumab) for the treatment of cancer patients with bone metastases after again recommending its use on the NHS.

An independent Appraisal Committee heard evidence from clinical experts during the original consultation on the draft guidance on current UK clinical practice concerning its comparison to existing options.

As a result, the latest draft guidance now recommends Xgeva for adults with bone metastases from breast cancer and those with solid tumours other than breast and prostate only if certain other bisphosphonates would otherwise be prescribed.

The updated guidance also states the treatment must be supplied under the terms of an agreed Patient Access Scheme, and that Xgeva is not recommended for preventing skeletal-related events in those with bone metastases from prostate cancer.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, said feedback from clinical experts “shed new light on the original recommendations”.

Earlier draft guidance recommended the treatment in people with bone metastasis from breast cancer; people with painful bone metastasis from hormone-refractory prostate cancer when treatment has failed; and for those with bone metastasis from other solid tumours for whom zoledronic acid is indicated.

The appraisal is considering the treatment as an alternative to bisphosphonates and as an alternative to best supporting care where they are not used.

Comments are now being invited by NICE and its Appraisal Committee on the updated draft guidance.

NICE has failed to recommend the use of Sanofi’s Jevtana (cabazitaxel) in final guidance in combination with prednisone or prednisolone as a second line treatment for prostate cancer.

Concerns were raised by NICE’s Appraisal Committee over the cost of the treatment and its side-effects, including haematological adverse events and diarrhoea.

Sir Andrew Dillon, Chief Executive of NICE, said the Committee queried the “nature of the health-related quality of life information” provided by Sanofi.

Sanofi had appealed the decision not to recommend the treatment however, this was dismissed on all points.

NICE recognised that Jevtana resulted in a mean improvement of greater than three months in mean overall survival. But the Committee considered that its cost per QALY gained would exceed £87,500 – considerably higher than the most expensive treatment it has recommended at £50,000.

Additionally, the numerous side-effects, such as fatigue, nausea, vomiting and constipation, associated with Jevtana raised concerns with the Appraisal Committee.

“We need to be sure that new treatments provide sufficient benefits to patients to justify the significant resources the NHS would need to make available,” said Sir Andrew.

“Although cabazitaxel can extend life for some patients, its price remains well above what the independent Committee appraising this drug considered acceptable, given the benefits it offers.”

NICE has failed to recommend Roche’s Avastin (bevacizumab) for the first-line treatment of metastatic breast cancer after raising a number of uncertainties over its use in new draft guidance.

Its independent Appraisal Committee questioned the clinical benefit of the drug in overall survival and concluded its high cost was not a good use of NHS resources.

Sir Andrew Dillon, NICE Chief Executive, says the evidence provided “did not conclusively” demonstrate Avastin to be clinically and cost effective.

The breast cancer drug was being appraised when used in combination with the chemotherapy drug Xeloda (capecitabine).

Data provided by Roche had demonstrated that median progression-free survival benefit associated with Avastin plus Xeloda was 2.9 months greater than Xeloda monotherapy alone.

However, the Committee claimed it was unclear whether that benefit translated into an improvement in overall survival.

The Committee also raised the issue of a lack of data showing whether patients would have a better quality of life than if they were treated with chemotherapy alone.

Also, NICE noted that Roche’s cost-effectiveness figures were based on a specific subgroup of patients who had previously received a taxane, and not on the whole capecitabine cohort.

Given these uncertainties the Committee concluded it was not possible to arrive at a plausible Incremental Cost Effectiveness Ratio (ICER) per QALY gained for bevacizumab plus capecitabine compared with capecitabine alone for the whole patient population and were not able to recommend the treatment.

“We can’t recommend a drug that has not been shown to work as well as, or better than, current treatments and costs much more,” said Sir Andrew. “We want to ensure people have access to the best treatments the NHS can afford; bevacizumab has so far not been proven to be clinically or cost-effective.”

Breast cancer is one of the most common cancers in England. In the UK, it is estimated that more than 48,000 women and around 300 men are diagnosed with the disease each year.

The draft guidance is now open for consultation.

NICE has recommended the use of Tasigna (nilotinib) and Glivec (imatinib) for the first-line treatment of chronic myeloid leukaemia (CML), but failed to recommend Sprycel (dasatinib) in final draft guidance.

The guidance reaffirms the use of standard dose Glivec and recommends the use of Tasigna after Novartis agreed a Patient Access Scheme with the DH to lower its cost.

Sir Andrew Dillon, NICE Chief Executive, says the Institute is “very pleased to be able to add a further treatment option” for patients.

CML is a rare condition that affects around 560 people in the UK each year.

NICE’s independent Appraisal Committee considered the results of clinical trials and noted the views of clinical specialists and patient experts when conducting the appraisal.

It found that more patients receiving Tasigna and Sprycel had a complete cytogenetic response and a major molecular response than people receiving Glivec at 12-month follow-up.

Also, experts said that Tasigna and Sprycel are more effective than standard-dose Glivece with a theoretically superior mechanism of action, although this option remains very effective.

The Committee concluded that available evidence suggests Tasigna and Sprycel provide superior clinical benefit as measured by surrogate outcome measures to standard-dose Glivec in people with chronic phase CML – despite no direct head-to-head data being available.

