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 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.

NICE has again failed to recommend the use of Eisai’s breast cancer drug Halaven (eribulin) after further questioning the treatment’s side-effects in new guidance.

Eisai had provided additional data after NICE’s original decision not to recommend the treatment back in November.

But NICE concluded the data, which analysed women previously treated with Xeloda (capecitabine), did not show “robust” survival advantage and decided there no “convincing cost effectiveness” for its use.

The Institute received one appeal on its earlier draft guidance. This was dismissed on all counts; however, the Appeal Panel did recommend that sections describing the adverse events experienced with the drug and its comparator were revised.

Halaven has been shown to potentially extend the life of women by 2.7 months compared with a ‘treatment of physician’s choice’. However, the cancer drug did not fulfil all of NICE’s end-of-life criteria.

Sir Andrew Dillon commented: “Although the evidence presented to the independent Advisory Committee indicated that eribulin may help some patients live for a little longer, it also caused more undesirable side effects than other treatments already available, and the committee felt that eribulin’s effects on health-related quality of life had not been adequately assessed.”

NICE’s Chief Executive added that the Committee heard from practising clinical experts who told the Institute that patients usually receive sequential treatment with Navelbine (vinorelbine), Xeloda and infrequently Gemzar (gemcitabine).

But Sir Andrew said that experts “stressed” that if Halaven were to be recommended it would “be unlikely” to replace existing treatments “because of its related side-effects”.

Common adverse effects of the treatment include fatigue, alopecia, peripheral, neuropathy, nausea, neutropaenia, leukopaenia and anaemia.

Eisai have again claimed they are “dismayed” at the decision and accused NICE of “not giving enough support to women” and the “physicians who want to treat them”.

Nick Burgin, European Director of Market Access, said: “We hope that NICE grant a rapid re-review as new data is constantly emerging that will help inform their decision.”

Despite the lack of NICE recommendation, Halaven is available through the Government’s Cancer Drugs Fund and is one of the top twelve drugs prescribed through the system.

On the same day as the final NICE guidance, Eisai signed a partnership deal with Valeant Pharmaceuticals to promote and distribute Halaven in eight central and eastern European countries.

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

The treatment is the first new oral-anticoagulant in nearly 60 years to be recommended after NICE concluded it was more effective than warfarin.

Boehringer Ingelheim called the appraisal a “landmark decision”.

The recommendation comes after a failed appeal on the draft guidance by NHS Salford who argued that NICE had failed to act fairly and had exceeded its powers.

It claimed NICE had underestimated the likely use of Pradaxa and that its recommendation would require a design overhaul of anticoagulant services at a PCT level.

But its objections were dismissed on all points by an independent Appeal Panel chaired by NICE’s chair Professor Sir Michael Rawlins.

The guidance recommends Pradaxa as a treatment option in accordance with its licensed indications, and that treatment should start after an informed discussion about its risks and benefits compared with warfarin.

Pradaxa has a UK marketing authorisation for the prevention of stroke and systemic embolism in adults with nonvalvular AF who have previously suffered a stroke, transient ischaemic attack or systemic embolism and other heart related problems. It is also authorised for those aged 65 years old or over with AF who have diabetes, coronary heart disease or hypertension.

It’s estimated that there are around 700,000 people with AF in England and Wales. Boehringer believes that the use of Pradaxa twice daily has the potential to prevent around 530 more strokes per 100,000 compared to warfarin. And, if all eligible patients received Pradaxa twice daily, it could prevent up to 5,000 strokes and save the NHS up to £59 million in the first year of use.

Charles De Wet, Medical Director at Boehringer Ingelheim, said “We are committed to sharing value and our priority and commitment now is to work closely with the Trusts to ensure dabigatran is accessible to all eligible patients on the NHS and appropriately prescribed by clinicians”.

Eli Lilly’s Bydureon (exenatide) has been recommended in final guidance in triple or dual therapy regimens as a treatment option for people with type 2 diabetes.

