NICE is to convene to decide the fate of Bayer Schering Pharma’s Nexavar (sorafenib) in the treatment of advanced hepatocellular carcinoma (HCC).

The Institute will consider Bayer’s appeal (submitted in December 2009) of its draft negative Final Appraisal Determination (FAD) for Nexavar in HCC, which was published after nearly two years of consultation.

Bayer appealed on the grounds that NICE failed to act fairly and in accordance with its own published procedures, as well as failing to consider the long-term benefits of innovation.

Following the outcome of the hearing, NICE will issue its final ruling in March 2010.

Nicole Farmer, Business Unit Head of Bayer Schering Pharma Oncology in the UK, said: “It is so frustrating that for the 600 liver cancer patients who could really benefit from Nexavar, there still remains the chance that they will be denied. We really hope that NICE takes this opportunity to reconsider some of the data and ensures that real innovations in healthcare, like Nexavar, are made available to all who need it, not just those who could afford to pay privately.”

Since NICE’s negative ruling in November, patient groups and clinicians have been involved in a high-profile media appeal, including a systematic letter writing campaign by oncologists to NICE and the media.

Nexavar is the first systemic drug for advanced HCC to show a significant survival benefit after 30 years of comparative trials, and has demonstrated a 44% increase in survival for advanced HCC patients compared to best supportive care alone.

Professor Karol Sikora, Professor of Cancer Medicine and Medical Director of CancerPartnersUK, said: “Since NICE’s decision not to fund the drug in November, many clinicians (a number of whom would have been involved in the UK trials of the drug) have been put in the painful position of having to deny their patients the only survival option for them.

“As we reach the end of this laborious process, which has left both patients and clinicians in limbo for far too long, we can only hope that NICE will use this final window of time to properly consider the very strong recommendations from UK oncologists, who only want the best for their patients.”

Cases of liver cancer have almost tripled over the past three decades, according to figures recently published by Cancer Research UK. In 1975 there were 865 cases of primary liver cancer in the UK, and in 2006 that had risen to around 3,200 new cases. HCC accounts for 80–90% of these primary liver cancers.

Top pharmaceutical and biotech companies will face a major decline in sales growth up to 2014, according to a new report.

However, those companies that have been able to diversify into biologics, niche products and generics will still enjoy positive growth, says business analyst Datamonitor.

The report, Pharmaceutical Company Outlook to 2014, considers the forecast performance of the world’s biggest pharma and biotech companies, termed the “PharmaVitae Universe”.

The analysis reveals prescription sales are expected to rise at a compound annual growth rate (CAGR) of 1.2% up to 2014, compared to a historical growth rate of 10.5% over 2002–08. The report blames patent expiry and generic competition for this sharp decline.

“The major obstacle to the PharmaVitae Universe’s continued expansion is undoubtedly the growth of the generics market, eroding sales of major brands and market value. While this will directly impact products facing patent expiry, there will also be an indirect impact to patented brands as they are forced to compete with alternative generics,” said Rebecca Whitham, Datamonitor Analyst.

However, the report forecasts that several companies will outperform the PharmaVitae Universe average growth rate, particularly those that have diversified into the biologics sectors of monoclonal antibodies and therapeutic proteins. Datamonitor expects Roche, for example, to benefit from its early move into the monoclonal antibody market due to the sector’s high growth.

Mid-sized companies Gilead, Actelion and Celgene are also expected to achieve double-digit CAGRs, due to their strategy of targeting niche indications and areas of high unmet need. In addition, the report points out, companies such as Novartis that have developed their own generic presence will benefit from the growth in this market.

Other companies will weather the storm through mergers and acquisitions. “Where M&A has acted as the PharmaVitae Universe’s biggest growth driver over 2002–08, this unknown quantity will likely continue to play a key role in driving company performance going forward,” concluded Whitham.

“Despite bearing the impact of generic competition to some of the world’s biggest pharmaceutical brands, the large-scale M&A witnessed in 2009 will see Pfizer-Wyeth and Merck-Schering-Plough become the first and second largest companies within the PharmaVitae Universe by 2014.”

