The European Medicines Agency has pledged to improve access to medicines in its Road Map to 2015, a publication that coincides with the Agency’s 15th anniversary.

Stakeholders, including the pharmaceutical industry, and the public have been invited to comment on the Agency’s strategic vision, The European Medicines Agency Road Map to 2015: The Agency’s contribution to Science, Medicines, Health.

Building on the achievements made by the previous Road Map initiative between 2005 and 2010, the focus of the new Road Map to 2015 is on continuous high-quality delivery of the Agency’s core business in an increasingly complex regulatory and scientific environment.

In addition, the document proposes three priority areas for future actions to strengthen the Agency’s role in protecting and promoting human and animal health in the EU:

• Addressing public health needs by: stimulating research and medicines development in areas of unmet medical need or for neglected and rare diseases; facilitating new and innovative approaches to the development of medicines; implementing effective preparedness plans to deal with public health threats.
• Facilitating access to medicines by: addressing the high attrition rate during the development process of medicines; improving the Agency’s model for the assessment of benefits and risks of medicines; improving the quality and scientific and regulatory consistency of the medicines review process.
• Optimising the safe use of medicines by: strengthening the evidence base on the benefits and risks of a medicine following its authorisation; applying novel pharmacovigilance methodologies and risk minimisation tools; taking patient experience into account for improved decision-making; becoming a reference point on information about medicines evaluated by the Agency.

The Agency will hold a number of workshops and face-to-face discussions as part of the public consultation process.

Approvals for products to treat neglected diseases have steadily increased in recent years, a recent US study has revealed.

The research shows that the number of products to treat neglected diseases receiving marketing approval from regulatory agencies has grown as R&D funding for those diseases has increased.

According to the study, completed by the Tufts Center for the Study of Drug Development, the annual rate of new product approvals worldwide for neglected diseases increased from an average of 1.8 in 1975–99 to 2.6 in 2000–09.

Neglected diseases include malaria, kinetoplastids, diarrhoeal diseases, helminths (e.g. roundworm), bacterial pneumonia and meningitis, and typhoid and paratyphoid fever.

“During this past decade, a significant increase in R&D funding for neglected diseases has led to marketing approval for 26 drugs and vaccines,” said Joshua Cohen, Senior Research Fellow at Tufts CSDD and author of the study.

“While increased approvals are necessary to improve access, policy-makers need to ensure that safe, effective and easy-to-administer products are adopted by healthcare systems and providers on a consistent basis, that they are affordable, and that they reach the people who need them.”

The Tufts CSDD analysis discovered that an original study, which reported that only 16 of 1393 new drugs marketed between 1975 and 1999 targeted tropical diseases and tuberculosis, was inaccurate and a more accurate count was 33. However, this earlier study prompted increased funding for neglected diseases, from less than $100 million annually a decade ago to more than $2.5 billion annually today.

The new analysis, reported in the November/December Tufts CSDD Impact Report, also found that:

• Drugs to treat HIV/AIDS and malaria accounted for 81% of approvals during 2000–09.
• Vaccines have displaced drugs as the main products being developed for neglected diseases, accounting for 76% of all products.
• Public-private partnerships accounted for 46% of all new product development to treat neglected diseases during 2000–09, up from 15% in the 1975–99 period.

The Tufts Center for the Study of Drug Development, based at Tufts University in Boston, provides strategic information to help drug developers, regulators and policy-makers improve the quality and efficiency of pharmaceutical development, review and utilisation.

A new report from the Office for Life Sciences (OLS) draws up a road map for the advancement of the life science industries in the UK through collaboration between the public, private and academic sectors.

Life Sciences 2010: Delivering the Blueprint has been welcomed by industry trade associations as taking forward and building on the measures proposed in the OLS Blueprint six months ago.

The OLS initiative has focused on improving the commercial environment of the life science industries, with participation from trade associations representing medtech, biotech and pharma.

The report brings together existing and impending measures aimed at improving the route from UK innovation to NHS practice. These include:

• Industry-Higher Education Forum – bringing together relevant stakeholders to identify critical disciplines and skills gaps within life science research.