The injection is now available for use on the NHS for patients when their control of blood glucose remains or becomes inadequate in certain circumstances.

Professor Carole Longson, Director, Centre for Health Technology Evaluation at NICE, says that Bydureon has been shown to be a “clinically and cost effective treatment option”.

The convenient prolonged release treatment improves glycaemic control and prevents hyperglycaemia.

In triple therapy regimens in combination with metformin and a sulphonylurea, or metformin and thiazolidinedione, Bydureon is recommended if a person has a certain body mass index (BMI) and has specific psychological or medical problems associated with their weight, or if therapy with insulin would have significant occupational implications or weight loss would benefit other significant obesity-related comorbidities.

Bydureon is also recommended in dual therapy regimens in combination with metformin or a sulphonylurea if either of the two together or separately is contraindicated or not tolerated or treatment with thiazolidinediones and DPP-4 inhibitors is contraindicated or not tolerated.

NICE notes that treatment with the medication for both regimens should only be maintained if a beneficial metabolic response has been demonstrated.

Diabetes UK estimates that around 2.25 million people in the UK now suffer from type 2 diabetes.

Eliquis (apixaban) has been recommended in final NICE guidance as a treatment option for the prevention of venous thromboembolism (VTE) in adults who have undergone planned total hip replacement or total knee replacement surgery.

The treatment, which was launched in the UK in September 2011, was fast-tracked by NICE after the convenient drug was proven to help prevent blood clots.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, says that Eliquis “has been shown to be a clinically and cost effective option”.

Hip and knee replacement surgery has a high risk of VTE developing and patients require effective treatment to prevent potentially fatal clots forming. Without the use of suitable medication, the incidence of deep vein thrombosis ranges from 41% to 85% after elective knee surgery and to 42% to 57% after elective hip surgery.

“VTE is often difficult to spot because patients may not always have symptoms to give a warning,” said Professor Longson. “Even if a blood clot does not come loose, it can still cause long-term damage to the veins – for example, ‘post-thrombotic syndrome’ may develop up to two years following DVT, causing chronic swelling and ulceration of the legs. We are therefore pleased to be able to recommend its use (Eliquis) in the NHS in England and Wales alongside other effective treatments already approved by NICE.”

Eliquis was also approved for the same indication by The Scottish Medicines Consortium (SMC) in December 2011.

NICE has issued final guidance to the NHS not recommending the use of Erbitux (cetuximab), Avastin (bevacizumab) and Vectibix (panitumumab) for the treatment of metastatic colorectal cancer that has progressed after chemotherapy.

Concerns were raised by NICE’s Appraisal Committee surrounding the clinical evidence of the products, especially the use of Avastin.

Sir Andrew Dillon, NICE Chief Executive, says the Institute is “disappointed” not to be able to add the three drugs to the six other recommended options available to the NHS.

No appeals were received on the final draft guidance which did not recommend the treatments after the evidence was considered.

Uncertainness were raised over the data supplied by Roche on Avastin plus non-oxaliplatin chemotherapy of its overall survival gain when used as a second or third-line treatment for those who had not responded to first-line or second-line chemotherapy.

Similar queries were also asked by the Committee of the estimates supplied by Merck Serono on Erbitux plus Campto based on the mixed treatment comparison, and on the magnitude of the survival benefit of Vectibix relative to best supportive care provided by Amgen.

“We have to be confident that the benefits that drugs offer patients really do justify what the NHS will have to pay for them,” said Sir Andrew.

“The independent appraisal committee which drafted the recommendations does not feel it has enough evidence, especially in the case of bevacizumab, to feel confident in recommending these drugs for use on the NHS.”

Campto (irinotecan), Eloxatin (oxaliplatin), Xeloda (capecitabine), tegafur with uracil and Erbitux (cetuximab) have all previously been recommended for various stages of colorectal cancer for use on the NHS.