NICE has not recommended Novartis’ Jakavi (ruxolitinib) in final draft guidance for the treatment of disease-related splenomegaly or symptoms in adults with myelofibrosis.

An independent appraisal committee concluded the treatment is clinically effective but raised concerns around the manufacturer’s economic model and many of its assumptions.

Sir Andrew Dillon, NICE Chief Executive, said the regulator had to be sure treatments are clinically and cost effective otherwise “money has to be diverted from elsewhere” to fund such drugs in the NHS.

The guidance states that Jakavi is not recommended for the treatment of disease-related cases of an enlarged spleen or its symptoms in adults with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.

Myelofibrosis is a rare type of blood cancer in which the bone marrow produces too many cells too rapidly. This affects the bone marrow making it less able to create cells. Other organs, such as the liver and spleen, then compensate for this by producing additional cells. The spleen, as it begins to create cells, becomes enlarged.

NICE noted that Jakavi was clinically effective in reducing the size of the spleen in these cases and related symptoms. However, it could not be considered a cost-effective option of NHS resources when compared with existing treatments.

The committee found there were “fundamental issues” with the structure of the economic model supplied by Novartis. It concluded that the associated assumptions made increased the uncertainty of the cost-effectiveness ratio (ICER) for the treatment and that rectifying this would actually increase the ICER.

Final guidance is now expected in June 2013.

NICE has recommended the use of Esbriet (pirfenidone) in final guidance for certain patients with idiopathic pulmonary fibrosis.

The treatment has been backed as an option for patients who have a forced vital capacity – the amount of air which can be forcibly exhaled after a deep breath – predicted between 50% and 80% of the normal level.

The Institute predicts that its guidance will see around 6,800 patients with the chronic lung condition becoming eligible to use the treatment.

The guidance also advises clinicians that patients should stop taking Esbriet if their disease continues to worsen.

Esbriet is manufactured by InterMune.

Bristol-Myers Squibb’s Orencia (abatacept) has been recommended by NICE as an option to treat rheumatoid arthritis after conventional treatments have failed.

NICE backed the drug following a rapid review of a previous negative appraisal after BMS submitted a patient access scheme to lower the price of the treatment for the NHS.

It is now recommended in combination with methotrexate only if the disease has responded inadequately to two conventional disease-modifying anti-rheumatic drugs (DMARDs) and is used in line with the recommendations for other types of these medicines.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, said the guidance will “widen the choice of treatments” available to patients and the health service.

Orencia was not recommended for the same indication in August 2011 as a second line treatment after concerns were raised by NICE over its cost effectiveness when compared to alternatives.

However, the treatment has been backed alongside other treatments as an option for rheumatoid arthritis patients if there has been an inadequate response to one or more TNF inhibitors and if individuals cannot receive MabThera (rituximab) because it is contraindicated or withdrawn because of an adverse event.

Cell Therapeutics’ Pixuvri (pixantrone) has not been recommended by NICE in draft guidance as a treatment option for an aggressive form of non-Hodgkin’s lymphoma.

NICE’s independent appraisal committee decided there was insufficient evidence to show the drug works more effectively than existing options and raised question marks over its cost.

Sir Andrew Dillon, NICE Chief Executive, said that clinical trial evidence left NICE with a “number of uncertainties” and questioned how many people may actually benefit from the treatment.

Non-Hodgkin’s lymphoma is the sixth most common cancer in the UK. In 2010, around 12,000 people were diagnosed with the disease. However, Cell Therapeutics estimates that only around 1,650 people with an aggressive form of the disease would be eligible to receive the treatment, if recommended.

Pixuvri has a conditional licence to treat adults with aggressive non-Hodgkin B-cell lymphoma that has either returned after treatment or become resistant to current therapy in those who have received at least two previous types of treatment.

NICE was developing guidance for this specific group of people but was told by clinical experts that the analysis supplied by the manufacturer did not include comprehensive data in patients who had received MabThera (rituximab) – an integral part of first-line treatment and often used in second-line therapy.

Because of this, the committee concluded that differences between previous treatment between the trial population and current practice meant there was considerably uncertainty in determining the treatment’s clinical effectiveness for a UK population.

The recommendation is now open for consultation until 1 May 2013.

Novartis’ Lucentis (ranibizumab) has been recommended in final guidance as an option for treating visual impairment caused by diabetic macular oedema (DMO).

NICE completed a rapid review of the original guidance it published in November 2011 after Novartis submitted an improved patient access scheme (PAS) and updated data showing the drug’s superior relative effect in certain patients.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the Institute reversed its 2011 guidance after analysing the updated data supplied by the manufacturer.

The injection is now recommended as an option if the eye has a certain central retinal thickness at the start of treatment and if it is supplied under the PAS terms agreed with the DH.

The terms of the revised PAS remain confidential between Novartis and the DH. However, NICE confirmed the scheme will remain in place until any review of the guidance is published.

The final guidance now replaces any local recommendations across England.

NICE has recommended the use of Eliquis (apixaban) as a treatment option for the NHS for the prevention of stroke and systemic embolism in some people with non-valvular atrial fibrillation (AF) in final guidance.

The recommendation came after NICE’s Appraisal Committee concluded Eliquis was more clinical effective than warfarin in reducing stroke and systemic embolism.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said that patients would benefit using the convenient treatment “because it doesn’t require such regular monitoring and dose adjustments.”

Eliquis, which only received its license for this indication in November 2012, is an orally administered anticoagulant that helps prevent the blood from clotting.