“Although no trials directly comparing dasatinib and nilotinib were available, the Committee concluded from indirect comparisons that dasatinib and nilotinib could be considered equally as effective in treating CML,” said Sir Andrew Dillon.

“However, the Department of Health and the manufacturer of nilotinib have already agreed to provide the drug to the NHS at a discounted price. This reduction in cost enabled the independent Committee to approve nilotinib for use on the NHS.”

NICE has requested more information on the long-term clinical and cost effectiveness of Bayer’s Xarelto (rivaroxaban) for the treatment of deep vein thrombosis (DVT) in draft guidance.

Its independent Appraisal Committee concluded the data supplied did not demonstrate its effectiveness in the context of UK practice and questioned the differences between subgroups using the treatment.

Meindert Boysen, Programme Director Technology Appraisals at NICE, said that Xarelto may be a “useful alternative” to existing options but Bayer needs to supply further data to meet various concerns.

The appraisal is considering the use of Xarelto to treat DVT and prevent recurrent DVT and pulmonary embolism (PE) after acute DVT in adults. It is estimated there will be more than 46,000 cases of acute DVT in England and Wales this year, rising to nearly 50,000 by 2016.

Bayer supplied NICE with information on Xarelto’s cost and clinical effectiveness for patients taking the treatment for up to 12 months.

But the Committee noted that treatment may extend beyond a year and that it wished to explore trial data between subgroups of patients using the treatment for different durations.

“The Committee was concerned that an analysis for patients who required treatment beyond 12 months was not presented, and noted the comments of clinical specialists to the effect that some people with DVT need to continue on anticoagulant therapy permanently,” said Meindert Boysen.

“The Committee also noted subgroup analyses in the main clinical trial that suggested that there could be differences in the effectiveness of rivaroxaban between the groups that were assigned treatment durations of 3, 6 and 12 months that it would wish to explore further.

“The Committee has therefore requested further information from Bayer about the clinical and cost effectiveness of rivaroxaban used as a long-term treatment and has also asked for comment on the differences between the subgroups receiving different intended treatment-durations.”

NICE recommended the use of Xarelto as a treatment option to prevent venous thromboembolism in adults having elective total hip replacement surgery or total knee replacement surgery in 2009.

NICE has requested further data on Tarceva (erlotinib) from Roche for the first-line treatment of locally advanced or metastatic EFGR mutation-positive non-small-cell lung cancer (NSCLC) in new draft guidance.

Roche failed to provide evidence on Tarceva’s clinical and cost effectiveness when compared to Iressa (gefitinib), NICE’s Appraisal Committee said.

Sir Andrew Dillon, NICE Chief Executive, said the data provided by Roche was not sufficient to compare Tarceva with a recommended option but hoped further information would be supplied.

The Committee has now asked Roche for an updated cost effectiveness analysis of Tarceva compared with Iressa with different progression-free survival and utility assumptions. Further sensitivity analyses on the cost of Tarceva changes depending on how many patients are able to continue taking the drug and Iressa after 60 days of treatment – the point at which the NHS currently has to start paying for Iressa under its agreed Patient Access Scheme – are also required.

“When it was asked to consider Tarceva, our independent advisory committee concluded that it did not have enough information to be able to make the decision to recommend or not recommend it for routine use in the NHS as an alternative to gefitinib,” said Sir Andrew. “It has therefore asked the manufacturer provide further analyses. We hope that Roche will be able to provide this additional information so that the Committee can consider it at its next meeting on the topic.”

The SMC recently approved the use of Tarceva as a first-line treatment for NSCLC. Lung cancer, after breast cancer, is the second most common cancer in England and Wales, with an estimated 40,800 newly diagnosed cases each year.

NICE has requested further information from Allergan on the use of Botox (botulinum toxin type A) to prevent headaches in adults with chronic migraines after failing to recommend its use in draft guidance.

The Institute’s Appraisal Committee does not believe the evidence supplied is sufficient for it to develop guidance after queries were raised over its cost effectiveness compared to existing treatments.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, says further information is required to develop “sound advice” to the health service.

The guidance analysed whether Botox should be considered as a treatment option for those who experience headaches associated with chronic migraine, and whose condition has not improved after at least three previous preventative treatments.

A chronic migraine, which is believed to affect 1.6% of adults, is defined as having a headache for at least 15 days a month over a period of three months, of which eight of the days are with a migraine.

Evidence demonstrated in clinical trials showed that Botox does have some benefit. But the amount of benefit proved to be small and the results were confounded by a large placebo effect, NICE found. This was due to people realising during clinical trials that they were receiving the injection due to its widely-recognised side effects, such as muscle paralysis. Concerns were also raised by the Committee into the long-term effectiveness of Botox.

NICE has now requested additional data on the injection’s cost effectiveness and asked Allergan to revise its economic modelling and analyses to clarity uncertainties over certain inputs and assumptions made in its cost model.

“Without this additional evidence, potentially we will be unable to advise the NHS that this drug is good value for money for these adults because there are currently too many uncertainties,” said Professor Longson.

The deadline for consultations on the draft guidance is 8 March 2012. Final guidance is expected in June 2012.