NICE has not recommended Novartis’ Jakavi (ruxolitinib) in final draft guidance for the treatment of disease-related splenomegaly or symptoms in adults with myelofibrosis.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

Asthma treatment Xolair (omalizumab) has been recommended by NICE in final guidance as an option for treating adults, adolescents and children with severe or persistent allergic forms of the condition.

Xolair has been recommended in people aged 6 years and older as an add-on to optimised standard therapy for patients who require continuous or frequent treatment with oral corticosteroids.

However, the treatment can only be used by the NHS if Novartis Pharmaceuticals UK provides the treatment under the terms of an agreed patient access scheme to lower its price.

The treatment has a UK marketing authorisation as an add-on therapy to standard care to improve asthma with severe persistent allergic asthma. Currently, it is only prescribed to those whose condition remains poorly controlled, despite receiving standard therapy options.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, commented: “NICE is pleased to recommend omalizumab, with the agreed patient access scheme submitted by the manufacturer, as an effective add-on therapy for adults, adolescents and children with severe, persistent allergic asthma, which can have a significant effect on a person’s life.”

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.

NICE has described the existing social care provision for people with dementia as ‘patchy’ and struggling to catch up with the increased demand.

As it extends its remit to social care, NICE (now the National Institute for Health and Care Excellence) has outlined standards for dementia care provided by social services and care homes.

However, it warned that some people with dementia were not even receiving basic care to a minimum standard.

Following the DH’s national dementia nursing strategy, the new NICE guidance aims to ensure that the social care sector maintains a comparable standard to, and is effectively integrated with, NHS care.

It covers such broad aspects of life for people with dementia as appropriate housing and access to dental services and community life.

“The general picture is that care is patchy,” said NICE’s Deputy Chief Executive, Professor Gillian Leng (pictured).

“My personal view is that we are all playing catch-up because the number of people with dementia has been increasing so dramatically.”

George McNamara of the Alzheimer’s Society commented: “These standards will be a useful tool for the care sector and show what people with dementia and carers should be able to expect.”

However, he warned, “as they are not mandatory, it’s a case of ‘wait and see’ as to whether this guidance will drive real change or just sit on the shelf.”

NICE has failed to recommend Pfizer’s Xalkori (crizotinib) for previously treated anaplastic-lymphoma-kinase-positive advanced non-small-cell lung cancer in new draft guidance.

An independent Appraisal Committee decided the drug did not meet NICE’s end-of-life treatment criteria so its cost exceeded the limit deemed cost-effective for NHS use.

Sir Andrew Dillon, NICE Chief Executive, said that although the clinical benefits of Xalkori had been recognised the high cost of the drug meant it could not be considered as a treatment option.

NICE usually recommends clinically effective treatments that cost up to at a maximum of up to £30,000 per quality adjusted life year (QALY) – the methodology current used to assess value.

If certain treatments meet the criteria to be considered under NICE’s supplementary advice for end-of-life treatments a higher cost per QALY may be accepted. The highest cost per QALY NICE has recommended has been around £50,000.

However, NICE’s Appraisal Committee concluded that the most plausible cost per QALY for Xalkori would be somewhere between £63,800 and £181,100 when compared with existing treatments and between £51,700 and £80,500 when compared with best supportive care.

“We have already recommended a number of treatments for the various stages of non-small-cell lung cancer,” said Sir Andrew Dillon. “However, although the independent committee that considered the evidence found crizotinib to be clinically effective treatment for ALK-positive non-small-cell lung cancer, even if the supplementary advice to the Committee for life-extending treatments had applied, crizotinib could not be considered a cost-effective use of NHS.”

NICE’s guidance is now open for consultation.

NICE has recommended two new treatments for lung infections in people who suffer from cystic fibrosis in final guidance.

Novartis’ Tobi Podhaler (tobramycin) has been recommended for treating chronic pulmonary infection caused by Pseudomonas aeruginosa in people with cystic fibrosis only if a nebulised version is considered appropriate and is supplied under an agreed patient access scheme (PAS).

