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The draft guidance recommends a nebulised version of Tobi Podhaler if it is considered appropriate when nebulised Colobreathe is contraindicated, not tolerated or has not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The draft guidance is now open for consultation.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.

Too many children are being admitted to hospital with throat infections that could be treated in primary care, according to a new study.

Researchers at Imperial College London found the number of children admitted to hospital with throat infections has increased by 76% over the past decade.

The increase correlates with a 115% increase in children’s short hospital stays (less than two days), and both point to over-reliance on A&E rather than GPs.

Children aged between one and four years showed the highest rates of acute admissions for throat infections.

The researchers did not find any evidence that the infections are more severe and thus demanding of secondary care.

The four-hour waiting time target in A&E is a major reason for the increase in admissions, the report said, with doctors making decisions under time pressure.

Difficulties in GP access were also identified as a factor in driving the use of A&E as a short cut to treatment.

Lead study author Dr Elizabeth Koshy commented: “Our findings relating to short hospital stays suggest that many of the children admitted with acute throat infections could have been effectively managed in the community.

“Our study highlights the need to urgently address the issue of healthcare access, with improved models of integrated care within primary and secondary care, to avoid potentially unnecessary hospital admissions for relatively minor infections.”

The end to the ‘postcode lottery’ will have to wait a little longer. Hopes were raised earlier this year when the Department of Health unveiled plans to introduce an ‘innovation scorecard’. The scheme would prevent hospitals blacklisting expensive drugs recommended by NICE. Patients, regardless of their location, would be able to receive the latest treatments without delay.

The DH initially planned to have the scorecard “fully implemented by the Autumn.” But, after discussions with the NHS and the pharmaceutical industry, it now looks likely that the scorecard will not be in place until the end of the year.

Speaking exclusively to Pharmaceutical Field, a DH spokesperson said talks were still ongoing between the health service and pharma to “collect all the data and information needed to ensure the scheme is accurate and effective. It will be launched in the coming months.”

The new scheme is expected to work in three different ways:

1. The innovation scorecard will allow patients and the public to see which NHS organisations have adopted the latest NICE guidance on recommended drugs and treatments.
2. The NHS will no longer have an excuse not to provide patients with NICE recommended products. Treatments recommended by the Institute will now be automatically added on to local formularies, allowing doctors to prescribe more expensive treatments if they wish.
3. A new group will be established to help the NHS overcome any barriers when implementing NICE guidance. The introduction of new medication or treatment may mean big changes in the
   way services are delivered. The group aims to spread best practice across the health service.

The DH added that NHS Trusts receive funding for each new NICE appraisal, so financial issues should not be used as a barrier to the uptake of innovative new treatments.

Speaking when the details were first announced in late August, then Health Minister Paul Burstow said the “new regime” would be a “catalyst for change”. He added that the DH is “determined to eradicate variation” across the uptake of NICE approved drugs. “NHS organisations must make sure the latest NICE approved treatments are available in their area, and if they are not, then they will now be responsible for explaining why not,” he said. “Being transparent with data like this is the hallmark of a 21st century NHS. It is
a fundamental tool to help healthcare professionals improve patient care.”

The introduction of the scorecard has been backed by the ABPI. Stephen Whitehead, ABPI Chief Executive, said it would be a “valuable tool” to support the latest NICE recommendations. “There is still a great deal of variation across the country on which treatments patients are able to access and so I am hopeful the scorecard will help highlight discrepancies which can then be addressed,” he said.

Whitehead called the scorecard a “definite step forward” in ensuring patients receive the latest treatments as quickly as their European counterparts. He said the existing system was bad news for the health of the nation which resulted in a lost opportunity to “drive efficiency savings through the use of medicines”.

The NHS Confederation was equally receptive to the introduction of the scorecard. But former Deputy Chief
Executive David Stout warned its implementation may cause “unnecessary bureaucracy” and stretch NHS finances even further. “It is also important to remember that the NHS is facing an unprecedented financial challenge and organisations must live within their means while providing high quality care,” he said. “The reality is we can only afford to provide new drugs or treatments where they are cost effective and demonstrably add real patient benefits. In a health system with no financial growth, any new costs have to be offset by savings elsewhere.”

Stout added that the introduction of the scorecard will only be a success if the NHS engages with local communities and clinicians to decide what local priorities are.

Incivo, Victrelis and Mepact win recognition at the 2012 UK Prix Galien Awards.

Two new medicines for the treatment of Hepatitis C have won the 2012 UK Prix Galien Innovative Product
Award. Incivo (Janssen) and Victrelis (MSD) fought of stiff competition to win the prestigious prize at London’s House of Commons. The chairman of the judging panel, Professor Sir Michael Rawlins, said the treatments provided a perfect example of how the pharmaceutical industry can “demonstrate and justify its place in healthcare by innovating for change and showing real gains to the world.”