The report Pharmaceutical Company Outlook to 2014: Analysis of the Top Pharma and Biotech Companies examines the forecast performance of the world’s 43 biggest pharmaceutical and biotechnology companies according to 2008 prescription pharmaceutical revenues.

Schering-Plough has achieved success at this year’s UK Marketing Excellence Awards, receiving the award for best medical supplier.

The company fought off competition from 3M Drug Delivery Systems, Mediplus and Nutricia Advanced Medical Nutrition to win the award, which was partly judged by Natalie Uhlarz, Editor of Pharmaceutical Marketing.

Schering-Plough Senior Brand Manager Russell Abberley was also a runner-up in the Marketer of the Year category, an award for professionals “who have demonstrated exceptional marketing skills, knowledge and initiative.”

The Chartered Institute of Marketing’s annual awards evening was held at the Grange St Paul’s Hotel, London and hosted by Hugh Dennis of Mock the Week fame.

The overall Chairman’s Award was given to Ernst & Young for their outstanding contribution to the profession with their winning entry in the Professional Services category. The award was accepted by Ernst & Young’s marketing director EMEIA, Andrew Shaylor.

David Thorp, Director of Research and Professional Development at The Chartered Institute of Marketing, commented: “The entrants demonstrated the creativity needed to demonstrate good marketing practice and innovation, even during the current financial downturn. The diversity and creativity of the entries resulted in much deliberation by the judging teams to judge and mark the final and worthy shortlist.”

The awards endeavour to acknowledge and reward organisations and individuals that have made an outstanding contribution to the profession. They invite applications from all marketers regardless of sector or organisation size, aiming to highlight the fundamental position that marketing occupies in business and the public sector and to celebrate marketing achievements across a range of industries.

The James Whale Fund for Kidney Cancer has launched an online petition at No. 10 Downing Street calling for a review of the current NICE system for appraising cancer drugs.

The petition has been launched in response to the recent revelation that the UK’s death rate is still around 6% higher than the European average, though spending on cancer medicines is only 60% that of other countries.

The relatively poor take-up of new treatments in the UK was one of the reasons listed in the report as “contributing to the higher death rate”.

The charity has described the current system for appraising drugs as “extremely bureaucratic, callous, time-consuming, outdated and in desperate need of reform”.

James Whale, Founder of the James Whale Fund, commented: “The Government needs to wake up to the fact that cancer services in the UK are abysmal and the survival rates for kidney cancer patients are hugely affected by this poor level of service.

“This is just one example of the dire situation facing many cancer patients in the UK today. No matter what the cancer is, the poor uptake by NICE of cancer treatments is leaving the UK lagging far behind in survival rates.”

The online petition, spearheaded by the Cancer Patient Support Group at the James Whale Fund for kidney cancer, is attracting over 100 signatures per day due to patients promoting the petition themselves.

This follows recent news that NICE has issued preliminary guidance to the NHS rejecting everolimus (trade name Afinitor, manufactured by Novartis), a new and innovative drug for advanced kidney cancer.

To view the petition or sign up, go to: http://petitions.number10.gov.uk/NewCancerDrugs/.

The methods for abandoning old drugs in favour of new ones must be improved, experts on bmj.com have warned.

Decisions about which drugs to abandon to fund new treatments recommended by the NICE are inconsistent and may be contributing to the postcode lottery – one of the key issues that NICE was set up to tackle, Dyfrig Hughes and Robin Ferner argue.

The article goes on to argue that the methods for identifying drugs that can be discontinued need to be “as rigorous as those for assessing potential new treatments to ensure best use of NHS resources”.

The NHS is legally obliged to fund treatments recommended by NICE but does not receive extra money to do so. This means that the funding of new, expensive medicines relies increasingly on displacing other treatments, but NICE does not specify which. The authors ask how we should establish which medicines to discontinue.

Obvious targets include treatments that have been replaced by more effective medicines, but the authors claim that it could be better to maintain an older treatment that is marginally less effective but much cheaper.

In conclusion, the article calls for an explicit framework for the identification and appraisal of medicines for disinvestment to provide better value for money while reducing inequity.

Basilea and Astellas have signed a co-promotion agreement for Basilea’s azole antifungal agent isavuconazole, currently in phase III development for the treatment of invasive fungal infections.

