Alimera Sciences’ Iluvien (fluocinolone acetonide intravitreal) has not been recommended within its marketing authorisation for the treatment of chronic diabetic macular oedema (DMO) in final draft guidance.

NICE again raised concerns over the economic model supplied by the manufacturer and concluded that the data did not accurately reflect current clinical practice.

Sir Andrew Dillon (pictured), Chief Executive of NICE, said “the evidence provided did not show that the benefits fluocinolone intravitreal implant provides to patients justify the price the NHS is being asked to pay.”

Iluvien was not recommended in draft guidance published in early August 2012 after NICE questioned the economic model submitted by Alimera and the evidence it supplied during the appraisal.

In response Alimera submitted an updated economic model. However, NICE says this model underestimated the incremental cost-effectiveness ratio (ICER) for Iluvien compared to optimised standard of care.

The independent Appraisal Committee concluded that the most plausible ICER well exceeded the range that NICE usually considers an effective use of NHS resources. The updated cost model also failed to take into account any possible negative effects.

Alimera also proposed a pseudophakic subgroup (with an implanted synthetic optical lens) in its modelling. NICE accepted this subgroup was reasonable, but the small numbers of patients in the evidence submitted led to uncertainties in the estimates of clinical effectiveness and economic modelling in this group.

“Unfortunately, in this case, despite additional information on a pseudophakic subgroup and updated economic modelling from the manufacturer, the Committee concluded that the evidence provided did not show that the benefits fluocinolone intravitreal implant provides to patients justify the price the NHS is being asked to pay,” said Sir Andrew Dillon.

NICE has confirmed its recommendation of Servier Laboratories’ Procoralan (ivabradine) for the treatment of chronic heart failure in final draft guidance.

The guidance states Procoralan should be taken in combination with standard therapy options or when beta-blockers are contraindicated or not tolerated – after a four week stabilisation period of standard therapy.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said the treatment “has been shown to have a beneficial effect in reducing mortality and improving quality of life in people with some types of chronic heart failure.”

Heart failure affects around 900,000 people in the UK. The most common cause of heart failure is coronary artery disease. The aim of treatment is to improve life expectancy and quality of life and to avoid patients being admitted to hospital.

“The Committee was mindful that there is robust evidence for the effectiveness of ACE inhibitors, beta-blockers and aldosterone antagonists that are used routinely in managing heart failure,” Professor Longson said.

“They concluded, therefore, that ivabradine could be considered a cost-effective use of NHS resources for treating chronic heart failure after optimal treatment with these drugs has been achieved and when patients are still symptomatic after receiving optimised initial therapies, or when beta-blockers are contraindicated or not tolerated by the patients.”

The recommendation means Procoralan can be supplied as an option for patients with systolic dysfunction, who are in sinus rhythm, whose heart rate is 75 beats per minute or more and who have a left ventricular ejection fraction of 35% or less.

Allergan’s Botox has been recommended in final draft guidance as an option for the NHS to prevent headaches in certain adults who experience chronic migraine.

NICE advises the injection can be used in patients whose condition has not responded to three prior preventative medications and whose conditions has been appropriately managed.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, said Botox is a “preventative therapy” for those with “extremely debilitating” chronic migraines.

In February, NICE failed to recommend the injection after questioning the evidence supplied by Allergan.

However, after additional information and analysis were supplied by Allergan, NICE’s independent Appraisal Committee deemed the treatment to be a good use of NHS resources.

The draft guidance adds that injections of the popular cosmetic treatment should be stopped if headaches have not improved enough after two cycles, or if the amount of “headache days” falls to fewer than 15 per month, over three consecutive months.

Chronic migraines are believed to affect around 1.6% of adults. They are defined as headaches on at least 15 days per month, of which at least 8 are with migraine.

The draft guidance is now open for consultation with final guidance expected in June.

Roche’s Tarceva (erlotinib) has been recommended in final draft guidance as a first-line option for people with EGFR mutation-positive non-small-cell lung cancer (NSCLC).

The decision comes after Roche provided NICE with additional data on the clinical and cost effectiveness of Tarceva when supplied under an agreed Patient Access Scheme (PAS).

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, said the health regulator is “pleased to recommend another treatment” for NSCLC.

NICE recently recommended Iressa (gefitinib) as a first-line treatment for NSCLC. Roche were unable to provide any clinical data comparing Tarceva with Iressa, but specialists confirmed the two were similar and equally effective.

Tarceva and Iressa work differently to chemotherapy. The oral treatments are known as “target agents” due to the way they block certain processes in the cancer cells.

Data supplied by Roche showed Tarceva showed longer progression-free survival and similar overall survival compared with current treatment options. It expects that NICE’s recommendation will benefit approximately 11% of patients with NSCLC.