The ceremony also saw Takeda become only the third winners of a Prix Galien Award for orphan drug development. Mepact – for the treatment of osteosarcoma, a rare malignant bone tumour – won the Orphan Drug Award.

UK Prix Galien 2012
The UK Prix Galien, organised and managed by the specialist market access consultancy WG Consulting – which owns the UK franchise – is held every two years. The 2012 awards were hosted by former shadow Minister for Health Kevin Barron MP, who was the event’s Parliamentary Sponsor. Barron, who is currently co-Chair of the Associate Parliamentary Health Group, said: “It’s a privilege to be able to witness, at first hand, just a glimpse of the deep volumes of medical innovations being developed here in the UK. As an MP, I’ve had a long-standing professional acquaintance with UK pharma. I know and recognise the many
benefits UK medicines have brought – and continue to bring – to patients all over the world. The sector’s continued commitment to the development of medicines to tackle disease, improve health outcomes and extend life is both remarkable and humbling.”

Barron said there was political consensus that driving improvements in health outcomes across all major diseases is a key priority for the NHS – and this focus had been reflected in the 2012 finalists. “It’s interesting to note that the shortlisted entrants for the 2012 UK Prix Galien show that pharmaceutical innovation is aligned with many of the priority needs identified in the NHS Outcomes Framework. Finalists include innovations for the treatment of diseases in cardiovascular, hepatology, mental health, neurology, gastroenterology and oncology. In addition, Prix Galien’s recognition of the industry’s attempts to treat rare, orphan diseases, once again underlines the very human value of R&D.”

Value-based message
The architect of the NHS Outcomes Framework, former Health Secretary Andrew Lansley, also addressed the audience. Attending his fourth consecutive UK Prix Galien, Lansley said: “Every time I come to this event I hear about fascinating innovations that I know are going to be at the heart of the health service for years to come. I’ve met – and continue to meet – patients that have benefited directly from innovations that I’ve previously heard about at Prix Galien. The HPV vaccination programme we have been able to roll out is just one example of that. So it’s a privilege to be here.”

Lansley said that recognising and rewarding innovation is a key Government priority – and that the publication of Innovation Health and Wealth last December was part of a consistent value-based message
it wanted to send to the NHS. “That message is that as you, the pharmaceutical industry, bring forward new treatments that will clearly add value and improve the quality of healthcare for patients then the NHS should be at the forefront, internationally, of demonstrating that value. Our health service can be an exemplar and inspiration to people around the world because of its capacity to demonstrate the effectiveness of new treatments when they are used within the NHS.”

Lansley praised the UK pharma industry, highlighting the value its innovations bring both to the economy and to patients worldwide. “What you are doing is part of how this country will pay its way in the future,” he said. “And it has the added value of knowing that, in the process, we can give patients in this country access to the very best healthcare anywhere in the world.”

The recognition of innovation that can lead to improved health outcomes is a core aim of Prix Galien, as outlined by Professor Sir Michael Rawlins, who announced the winners. “Prix Galien is about honouring excellence in pharmaceutical research and development,” said Professor Sir Michael. “It is about recognising the contribution that new medicines can make to the lives of people with life-threatening conditions. It is about celebrating the achievements of all those individuals – working as teams – upon whom we rely for the discovery and development of new medicines. Most will be unknown to us – but we all owe them a huge debt of gratitude.”

Innovative Product Award
The prestigious Prix Galien medal for innovation was jointly awarded to Janssen and MSD for their respective hepatitis C treatments Incivo and Victrelis. In the UK, it is estimated that there are between 200,000 and 400,000 people chronically infected with hepatitis C virus. This may lead to liver cancer as well as other serious liver diseases. Infection with the hepatitis C virus poses a substantial global health burden, and is responsible for 40% of all cases of end-stage cirrhosis, 60% of hepatocellular carcinoma and 30% of liver transplants.

Professor Sir Michael Rawlins said: “Hepatitis C virus has become an enormous area of need globally, with many patients unaware that they are infected. The consequences of this virus are considerable and burdensome to both patients and the healthcare system; current treatments remain ineffective in a significant number of cases whilst being unpleasant and poorly tolerated by patients themselves.

“Hepatitis C infection is a perfect example of where the pharmaceutical industry can demonstrate and justify its place in healthcare by innovating for change and showing real gains to the world. It is for this reason that the panel felt that both Janssen and MSD should be celebrated and congratulated for their part in addressing the ongoing challenge in managing HCV and its associated complications.”

Brilique (AZ) and Resolor (Shire) both received commendations. Gilenya (Novartis), Xarelto (Bayer), Xeplion (Janssen), Xgeva (Amgen), Yervoy (Bristol-Myers Squibb), Zelboraf (Roche) and Zytiga (Janssen) were all shortlisted.