Under the terms of the agreement, Basilea will receive an upfront payment of CHF 75 million and will be eligible to receive up to CHF 478 million in additional payments on achievement of pre-specified development and sales milestones.

In return, Astellas has gained the right to commercialise isavuconazole, though Basilea will retain an option to co-promote the product in the US, Canada, major European countries and China.

Dr Anthony Man, CEO, Basilea Pharmaceutica Ltd, said: “Astellas has a successful track record in developing and commercialising antifungals in the hospital sector internationally. We look forward to working together with Astellas to achieve isavuconazole’s full potential as a novel therapeutic option for treating serious and life-threatening fungal infections.”

Astellas will lead the clinical development program investigating isavuconazole for the treatment of invasive fungal infections caused by Aspergillus or other filamentous fungi (mould infections) and Candida fungi (yeast infections).

Masafumi Nogimori, President and CEO, Astellas, commented: “Astellas already has experience in the development and marketing of the echinocandin antifungal agent Mycamine (micafungin) worldwide and is committed to focusing on infectious diseases. This partnership is an important step to further expand our business and to reinforce our franchise in infectious diseases.”

The MS Society is campaigning for an end to a scheme that gives multiple sclerosis patients access to drugs that are not recommended by NICE.

The Risk Sharing Scheme, which was to run for ten years, was implemented by the DH in 2002 when NICE decided that certain disease-modifying drugs were not cost-effective. The drugs involved are Biogen Idec’s Avonex, Bayer Schering Pharma’s Betaferon, Teva/sanofi-aventis’ Copaxone and Merck Serono’s Rebif.

However, the MS Society argues that the scheme has been poorly managed and is even affecting patient access to the NICE-approved drug Tysabri.

A review of the scheme was published in the British Medical Journal in December 2009, which highlighted various methodological difficulties.

The MS Society also points out that the scheme is causing confusion over entitlement to Tysabri, that access to the drugs is governed by a post-code lottery and that NICE’s MS guidance is out of date due to awaiting the outcome of the programme.

Simon Gillespie, Chief Executive of the MS Society, said: “This is a deeply frustrating situation. The four drugs involved are not the issue and many have benefitted from taking them. It is the way the scheme has been run that is the problem.

“People with MS and taxpayers deserve much better. Government’s approach towards the treatment of 100,000 people with MS across the UK doesn’t give them, or their families, confidence for the future. We already have evidence that the ineffective operation of the scheme has exacerbated the post code lottery in treatments, for example in prescribing the newer drug Tysabri for people with severe MS.”

James Gray MP, Chair of the All Party Parliamentary Group on MS, represented these feelings in a debate with Health Minister Mike O’Brien MP, concluding: “the scheme is knackered, it cannot be mended and anyone with half a brain would call for its abolition!”

Mike O’Brien responded that it was too early to reach firm conclusions about the cost-effectiveness of the drugs. He added that he would “investigate any concerns that primary care trusts are refusing to fund the use of Tysabri as recommended by NICE” and that “if there is any evidence of postcode prescribing, we will be anxious to see it.”

Simon Gillespie concluded: “Unless the shortcomings of the scheme are addressed rapidly, the introduction of other new therapies for MS is also likely to be delayed. Most people live with their MS for many decades, and it is imperative that people with MS receive the correct treatment at the right time.

“Anything short of this would be a grave injustice to all those with MS, as well as ultimately costing society more for hospital stays and care that could be avoided.”

NICE has finalised its revised Cimzia (certolizumab pegol) guidance, recommending the treatment for some people in England and Wales suffering with rheumatoid arthritis.

The recommendation is another example of a negative NICE decision being reversed due to a patient access scheme agreed with the manufacturer.

In this case, UCB has agreed to provide the first 12 weeks of certolizumab pegol free for all patients starting treatment. The company also provided additional cost effectiveness data that influenced NICE’s decision.

Dr Carole Longson, Director, Health Technology Evaluation Centre, said: “The recommendations have been facilitated by the positive response of the manufacturer (UCB Pharma) in providing more detailed information on the clinical and cost effectiveness of its treatment, and by offering the patient access scheme. Rheumatoid arthritis can be very painful and consequently, very debilitating, so I am sure this decision will be welcomed by all those affected by the disease.”

