NICE has confirmed its decision to reverse it opinion on Pharmaxis’ Bronchitol mannitol dry powder for the treatment of cystic fibrosis in some adults in final guidance.

The powder is recommended as an option in adults who cannot use a daily recombinant human deoxyribonuclease due to ineligibility, intolerance or inadequate response, whose lung function is rapidly declining and for whom other osmotic agents are not considered appropriate.

Bronchitol is inhaled with a hand-held, breath activated device. It has a marketing authorisation as an add-on therapy to best standard of care in adults with cystic fibrosis.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the treatment is “associated with fewer unpleasant side-effects, requires less costly equipment and less time to administer than nebulised treatments.”

Cystic fibrosis currently affects around 8,000 people in the UK. It is one of the most life-threatening inherited diseases.

Although there is no cure, treatment in adults aims to clear the respiratory secretions in order to maintain lung function, as well as reducing inflammation and bacterial infection in the respiratory tract.

“Cystic fibrosis has a major impact on the quality of life of patients and their carers so we are pleased to be able to recommend a new treatment that could ease some of this burden,” said Professor Longson.

“The Committee concluded that mannitol was a good use of NHS resources when offered to some adults who can’t use other treatments, and whose lung function is rapidly declining.”

NICE original questioned “gaps and uncertainties in the evidence” of Bronchitol’s effectiveness and raised concerns about its long-term effect on lung function when it failed to recommend the treatment in draft guidance.

Cancer patients whose disease has spread from a solid tumour to their bones have now been given a new treatment option after NICE backed the use of Amgen’s Xgeva (denosumab).

Xgeva has been recommended to treat the condition known as bone metastases in people suffering from breast cancer or solid tumours other than prostate who would otherwise be prescribed bisphosphonates.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, said Xgeva was a “welcome addition” alongside existing treatment options.

Final guidance states that Xgeva should only be prescribed under the terms agreed between the Department of Health and Amgen as part of a patient access scheme.

Amgen estimates there are around 150,000 patients in the UK with solid tumours and bone metastases, of which breast and prostate cancer account for more than 80%.

“We’re pleased to be able to recommend another treatment option for people with bone metastasis from most solid cancer tumours,” said Professor Longson. “This type of metastasis can reduce a person’s mobility and quality of life in general, increasing the risk of complications from bone weakness.”

NICE has recommended the use of Boehringer Ingelheim’s Actilyse (alteplase) in final guidance for the treatment of acute ischaemic stroke.

Actilyse has been recommended as an option for use on the NHS as long as it is administered as early as possible within 4.5 hours after the onset of stroke symptoms and once bleeding on the brain has been ruled out with a scan.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said the benefits of Actilyse in reducing long-term disability are “well recognised”.

The UK Stroke Association estimates there are more than 130,000 people in England and Wales who have a stroke each year. Ischaemic strokes are believed to account for around 80% of all strokes.

The recommendation follows an update to Actilyse’s license which allows an extension in the time period it can be used – from three hours to within 4.5 hours of the onset of symptoms.

“Today’s guidance recommending the use of alteplase within the extended time frame for which it is now licensed has the potential to have a significant impact on the treatment of thousands of patients,” said Professor Longson.

NICE has recommended Bayer Healthcare’s convenient Xarelto (rivaroxaban) in final guidance as a treatment option for adults with acute deep vein thrombosis (DVT).

The positive recommendation sees Xarelto become the first non-VKA oral anticoagulant to be recommended for use on the NHS after it impressed in phase III clinical trials.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said the treatment is “a potential benefit for many people who have DVT”.

It is estimated there will be more than 46,000 cases of acute DVT in England and Wales this year, with that figure rising to 50,000 within the next four years.

Xarelto is orally administered, enabling patients to avoid the process of regular monitoring through blood tests, dosage adjustments and concerns over diets due to the existing treatment’s interaction with certain food groups.

Dr Gerry Dolan, Consultant Haematologist, Department of Haematology, Nottingham University Hospital, said the guidance provides patients with “an important new therapy choice”.

He added: “Xarelto is proven as an effective agent for DVT treatment which removes some of the challenging constraints of current standard therapy, and can help re-shape and improve anticoagulation services by reducing our reliance on regular coagulation monitoring.”

Allergan’s Botox (botulinum toxin type A) has been recommended in final NICE guidance as a treatment option for adults who experience chronic migraine.

The popular cosmetic injection has been recommended in adults whose condition has not responded to three other preventative medicines but has previously been appropriately managed.

Professor Carole Longson, Director of the Heath Technology Evaluation Centre at NICE, said the Institute was pleased to recommend Botox for the “extremely debilitating” condition.

The guidance recommends Botox may be used on the NHS as an option unless a patient’s headaches have not improved after two cycles of treatment. Also, if a person’s ‘headache days’ have reduced to fewer than 15 days a month over three months then treatment should stop.

A chronic migraine is defined as headaches on at least 15 days per month, of which at least 8 days are with a migraine. It’s believed that around 1.6% of adults are affected with the condition.

The guidance applies to NHS settings in England and Wales. In April last year, the Scottish Medicines Consortium advised against the use of the injection to treat the condition.

NICE’s decision to recommend the use of Zytiga (abiraterone) in final guidance to treat prostate cancer has left Janssen “delighted”.

