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to one or more TNF inhibitors and if individuals cannot receive MabThera (rituximab) because it is contraindicated or withdrawn because of an adverse event.

Draft guidance from NICE does not recommend Avastin (bevacizumab) for treatment of recurrent advanced ovarian cancer.

NICE’s appraisal committee determined that the Roche drug, when used in combination with the chemotherapy drugs gemcitabine and carboplatin, did not represent good value for the NHS.

The main reason for the decision was that clinical data were unavailable for a third of clinical trial participants, for reasons unknown to NICE.

Recurrent advanced ovarian cancer, when the cancer has returned following initial treatment and has spread beyond the ovaries, is terminal. However, NICE did not accept that Avastin qualified as an end of life treatment.

Roche’s submission highlighted the fact that Avastin together with chemotherapy offers a median progression-free survival benefit of four months more than chemotherapy alone.

However, NICE stated that “the data from around 30 of the patients had been censored” and the impact of that on progression-free survival rates was “unclear”.

The committee further noted that Roche’s estimated ICER of £149,050 per QALY gained “was likely to be optimistic”.

In addition, it said, there was insufficient evidence of overall survival benefit, and there was no patient access scheme. The latter has become a key deal-breaker for NICE in recent years.

Ovarian cancer affects around 7,000 new patients in the UK each year, and Roche estimates that over 2,000 women would be eligible for treatment with Avastin if it were approved in this indication.

Avastin has received several NICE rejections in recent years, as it offers some progression-free survival benefit but is costly. Many UK patients currently receive it via the Cancer Drugs Fund, which is soon to be discontinued.

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 approved tobramycin inhalation powder, and the inhaler used to apply it, for treatment of a dangerous complication of cystic fibrosis (CF).

The TOBI Podhaler from Novartis has received final approval from NICE as a cost-effective therapy for Pseudomonas aeruginosa (Pa) lung infection in CF patients aged six and older.

The dry powder therapy is approved as part of a patient access scheme and when the standard treatment, nebulised colistimethate, is not tolerated or not effective.

Tobramycin inhalation powder saves the patient half an hour treatment time per day relative to the nebulised version of the same drug.

Pa infection is the main cause of premature death in people with CF, a genetic disorder that makes the lungs susceptible to infections. CF affects more than 9,000 people in the UK, and only half of CF patients live beyond the age of 41.

The Podhaler delivers tobramycin inhalation powder via a small hand-held inhaler, taking only six minutes for each dose compared to 20 minutes for each nebuliser dose. In addition, the inhaler does not need a power source, and the dry powder capsules do not need to be refrigerated.

Trials have shown the dry powder to have similar clinical efficacy to the nebulised drug, with clear benefits in terms of patient convenience. It was launched in the UK in 2011.

Dr Diana Bilton, Consultant Respiratory Physician at Royal Brompton Hospital, London, said: “Our experience is that patients really benefit from this antibiotic inhaler in terms of a reduction in treatment burden and being able to get on with life instead of spending time on a nebuliser.”

NICE has recommended the use of GSK’s Revolade (eltrombopag) in draft guidance for treating chronic immune (idiopathic) thrombocytopenic purpurai (ITP).

Original guidance, published in October 2010, was reviewed by NICE after GSK agreed a Patient Access Scheme with the DH to supply the treatment at a discounted rate.

The guidance now recommends Revolade in adults who have had their spleen removed and whose condition is refractory to other treatments and as a second-line treatment in patients who have not had their spleen removed due to surgery being contraindicated.

ITP is a bleeding disorder caused by abnormally low levels of platelets in the blood. It is estimated there are around 3,000-3,500 cases in the UK.

Revolade increases platelet production through activation of the thrombopoietin receptor. By stimulating production, the drug helps to reduce bleeding.

“People living with ITP are at daily risk of bleeding and subsequent bruising, and this can have a detrimental effect on quality of life,” said Dr Carole Longson, Health Technology Evaluation Centre Director at NICE. “NICE is, therefore, pleased to be able to recommend eltrombopag for this condition.”

Final guidance is now expected to be published in May 2013.

NICE has reversed its opinion on the use of Bristol-Myers Squibb’s Yervoy (ipilimumab) and Roche’s Zelboraf (vemurafenib) for the treatment of advanced malignant melanoma.

The new final draft guidance recommends the use of Yervoy in people who have received prior chemotherapy.

Zelboraf is also recommended for the treatment of unresectable locally advanced or metastatic BRAF V600 mutation-positive melanoma.

The U-turn came after both manufacturers agreed to supply the treatments at a discounted rate under the terms of separate patient access schemes with the Department of Health.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the updated guidance was “really good news” for patients with skin cancer.

“Vemurafenib and ipilimumab are breakthrough treatments that can potentially significantly affect prognosis for these patients and we are very pleased that the manufacturers have worked with us so that we are now able to recommend both ipilimumab and vemurafenib,” said Professor Longson.

Since the publication of the first draft guidance, which NICE failed to recommend the use Yervoy due to its £80,000 price tag, BMS provided additional data and analysis surrounding the cost-effectiveness of the drug.

Roche also supplied additional analysis on the effectiveness of the drug in relation to its clinical and cost effectiveness.

Cancer patients whose disease has spread from a solid tumour to their bones have now been given a new treatment option after NICE backed the use of Amgen’s Xgeva (denosumab).

Xgeva has been recommended to treat the condition known as bone metastases in people suffering from breast cancer or solid tumours other than prostate who would otherwise be prescribed bisphosphonates.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, said Xgeva was a “welcome addition” alongside existing treatment options.

Final guidance states that Xgeva should only be prescribed under the terms agreed between the Department of Health and Amgen as part of a patient access scheme.

Amgen estimates there are around 150,000 patients in the UK with solid tumours and bone metastases, of which breast and prostate cancer account for more than 80%.

“We’re pleased to be able to recommend another treatment option for people with bone metastasis from most solid cancer tumours,” said Professor Longson. “This type of metastasis can reduce a person’s mobility and quality of life in general, increasing the risk of complications from bone weakness.”

NICE has recommended the use of Novartis’ Tobi Podhaler tablets but failed to back Forest Laboratories UK’s Colobreathe tablets for treating cystic fibrosis patients with a pseudomonas lung infection.

The draft guidance recommends a nebulised version of Tobi Podhaler if it is considered appropriate when nebulised Colobreathe is contraindicated, not tolerated or has not produced adequate clinical response.

Tobi Podhaler tablets should also only be prescribed under the terms of an agreed patient access scheme between Novartis and the Department of Health.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the Institute was “pleased to recommend” another treatment option for patients.

Cystic fibrosis is one of the UK’s most common life-threatening inherited diseases. It currently affects around 8,000 people – although more than two million people carry the faulty gene.

Sufferers are prone to lung infections by a range of pathogens. The aim of treatment is to clear the respiratory sections to maintain lung function whilst reducing inflammation and bacterial growth. Although there is no cure, treatment includes regular physiotherapy, antibiotics, and inhaled mucolytics through a nebuliser.

Tobi Podhaler is inhaled using a breath activated hand-held device which works by reducing the amount of bacteria in the lungs. NICE’s independent Appraisal Committee concluded the Podhaler was a cheaper and more effective option than a nebulised option.

However, the Committee said the economic analysis for Colobreathe was less effective and less costly than nebulised Tobi Podhaler. Additionally, analysis could not convey that Colobreathe is a cost-effective use of NHS resources.

The draft guidance is now open for consultation. NICE expects to publish final guidance in March 2013.