Rheumatoid arthritis (RA) is a chronic disease in which joints in the body become inflamed, causing pain, swelling and stiffness. It often affects the small joints of the hands and the feet, and usually both sides equally and symmetrically.

Professor Peter Taylor, investigator and Professor in Experimental Rheumatology, Imperial College London NHS Trust, commented: “Certolizumab pegol has been shown to rapidly improve patients’ symptoms and to significantly reduce the rate of progression of joint damage associated with rheumatoid arthritis. This fast and lasting effect is important as it quickly improves function, reduces work disability and leads to a better quality of life for patients.”

Certolizumab pegol is a TNF (tumour necrosis factor) inhibitor, which is indicated in combination with methotrexate for the treatment of moderate to severe, active rheumatoid arthritis in adult patients, when the response to disease-modifying antirheumatic drugs (DMARDs) including methotrexate, has been inadequate.

NICE has overturned its previous guidance on Yondelis (trebactedin) to recommend access to the drug for some cancer patients.

The new final guidance recommends trabectedin for the treatment of certain patients with advanced soft tissue sarcoma, reversing the Institute’s earlier negative assessment.

The recommendation was made following the manufacturer PharmaMar’s agreement to meet the costs of the drug when it is needed beyond the fifth cycle of treatment.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE, said: “It’s thought between 500 and 600 people live with advanced soft tissue sarcoma in England and Wales. We are pleased to be able to recommend trabectedin for NHS use as it will provide a treatment option for some patients.”

NICE has recommended the use of trabectedin as a treatment for people with advanced soft tissue sarcoma if treatment with anthracyclines and ifosfamide has failed or they are intolerant to these therapies.

Roger Wilson, from Sarcoma UK, commented: “It has been a challenging journey for everyone concerned, doctors, patients, regulators and the manufacturer. This news encourages everyone as it opens the way for a new era in the treatment of advanced soft tissue sarcoma, for which there have been few advances in over 20 years.

“Few pharmaceutical companies pay much regard to rare cancers so I would pay tribute to PharmaMar for their determination to see Yondelis into clinical use, and to thank them for the patient access scheme which makes it possible.”

Soft tissue sarcomas are tumours that develop in the soft, supporting tissue in the body, such as fat, muscle and blood vessels and can occur anywhere in the body.

Trabectedin (Yondelis) is an intravenous drug that works by damaging the DNA in cancer cells, making them unable to grow and spread. It is administered over 24-hours with a three week interval between cycles.

Research has shown that the drug can extend the life of a person with advanced soft tissue sarcoma by at least three more months than current NHS treatment.

The UK’s cancer rate is 6% higher than the European average, yet spending on cancer medicines is only 60% that of other advanced western countries.

England currently spends £18.33 billion on cancer care annually, but this could increase to £24.72 billion over the next few years, found a new report by think tank Policy Exchange, The Cost of Cancer.

The figures take into account healthcare costs, costs to patients and families and losses in productivity, making it the most comprehensive study of the total costs of cancer to have taken place in England.

The report blames late diagnosis, poor survival rates for older people and those in deprived communities, and relatively poor take-up of new treatments and technologies for the UK’s high mortality rate.

Overall, the study found, England spends 5.6% of its public healthcare budget on cancer, compared to 7.7% in France, 9.2% in the US and 9.6% in Germany.

If UK survival rates were improved in line with the best in Europe, by 2020 the cost of cancer care could be reduced by £10 billion, and 71, 500 lives could be saved.

The report recommends that the DH should adopt best practice in cancer services from other European countries, refocus resources where the largest reductions in mortality can be achieved and focus on prevention strategies, including smoking reduction initiatives.

Henry Featherstone, Head of Policy Exchange’s Health Unit and author of the report, said: “Cancer kills over one in four people in England, and is seen by the public as being the top disease priority for the NHS. The current cost of cancer – to say nothing of the human tragedy involved – is currently at a staggering £18.33 billion, and will only rise further.

“With cross-programme action on earlier diagnosis and better targeting of resources towards older people and communities who are most at risk we could make significant reductions in mortality rates, saving not only billions of pounds, but also the upset and suffering caused to thousands of individuals and families.”