The treatment originally failed to get backing from NICE in combination with prednisone or prednisolone after the Institute deemed it to be too expensive – despite its clinical benefits.

However, after Janssen provided additional patient data and a revised Patient Access Scheme (PAS), NICE said it was happy to recommend the treatment as an option for the NHS.

Martin Price, External Affairs Director, Janssen UK, said the company had gone to “significant lengths” to find a solution that allows patients to be treated with the “innovative, UK discovered medicine, routinely on the NHS”.

The decision to recommend the treatment has also been backed by Dr Heather Payne, Consultant Clinical Oncologist, University College London Hospitals. She commented: “This is good news for patients – for whom historically there have been few treatment options available – and also for the patients’ families.”

Zytiga may potentially extend the lives of patients by more than three months. It is now recommended in combination with prednisone or prednisolone as a treatment option for castration-resistant metastatic prostate cancer that has progressed on or after one docetaxel-containing therapy.

NICE has recommended Xarelto (rivaroxaban) in final guidance as an option for the prevention of stroke and systemic embolism in people with atrial fibrillation (AF).

The recommendation means patients will get access to the first single tablet, once-daily, oral stroke prevention medicine since the introduction of warfarin in the 1950s

Luis-Felipe Graterol, Medical Director, Bayer HealthCare UK, said the company is “delighted” with NICE’s decision and will now work with local NHS fundraisers to “help evolve services” with Xarelto.

Up to 700,000 people in England and Wales have AF. People with the condition are at a higher risk of developing blood clots and subsequent stroke.

Xarelto is an orally administered drug that helped prevent blood from clotting and has a UK marketing authorisation for the prevention of stroke and system embolism in those with non-valvular AF who have associated risks.

The guidance adds that the decision to swap treatment from warfarin to Xarelto should be discussed between the clinician and patient to highlight any reported risks and benefits.

“We know that some people taking warfarin can find it difficult to maintain their blood clotting at a proper level,” said Professor Carole Longson, NICE Health Technology Evaluation Centre Director. 

“Rivaroxaban, like dabigatran etexilate, which NICE recently approved as an option for this indication, can benefit people with AF. We are therefore pleased to recommend rivaroxaban as another cost-effective option for the prevention of stroke and systemic embolism in people with atrial fibrillation.”

NICE recommended Boehringer Ingelheim’s Pradaxa as a treatment option in final guidance for the same indication back in March.

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 recommended Novartis’ Tasigna (nilotinib) and Glivec (imatinib) for the first line treatment of chronic myeloid leukaemia (CML), but failed to recommend BMS’ Sprycel (dasatinib) in final guidance.

Despite evidence demonstrating Sprycel and Tasigna to be more clinically effective than standard dose Glivec, Sprycel’s cost swayed NICE’s decision after Novartis agreed to supply Tasigna at a reduced price.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, says the cost reduction “enabled” the independent Appraisal Committee to recommend its use on the NHS.

The appraisal incorporates a partial review of guidance published in October 2003 which recommended standard dose Glivec for first line treatment of CML.

NICE’s independent Appraisal Committee considered results of clinical trials that showed statistically more people receiving Sprycel and Glivec had a complete cytogenetic response and a major molecular response compared with people using Glivec after a 12 month review.

The Committee also noted the opinions of clinical specialists and patient experts who suggested that Tasigna and Sprycel were more effective than standard dose Glivec – despite it being a “very effective” option for the majority of patients.

Sprycel and Tasigna both cost more than £30,000 per patient, per year. Standard dose Glivec costs in the region of £20,000. However, after Novartis had agreed a Patient Access Scheme with the Department of Health for Tasigna, NICE deemed it was a cost effective use of NHS resources.

“The recommendations reaffirm the use of imatinib as an effective treatment for the majority of patients and a cost effective use of NHS resources, and we are also very pleased to be able to add a further treatment option for these patients by recommending nilotinib,” said Professor Longson.

“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.

“However, the manufacturer of nilotinib has already agreed with the Department of Health to provide the drug to the NHS at a discounted price.”

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

Gilenya (fingolimod) has become the first pill-based treatment for highly active relapsing-remitting multiple sclerosis (RRMS) recommended on the NHS after being backed in final guidance by NICE.

NICE had originally failed to recommend the pill in draft guidance but reconsidered its decision after Novartis and clinicians provided additional information and analysis on the effectiveness of the medication.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, says the convenient treatment “is a valuable new therapy” for patients with RRMS.

RRMS is estimated to affect around 27,500 people in England and Wales and is the most common type of multiple sclerosis. Treatments to manage relapses are usually administered by injection.

NICE now specifically recommends the “innovative” treatment for adults who have an unchanged or increased relapse rate or ongoing severe relapses compared to the previous year, despite treatment.

But NICE states the recommendation only applies if Novartis provides Gilenya under the proposed terms in its confidential Patient Access Scheme it agreed with the DH.

“We are pleased to recommend fingolimod as a treatment option for the specific patient population for whom it has been demonstrated to be cost effective, providing Novartis applies its proposed discount,” said Professor Longson. “Multiple sclerosis can be a disabling condition and so we hope that this novel treatment will help to reduce relapses for these people.”

The treatment recently had its label updated to include new prescribing guidelines after the EMA and FDA raised concerns over cardiovascular events in certain patients.