Orphan Drug Award
The Orphan Drug Award was introduced as a dedicated category at 2008 UK Prix Galien. There had previously been a special award for orphan products in 2006. The term ‘orphan condition’ is used to describe conditions that affect a very small number of patients in a given population – many of which are either untreatable or treated very inadequately. It is estimated that there are 6,000 orphan diseases – which, in total, affect about 30 million EU citizens.

“For orphan diseases that are potentially treatable with medicines, pharmaceutical manufacturers face a number of hurdles – including concerns about the size of the market and difficulties because of the small numbers of patients – in their development,” said Professor Sir Michael.

The 2012 Orphan Drug Award was won by Mepact from Takeda. Mepact (mifamurtide) is for the treatment of osteosarcoma, a rare malignant bone tumour – mainly of children and adolescents – that affects fewer than 1 per 10,000 individuals in the EU. This is equivalent to 150 children and young adults each year in the UK. Tumours most frequently occur in the long bones and are highly aggressive with a propensity to metastasise, particularly to the lung. If left untreated, the primary tumour will undergo local and systemic progression, leading to death within months.

“To investigate the role of this immune modulator in osteosarcoma required extensive and complex trial design with careful implementation of the study programme,” said Professor Sir Michael. “Apart from its novel mechanism of action – and clear evidence of its clinical effectiveness – the jury were also extremely impressed that such an advance in the management of osteosarcoma represents the first significant change in outcomes in 10–20 years of managing this disease. That Takeda managed to undertake the clinical development of this product – in such a niche indication – is hugely to their credit.”

The UK economy has shown 1% growth in the last quarter after nine months of recession, but analysts are unsure whether this represents a true recovery.

The slight upturn in gross domestic product (GDP) falls far short of a return to the output levels seen before the financial crisis of 2008.

The Olympics are thought to have given the UK economy a temporary boost alongside the automatic ‘dead cat bounce’ following a steep decline.

The term ‘dead cat bounce’ comes from a saying in the financial sector: Even a dead cat will bounce if it you drop it far enough.

According to the Office for National Statistics, the UK’s GDP increased by 1% between July and September after falling by 1.1% in the previous three months.

However, the ONS noted, this included a 0.2% boost due to Olympic ticket sales, with further benefits from hotel and restaurant activity and temporary jobs.

In addition, the preceding three months had been unexpectedly difficult due to bad weather and the Jubilee public holiday.

The emphasis placed on these minor factors underline the absence of major dynamic change in the UK economy.

“While the news is positive, the estimate must be put in context,” said David Kern, Chief Economist at the British Chambers of Commerce. “Compared to a year earlier, the figures show that the economy is stagnant.”

Richard Halstead, Midlands Region Director at EEF (a manufacturers’ organisation), likewise warned: “With survey data, particularly in our major markets, pointing to difficult trading conditions in recent months, it’s unlikely this pace of expansion will be maintained into the New Year.”

In the last quarter, the production sector grew by 1.1% – an encouraging figure for the pharmaceutical industry.

The restrictions on use of existing drugs in pharmaceutical R&D could be lifted under new laws proposed by the Intellectual Property Office (IPO).

The IPO’s proposed amendments to the Patents Act would rule that the use of a branded drug in a clinical or field trial does not infringe copyright.

This would make it easier for companies to compare a new drug to a branded product that is not their own, or to test a combination of the two drugs.

Current UK law allows limited use of patented drugs in tests required for the regulatory approval of generic drugs, but not of new brands.

The aim of the proposals, now out for consultation, is to create a better environment for pharmaceutical R&D in the UK.

IPO Chief Executive Sean Dennehey said: “Previous discussions with the pharmaceutical industry revealed a widespread appetite for change in the way UK patent law treats clinical or field trials.

“This consultation now offers a formal opportunity to shape the patent infringement provisions so that they can better support growth in this key industry sector.”

The consultation will run for eight weeks, until 19 December.

US researchers have defined and tested new criteria for ‘hypersexual disorder’ as a distinct type of mental illness.

The diagnostic criteria identify a pattern of compulsive sexual thoughts and behaviours that impact harmfully on the patient’s life.

Hypersexual disorder (HSD) or sex addiction often develops in adolescence and young adulthood, making early medical intervention possible.

A team of mental health specialists and other experts led by UCLA psychologist Rory Reid have proposed the criteria with a view to inclusion in the Diagnostic and Statistical Manual of Mental Disorders.

The recognition of HSD as a distinct mental health condition would open up a new field for medical interventions.

The proposed criteria were shown in a test of 207 patients to be 88% accurate in positive identification and 93% accurate in negative identification, taking negative life consequences as evidence.

Such a low (7%) rate of misdiagnosis compares favourably with other categories of mental disorder.