Forest Laboratories UK’s Colobreathe (colistimethate sodium) has also been recommended for the same indication if patients would benefit clinically from continued use of the treatment but do not tolerate its nebulised form and Podhaler therapy would otherwise be considered. The treatment must also be supplied under the terms of an agreed PAS.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the Institute was “pleased” to recommend the treatments for one of the primary causes of death in people with CF.

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

NICE has revised its guidance on two drugs from a negative to a positive recommendation, based on new patient access schemes.

Esbriet (perfenidone) from InterMune is provisionally recommended for treatment of some patients with idiopathic pulmonary fibrosis (IPF), a progressive and serious lung condition.

Revolade (eltrombopag) from GSK is provisionally recommended for treatment of some patients with chronic immune (idiopathic) thrombocytopenic purpurai (ITP), a bleeding disorder caused by low platelet levels.

In both cases, the drug is recommended for use only within the patient access scheme submitted to the DH by the manufacturer after previous guidance (draft guidance in the case of Esbriet).

IPF is scarring to the lung tissues without apparent cause, causing progressive inability to breathe. Esbriet, an oral medication, can slow down the growth of the scars and hence the progression of symptoms.

In its final appraisal determination, NICE recommends the drug for people with IPF whose forced vital capacity is predicted at 50–80%.

NICE’s appraisal committee noted that in addition to the patient access scheme, InterMune submitted further evidence of the drug’s potential benefits. It estimated that about 6,800 patients will receive Esbriet through the NHS.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, commented: “We are pleased that InterMune provided further evidence during our public consultation and revised its terms to make pirfenidone available to the NHS.”

ITP is a progressive condition in which falling platelet count leads to failure of blood clotting, with risk of severe bleeding. Revolade, an oral medication, increases platelet production.

NICE has provisionally recommended Revolade for treating ITP in adults who have had a splenectomy and whose condition is refractory to other treatments, and as a second-line treatment in adults for whom surgery is contraindicated.

In addition, it notes, the drug should only be used if the patient’s condition is refractory to rescue therapies or their bleeding has become severe.

NICE reviewed its original guidance, published in 2010, because GSK had submitted a patient access scheme. It also recognised that there are few clinically effective treatments for people with ITP.

Professor Longson said: “Living with ITP can have a significant effect on daily life and those affected are at risk of bleeding and subsequent bruising. NICE is, therefore, pleased to be able to recommend eltrombopag for this condition.”

NICE has again failed to recommend Roche’s cancer drug Avastin (bevacizumab) – this time in new draft guidance for the treatment of metastatic ovarian cancer.

An independent Appraisal Committee decided that Avastin is not a cost-effective treatment option after a consultation period, following initial draft guidance issued in December 2012.

Roche said it was “disappointed” by NICE’s decision and claims that Avastin is the only drug proven to improve outcomes in women with ovarian cancer in the last 15 years.

Avastin recently failed to gain a recommendation as a treatment option for advanced ovarian cancer in separate guidance with NICE again citing cost reasons.

The most recent appraisal assessed whether Avastin, when used in combination with the chemotherapy treatments paclitaxel and carboplatin, would be a cost-effective option for the NHS. Avastin’s licensed dose is 15mg per kilogram of body weight – although many doctors use lower dosages when treating patients.

NICE’s assessment considered the treatment according to its marketing authorisation and not in line with current practice, which saw its cost-effectiveness analysis fall outside the range considered for NHS use.

Roche, who did not submit a patient access scheme to lower the cost of the drug, said that women will now have to rely on the Cancer Drugs Fund (CDF) to access the treatment.

But it raised questions around the “considerable uncertainly” on how medicines currently funded by the CDF will be available to patients when the scheme ends in March 2014. Roche has called for “a clear transition plan” for such medicines to reassure patients currently using these treatments.

The updated draft guidance is now subject to a two-week appeal period.