The PAS agreed between Roche and the DH may mean that Tarceva is supplied to the NHS free of charge in certain circumstances. Tarceva will be made available at a single cost of £12,200 per patient irrespective of the duration of treatment. However, Roche will only invoice the health service after the third monthly pack of Tarceva is supplied. Any patients who receive only one or two months of treatment will receive the drug without the NHS being charged.

NICE’s decision not to recommend the use of Benlysta (belimumab) for the treatment of systemic lupus erythematosus (SLE) in final draft guidance has been called ‘devastating’ by GSK.

Benlysta failed to get NICE backing after its clinical benefits compared with standard options and cost effectiveness to the NHS were both questioned by the regulator – weeks after the SMC came to the same decision.

Simon Jose, General Manager, GSK UK, says the recommendations are “devastating decisions for patients with lupus whose disease is currently uncontrolled by existing therapies”.

SLE is an incurable autoimmune condition which currently affects around 15,000 people in England and Women – 90% of whom are women.

Benlysta is the first new treatment licensed in the UK for lupus in half a century after the European Commission granted marketing authorisation in July 2011.

But NICE questioned the health benefit for patients and the cost of the treatment in relation to its clinical effectiveness after analysing evidence and consulting with people with the condition and clinical specialists.

Sir Andrew Dillon, NICE Chief Executive, commented: “Whilst recognising the severity of the disease, the Committee concluded that based on this evidence, belimumab could not be considered a good use of NHS resources compared with current clinical practice.”

GSK insist that failure to recommend Benlysta sees patients being left behind those from Germany and Spain, where the treatment is already approved for use. “We remain committed to creating solutions that will help ensure that the small number of patients across the UK who we believe will benefit most, are able to access this important new medicine,” Simon Jose said.

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.

Bayer HealthCare’s Xarelto (rivaroxaban) has been recommended in final draft guidance as an option for the prevention of stroke and systemic embolism in people with atrial fibrillation (AF).

NICE revised its original decision not to recommend the convenient pill after Bayer supplied requested additional evidence on the clinical and cost effectiveness of the medication.

Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said she was “pleased” additional information had been supplied enabling NICE to recommend the treatment.

It is estimated there are currently up to 700,000 people with AF in England and Wales. People with the condition are at a higher risk of developing blood clots and subsequent stroke.

However, the risk of stroke can be reduced by the appropriate use of antithrombotic therapy. Xarelto is the first in a class of drugs known as Factor Xa inhibitors, which act at a critical point in the blood-clotting process to prevent clots forming.

It has a UK marketing authorisation for the prevention of stroke and systemic embolism in patients with non-valvular AF who have one or more risk factors and has been demonstrated in a clinical study to be non-inferior to warfarin, the current standard of care.

Professor John Camm, Professor of Clinical Cardiology at St George’s University of London, welcomed its recommendation. He said: “This news could be particularly important for patients who require long-term or lifelong anticoagulation (for non-valvular AF) who may be seeking a simplified regimen.”

Final guidance is now expected as early as next month.

NICE recently published final guidance recommending the use of Pradaxa (dabigatran) – which recently had its UK price reduced by Boehringer Ingelheim – for the same indication.

Janssen UK’s Incivo (telaprevir) has been recommended in final draft guidance as a treatment option for genotype 1 chronic hepatitis C (CHC) in adults with compensated liver disease.

NICE has recommended the treatment in combination with peginterferon alfa and ribavirin after it demonstrated significant improvements in sustained virological response rates.

Meindert Boysen, Programme Director Technology at NICE, said Incivo “represents a major benefit for people with chronic hepatitis C”.

It was estimated that in 2009 around 250,000 people in England and Wales were carrying the hepatitis C virus. Approximately 146,000 people were chronically infected.

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

Poor diagnosis and compliance rates coupled with a high incidence of new infection means that CHC presents a major public health issue, despite the availability of treatments.

The final draft guidance recommends Incivo as an option for the treatment of genotype 1 chronic hepatitis C in adults with compensated liver disease who have not received treatment, or in whom treatment with peginterferon alfa and ribavirin has failed.

Dr Martin Price, External Affairs Director, Janssen UK commented: “We welcome this final appraisal determination from NICE and we expect that genotype-1 hep C patients will now be able to access this treatment which will offer them a greater chance of clearing the hep C virus and potentially a shorter treatment duration than is available with standard treatment.”

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.”

NICE has confirmed its recommendation of Eli Lilly’s prolonged release Bydureon (exenatide) for people with type 2 diabetes in final draft guidance.

The guidance recommends the injection as part of triple or dual therapy regimen for patients when their control of blood glucose remains or becomes inadequate in certain circumstances.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, says that the estimated 2.25 million people in the UK need as many effective treatments as possible to control the condition.

The injection improves glycaemic control by lowering the rise in blood sugar as a result of eating 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 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 says that treatment with the injection for both triple and dual therapy regimens should only be continued if a beneficial metabolic response has been proven.

Metformin, sulphonylureas and thiazolidinediones are other treatments used to lower glucose levels in the blood in the management of type 2 diabetes mellitus.