The diagnostic criteria include: a persistent pattern of sexual thoughts and urges; a pattern of sexual activity in response to stress or negative mood states; failure to reduce or stop problematic sexual behaviours; and personal distress caused by the impact of these behaviours on relationships.

The study reported that 54% of hypersexual patients traced the problems back before the age of 18 and another 30% to the age period 18–25. This pattern, Reid noted, “has ramifications for early intervention and prevention strategies”.

Characteristic problem behaviours included heavy reliance on pornography, cybersex, bought sex or high-risk sex.

Reid noted that such behaviours are common – “but for these patients, it’s a constant pattern that escalates until their desire for sex is controlling every aspect of their lives and they feel powerless in their efforts to change.”

The Jimmy Savile Stoke Mandeville Hospital Trust, a registered charity, is closing due to the ongoing police enquiry into alleged sexual abuse crimes by its founder.

Claims that Savile had abused patients at the renowned specialist hospital made the charity’s continuation impossible, even under another name, the Trust stated.

The Trust, established in 1981, has raised funds in support of Stoke Mandeville Hospital’s National Spinal Injuries Centre (NSIC), which opened in 1984.

Broadcaster Jimmy Savile, who had suffered a spinal injury in a mining accident in his teens, helped to raise £40 million for the establishment of the NSIC. He was awarded a knighthood in 1990 for this achievement.

However, since Savile’s death in 2011, some 200 accusations of sexual abuse of minors have been made against him, and a major police enquiry is under way.

The value of Savile’s fund-raising work for Stoke Mandeville Hospital Trust was called into question by allegations that he had recurrently abused young, disabled patients at the hospital.

The charity, together with the Leeds-based Jimmy Savile Charitable Trust, issued a joint statement announcing their closure and saying that media attention following the scandal was “potentially damaging” to people whose care they funded.

Their funds, including £1.7m held by the Stoke Mandeville Hospital Trust, will be passed on to other charities.

The long-term implications for the NSIC are unclear, but its international clinical reputation means there will be widespread pressure to find a funding solution.

This unprecedented case has raised difficult questions about child protection in the context of charity work, as well as the dependence of essential medical services on the voluntary sector.

Joint working has been recognised as a driver of therapeutic innovation at the second annual Quality in Care (QiC) Diabetes awards.

Partnerships between Eli Lilly UK and NHS and voluntary sector organisations were commended in an event that stressed the contribution of cross-sector work to the QIPP agenda.

Held at Sanofi’s UK headquarters in Guildford, the QiC Diabetes awards highlighted innovation and good practice in UK diabetes care.

A partnership between Lilly and diabetes charity JDRF to develop an educational programme on type 1 diabetes for schools was highly commended in the Best Cross-Organisational Partnership category.

Lilly was also commended for its partnership with Royal Surrey County Hospital NHS Trust to develop the ‘Piecing Together Diabetes’ clinician training tool, a jigsaw that helps staff to improve inpatient diabetes care.

“The awards have provided a unique platform to showcase good practice and innovation from across the country,” said Anna Morton, Director of NHS Diabetes. “We are all working under increased pressure and uncertainty and the examples highlighted have shown how despite adversity, high-quality diabetes care can still be achieved.”

Caroline Horwood, diabetes division director at Sanofi, commented: “We witnessed some inspirational work last year, and it is heartening to see that 2012 brings some new award categories and a 50 per cent increase in the number of entries.”

Quality in Care is a series of programmes aimed at recognising good ideas for improving care quality and productivity, in line with the QIPP agenda, and sharing them across the NHS.

Mike Farrar, Chief Executive of the NHS Confederation, has said the growing crisis in hospital funding demands major investment in community-based healthcare.

His statement follows an Audit Commission report showing that the number of hospital trusts in deficit increased from 13 in 2010/11 to 31 in 2011/12.

While the report called for tight control of trust finances, Farrar argued that the underlying problem is the over-dependence of the NHS on acute care.

The Audit Commission’s annual report on NHS finances said that while PCT finance was mostly healthy, there had been a dramatic increase in the number of foundation trusts and NHS trusts in debt.

There was “no room for complacency” over hospital trust finances and the need for tight spending controls, the Commission said.

Andy McKeon, its Managing Director of Health, commented: “The Department of Health and other relevant national authorities need to focus their attention on the minority of organisations whose financial position is deteriorating and on their geographical distribution and service standards.”

Farrar, who has long argued for a shift in funding from acute to preventative and long-term care, said: “This is the time for the NHS Commissioning Board to help providers, not with bailouts, but by releasing money to new CCGs so they can work with providers to help put them on a sustainable footing by changing the type and range of services they provide.

“Now is the time for big investment in community and primary care. It is worrying that the number of trusts in deficit has more than doubled in the past year. This situation is likely get worse unless we take radical action.”