AF is the most common irregular heart beat. People with the condition are at a higher risk of developing blood clots and subsequent stroke. Existing therapies, such as warfarin, substantially reduces the risk of stroke.

“Many people with the condition find it difficult to comply with the most commonly used antithrombotic, warfarin, because, among other things, its use requires regular monitoring of the blood’s clotting properties and dose adjustments which can cause disruption and inconvenience,” said Professor Longson.

“From the evidence submitted, the Committee concluded that Eliquis was more clinically effective than warfarin for the primary efficacy outcome of reducing stroke and systemic embolism. The Committee also noted that treatment with Eliquis resulted in fewer bleeding events than warfarin, including a reduced rate of intracranial bleeding. The Committee recognised that intracranial bleeding has a high mortality rate and a large impact on a person’s quality of life, and is the most feared bleeding outcome for people taking any type of anticoagulant.”

Eli Lilly’s Alimta (pemetrexed) has not been recommended in new draft guidance as a maintenance treatment option for non-small-cell lung cancer (NSCLC).

NICE recognised the effectiveness of the treatment but said the potential gain for patients is less than the NHS is being asked to pay for the drug.

A disappointed Sir Andrew Dillon said the Institute can only recommend treatments “which are both clinically and cost effective” for the NHS.

The Institute calculates that the average cost of the treatment is approximately £11,640.

Lung cancer is one of the most common cancers in the UK. NSCLC is the most common type of lung cancer and accounts for around 80% of all cases.

Alimta was being considered as a maintenance treatment option following induction therapy with the drug and cisplatin. It is already recommended as a first line treatment option for NSCLC and as a maintenance treatment option following platinum-based chemotherapy in combination with gemcitabine, paclitaxel or docetaxel.

“Alimta is already recommended as maintenance treatment following a different first line treatment,” said Sir Andrew Dillon. “However, in this case, as maintenance treatment following pemetrexed and cisplatin, although effective, the potential gain for patients is less but the cost to the NHS remains the same.

“It is disappointing not to be able to recommend pemetrexed in our preliminary guidance.”

Roche’s Avastin (bevacizumab) has not been recommended by NHS in draft guidance as a treatment option for advanced ovarian cancer.

Although NICE confirmed the clinical benefits of the treatment it claimed its high cost was too much to justify its use on the NHS.

Sir Andrew Dillon, NICE Chief Executive, said evidence supplied by Roche “did not show that bevacizumab justifies its very high cost and could not be recommended.”

Avastin was analysed as a treatment option in women whose cancer had returned six months or more after initial treatment with platinum-based chemotherapy.

Ovarian cancer is among the top five most common cancers in women in the UK. In 2010, there were more than 7,000 new cases diagnosed.

NICE’s Appraisal Committee found Avastin may help the delay of cancer in patients for a limited time. But determined its cost was too much to expect the NHS to pay.

“We understand there are limited treatment options available to women with recurrent advanced ovarian cancer and it is always disappointing when we are not able to recommend a treatment,” said Sir Andrew. “However, it is important to remember that there are other treatments already available in the NHS for treating this condition.”

NICE has requested further information on Forxiga (dapagliflozin) as a combination therapy option to treat type 2 diabetes after failing to recommend the product in draft guidance.

Bristol-Myers Squibb and AstraZeneca have been asked for further clarification and information after concerns were raised about trial data and the cost modelling of the drug.

Forxiga has a UK marketing authorisation in adults with type 2 diabetes mellitus. It’s used as monotherapy when diet and exercise alone do not provide adequate control and as an add-on combination therapy with other treatments, such as insulin and metformin.

During the appraisal, NICE’s independent Appraisal Committee questioned the evidence on the clinical effectiveness of Forxiga as an add-on to insulin and metformin.

The Committee noted that the trial data for the drug as an add-on therapy to insulin came from two placebo-controlled trials – one of which was only 12 weeks in duration. The evidence supplied for Forxiga as an add-on to metformin came from three clinical trials and a network meta-analysis.

Concerns were also raised by the Committee surrounding the comparisons made by BMS and AZ of the cost of Forxiga and with that of other anti-diabetic drug therapies and how these were initially made.

“Type 2 diabetes is a serious problem in the UK and it is important that there is a range of different treatment options available,” said Professor Carole Longson, Health Technology Evaluation Centre Director at NICE. “Unfortunately the Appraisal Committee is currently unable to recommend dapagliflozin, one of the options, for the treatment of this condition. They have requested further information from the manufacturer, which will be considered at the next Appraisal Committee meeting in April.”

Final guidance is expected in June 2013.

Savient Pharmaceuticals’ gout treatment Krystexxa (pegloticase) has not been recommended by NICE in draft guidance.

NICE’s independent Appraisal Committee decided the evidence supplied as part of the appraisal raised questions around the drug’s long-term benefits and its cost-effectiveness to the NHS.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, said that despite Krystexxa demonstrating clinical benefits “there was considerable uncertainty about the long-term efficacy and safety of pegloticase, and a very high cost compared with the known benefit”.

A consultation period on the decision not to recommend the treatment as an option for severe debilitating chronic tophaceous gout in adults has now been opened.

During the appraisal, NICE analysed evidence supplied by Savient which showed how Krystexxa effectively lowers levels of uric acid for a significant proportion of patients with the form of arthritis.

But despite its benefits to patients, NICE’s Committee noted the risk of “serious adverse reactions” and questioned its long-term benefits. The high cost of the treatment was outside the price range for treatments the NHS can afford – usually between £20,000 and £30,000.