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 GSK’s Tyverb (lapatinib) or Roche’s Herceptin (trastuzumab) with aromatase inhibitors as a first line treatment for a particular type of breast cancer in final draft guidance.

The decision is based on uncertainties over the overall survival benefits compared to existing treatments of both medicines and the high cost of the treatments.

Sir Andrew Dillon, Chief Executive of NICE, said that while the two have been shown to reduce the growth and spread of breast cancer the extent of overall survival extension “appears to be small or difficult to quantify”.

Final guidance on the appraisal is now expected in June.

The guidance only advises the use of the drugs alongside aromatase inhibitors as a first line treatment option to delay the growth of advanced breast cancer that has spread and reacts with oestrogen or progesterone and has high levels of HER2.

Alongside the clinical benefits, NICE also raised concerns around the cost effectiveness of both products. GSK estimates that the most plausible incremental cost effectiveness ratio (ICER) for Tyverb is likely to be around £74,400 per QALY gained. Roche estimates the most plausible ICER for Herceptin to be around £51,000 per QALY gained – both far in excess of the £20,000-£30,000 NICE typically deems to be a cost effective use of NHS resources.

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.

MSD’s Victrelis (boceprevir) and Janssen’s Incivo (telaprevir) have been recommended in final guidance for the treatment of genotype 1 chronic hepatitis C in adults with compensated liver disease.

The guidance recommended the treatments after they were shown to improve sustained virological response rates compared to existing options.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, says the new recommendations “represents a major benefit for people with chronic hepatitis C”.

Figures from 2009 suggest that around 250,000 people were carrying the hepatitis C virus in England and Wales, of whom 146,000 were chronically infected.

Genotype 1 is the most common subtype of hepatitis C in England and Wales with between 40% and 50% affected. It is also the most resistant to treatment.

Existing NICE guidance recommends pegylated interferon and ribavirin combination therapy for people with genotype 1 chronic hepatitis C.

Its new recommendation advises Victrelis as an option in combination with peginterferon alfa and ribavirin in adults who have not received treatment or when treatment has failed.

For Incivo, the guidance recommends the treatment as an option in combination with peginterferon alfa and ribavirin in individuals previously untreated, in whom treatment has failed, or for those whose treatment has relapsed, partially responded or did not respond.

“The significant improvement in sustained virological response rates seen with boceprevir plus peginterferon alfa and ribavirin, and telaprevir plus peginterferon alfa and ribavirin, compared to peginterferon alfa and ribavirin alone represents a major benefit for people with chronic hepatitis C,” said Professor Longson.

“The Committee also acknowledged the significant public health impact that a sustained virological response can have in reducing transmission of the hepatitis C virus to uninfected people. We are pleased to be able to recommend boceprevir and telaprevir as a cost effective use of NHS resources.”

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 reversed its original decision on Novartis’ Gilenya (fingolimod), the first pill to treat multiple sclerosis, in final draft guidance.

Gilenya is now recommended after NICE assessed a public consultation which Novartis and clinicians provided additional information and analysis on the effectiveness of the medication.

Sue Webb, General Manager, UK & Ireland and Country President, Novartis UK, said the treatment “will make a real difference” to people living with MS.

The recommendation follows two previously failed appraisals. After the first in August 2011, Novartis submitted a Patient Access Scheme (PAS) to provide a discount to the NHS.

But, following another consultation, NICE said the MS pill still did not represent value for money in a separate draft guidance document last December.

However, during the second consultation period, Novartis revised its analysis for a subgroup of patients for the licensed population. Following this, NICE’s independent Appraisal Committee now believes the treatment offers value for money to the NHS, when provided under the terms of the PAS.

The subgroup of patients includes adults with highly active relapsing-remitting multiple sclerosis, whose relapses have increased or remained compared to the previous year, despite the use of beta interferons.

NICE now provisionally recommends the treatment for adults who have an unchanged or increased relapse rate or ongoing relapses compared to the past year, despite treatment.

Dr Eli Silber, a consultant neurologist at King’s College Hospital, said she was delighted NICE reversed its original decision. “We’ve waited a long time for an effective oral treatment to offer patients who are continuing to relapse on first line injections,” said Dr Silber. “Today’s decision increases treatment choice. Because it is a highly effective oral agent it may change the way MS is managed in the UK forever.

“With more active forms of MS, we have a limited window of opportunity to make a difference to patients’ lives – many are young people who are raising families and starting their careers. I want to get appropriate patients onto this therapy as quickly as possible.”

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.

NICE has recommended Novartis’ Tasigna (nilotinib) for the treatment of chronic myeloid leukaemia (CML) but failed to recommend Sprycel (dasatinib) and high dose Glivec (imatinib) in final guidance.

The Appraisal Committee concluded all three treatments provided clinical benefit for people with Glivec-resistant CML but opted for Tasigna when provided under the terms of a patient access scheme.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, says that although Tasigna is “expensive” the discount enabled the Committee to approve its use.

NICE estimates that Tasigna costs more than £30,000 per patient, per year.

The Institute received two appeals on its final draft guidance not to recommend Bristol-Myers Squibb’s Sprycel and Novartis’ Glivec, but both of these were dismissed on all accounts.

When completing the FAD, it found that patients with Glivec intolerance generally had a higher response rate to Tasigna and Sprycel than those with Glivec-resistant CML. But a lack of evidence base meant the magnitude of the benefit was uncertain.

When considering the updated analysis submitted during consultation on the first draft from Novartis, the Committee concluded that the ICER of £22,800 per QALY gained for Tasigna compared with hydroxycarbamide was optimistic. But, when provided at a discounted rate, it was regarded as a cost-effective use of NHS resources.

However, the ICER for Sprycel compared with hydroxycarbamide would be more than £43,800 and could rise further. The Committee also noted that high-dose Glivec was more expensive and less effective than the other treatments under evaluation.

“We are very pleased to be able to recommend nilotinib as a treatment option for the chronic and accelerated phases in people who are resistant or intolerant to standard dose imatinib,” said Professor Longson.

CML is a very rare condition that affects around 560 in the UK each year. If treatment with Glivec does not work, current options include interferon-alfa, hydroxycarbamide or a bone marrow transplant.

NICE has recommended the use of Roche’s RoActemra (tocilizumab) for the treatment of systemic juvenile idiopathic arthritis (JIA) in final guidance.

The Final Appraisal Decision (FAD) recommends RoActemra’s use in children aged two and over where specific treatments have not produced adequate response and the treatment is provided as part of a Patient Access Scheme.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, says the recommendation “is good news for children with systemic JIA and those caring for them”.

JIA has no known cause. Systemic JIA may start with symptoms such as a fever or rash and eventually lead to joints becoming swollen and inflamed. It affects children of any age and causes severe pain which effects everyday life.

The full recommendation by NICE states that RoActemra can be used as a treatment option for youngsters with systemic JIA whose disease has not responded well to non-steroidal anti-inflammatory drugs (NSAIDs), systemic corticosteroids and methotrexate.

However, the treatment is not recommended in those whose disease continues to respond to methotrexate, or who have not been treated with methotrexate.

“We are pleased to be able to recommend tocilizumab for systemic juvenile idiopathic arthritis,” said Professor Longson.

“This arthritic condition has a huge impact on the lives of the children and young people with the disease, causing severe pain, fatigue and disability. This affects the child’s family and school life, as well as their physical and emotional well-being.”