NICE has issued final guidance recommending Lucentis (ranibizumab) as a treatment for visual problems caused by macular oedema.

The injectable Novartis drug is recommended as a treatment for macular oedema caused by central retinal vein occlusion (RVO), or by branch RVO where laser treatment has failed or is not suitable.

NICE recommends the drug should only be prescribed under the terms of the patient access scheme agreed by Novartis with the Department of Health.

Macular oedema occurs when fluid collects in the central area of the retina, harming the ability to see detail and colour.

The condition is associated with increased production by damaged cells of vascular endothelial growth factor (VEGF), which worsens the leakage of fluid.

The underlying cause of macular oedema is often RVO, risk factors for which include advanced age, high blood pressure and diabetes.

Lucentis, the only anti-VEGF drug currently licensed in the UK for treatment of RVO, can reduce oedema and limit (or even reverse) visual loss.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, said: “Macular oedema can cause blurred vision or a sensitivity to light, both of which can be very frightening and can significantly affect a person’s everyday life. These symptoms can come on suddenly, and if left untreated can cause blindness.

“NICE is, therefore, pleased to recommend ranibizumab in people with CRVO and some people with BRVO, following the submission of a patient access scheme, which makes the treatment more cost-effective.”

Novartis commented that the NICE announcement was “an important and long-awaited milestone in the management of this common eye disease.”

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.”

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.

Final draft guidance from NICE recommends Xarelto (rivaroxaban) to treat pulmonary embolism (PE) and deep vein thrombosis (DVT) and prevent their recurrence.

The Bayer drug offers an alternative to warfarin, the standard treatment for dangerous internal blood clotting.

Xarelto presents fewer dose management challenges than warfarin, and has fewer interactions with other drugs and with foods.

DVT, an abnormal blood clot formation in the leg or pelvis, can lead to PE and other dangerous circulatory malfunctions that cause disability or death. Risk factors for DVT include prolonged travel and/or immobility.

Suspected PE is treated with an anticoagulant, usually initial injections of heparin followed by longer-term oral doses of warfarin. However, warfarin presents complex dose adjustment challenges and can interact dangerously with other medications and with foods.

NICE determined that Xarelto was cost-effective both as a treatment for PE and DVT over three, six or 12 months and as a lifelong treatment to prevent the recurrence of PE or DVT.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said: “The regular monitoring and dose adjustment needed with warfarin, which needs regular visits to hospital or GP appointments, can be costly and inconvenient. Also, because warfarin has many drug interactions, it may be unsuitable for people with comorbidities. In addition, the Committee heard that warfarin has various food interactions which often require people to adjust and monitor their diet.

“Rivaroxaban therefore represents a significant potential benefit for people with PE and DVT because it avoids the need for initiation with heparin and the subsequent transition to warfarin.”

Final NICE guidance is expected in May 2013.

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 reversed its recommendation on Novartis’ Xolair (omalizumab) and has now backed the treatment as an option for severe and persistent allergic asthma in final draft guidance.

The decision by NICE’s Appraisal Committee comes after Novartis supplied additional analysis on the product and submitted a patient access scheme to provide Xolair at a discounted rate.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the Institute was “pleased to now be able to recommend Xolair as an effective therapy for adults, adolescents and children with this condition.”

Xolair was originally recommended for use in adults but not for use in children.

But after additional analysis the health-related quality of life benefits – which NICE said were “not quantifiable” – were acknowledged alongside the cost effectiveness of the treatment.

Final guidance is now expected to be published next month.

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.”

NICE has issued preliminary guidance not recommending a drug for myelofibrosis, a rare blood cancer, which it says is clinically effective but too costly.

Jakavi (ruxolitinib) from Novartis is provisionally refused for NHS treatment of enlarged spleen caused by myelofibrosis.

Novartis has said it is encouraged by NICE’s acceptance of the drug’s clinical value and will work to reach agreement with the Institute on cost issues.

Myelofibrosis causes scarring of the bone marrow tissue, affecting their ability to produce blood cells. The spleen becomes enlarged to compensate, which has debilitating effects.

The NICE appraisal committee concluded that Jakavi offered a “step change” in treating the condition: it reduced spleen size and symptoms such as fatigue, pain and itching.

However, it did not consider the drug, which costs £43,200 per patient per year, more cost-effective than the next best alternative.

NICE’s value model – recently criticised by the European Commission – sets a threshold price of £30,000 per quality-adjusted life year (QALY) gained. Novartis claims a price per QALY of £74,000, but NICE claims the true figure is twice that.

Professor Carole Longson, Director of NICE’s Health Technology Evaluation Centre, said: “It is disappointing not to be able to recommend this new treatment in our preliminary recommendations, but in order to do this we have to be sure that the treatment is both clinically and cost effective.”

Consultant haematologist Professor Claire Harrison commented: “The lives of patients affected by myelofibrosis are improved with ruxolitinib therapy. In many cases this improvement is dramatic with long-lasting tangible benefits.”

“We are encouraged that the Committee considers ruxolitinib to be an innovative treatment and Novartis is committed to working alongside clinicians and patient groups in this area to address all queries raised by NICE,” said Panos Alexakos, Oncology General Manager, Novartis UK & Ireland.

Final guidance is expected in June 2013.