• Capability Clusters – strengthening collaborative work between the NHS, academia and industry to develop new therapies (focusing initially on respiratory and inflammatory joint disease).

• NHS Operating Framework – embedding clinical trials within core NHS activity.

• RegenMed – a £21.5m programme managed by the Technology Strategy Board to support key areas of commercial R&D and develop R&D partnerships.

• Patent Box – reducing corporation tax on innovative UK industry activity.

• Innovation Pass – assisting rapid NHS uptake of treatments that show potential to deliver patient benefits but struggle to show cost-effectiveness at launch.

• NHS Life Sciences Delivery Board – improving the uptake and adoption of innovative technologies and medicines.

Richard Barker, Director General of the Association of British Pharmaceutical Industry (ABPI) said: “Life Sciences 2010 is a major milestone in the OLS initiative and demonstrates the potential of this new model of industry and government collaboration to deliver truly tangible results. Work to implement and extend this strategy must be a high priority for both government and industry in the coming years, to maintain the UK’s leadership role and the flow of life-changing, innovative therapies for patients.”

Doris-Ann Williams, Director General of BIVDA, commented: “The work of the OLS has provided an excellent example of what can happen when Government and business work together.”

Two of Sanofi Pasteur’s vaccines have made it onto Time magazine’s list of the 10 most important medical breakthroughs of 2009.

The firm’s HIV vaccine came in second place. The magazine highlighted that trials of its effectiveness yielded “reasonable” results, with an infection prevention rate of 31%.

However, it added that the implications of this are important: “Given that no other inoculation has shown any effect against the AIDS virus, it was reason to celebrate – cautiously.”

In fourth place was Sanofi Pasteur’s H1N1 vaccine, for which demand outstripped supply when it was launched last year. The publication highlighted that, in some areas, “there was not enough vaccine even to cover members of priority groups”.

Another pharmaceutical product to make it onto the list was Amgen’s denosumab at number 8, an innovative treatment for Alzheimer’s disease currently under review by the FDA.

Other breakthroughs listed included the lifting of the funding ban on stem-cell research, prostate cancer screening, new autism research that highlighted variations in DNA and studies into brown fat in adults.

The number of women under 30 diagnosed with cervical cancer will fall nearly two thirds by 2025 due to the HPV vaccine, new research has revealed.

The study, published in the British Journal of Cancer today, predicted that the number of women in their twenties diagnosed with cervical cancer will drop by 63% in the next 15 years.

Girls aged 12 and 13 have been offered the HPV vaccine in the UK since 2008. It protects against two types of the virus – HPV 16 and HPV 18 – which cause around 70% of cervical cancers.

The researchers calculated the number of cancers that would be prevented by the vaccine, assuming 80% of girls took it up (latest Government figures suggest that 78% of girls had received all three doses of the vaccine).

The researchers also predicted that 51% fewer women will have CIN3 – severe cell changes that need treatment because they could lead to cervical cancer – and that there will be 27% fewer abnormal smear tests.

Cancer Research UK’s Professor Jack Cuzick, lead author from Queen Mary, University of London, said: “In women in their twenties alone, around 145 cases of cervical cancer will be prevented each year in the UK thanks to the HPV vaccine. And around 13,000 women each year will be spared from having an abnormal screening test result.

“This is the most realistic estimate of the impact the vaccination programme will have on the number of women who develop cervical cancer. It shows that the vaccine has great potential in preventing the disease in the near future, but also that it’ll take several decades before we see its full benefits.”

There are around 2,800 new cases of cervical cancer diagnosed in the UK each year, and around 225 of these are in women in their twenties. Around 940 women die from cervical cancer each year in the UK.

Dr Lesley Walker, Director of Cancer Information at Cancer Research UK, said: “This is really good news for girls who have had the vaccine and for those who will have it in the future. But it’s important to remember that the vaccine will not completely wipe out cervical cancer because it doesn’t protect against every type of high-risk HPV.

“Now and for the foreseeable future, it’s vital that women go for cervical screening when they’re invited. Screening can prevent cervical cancer by detecting unusual changes in the cervix before cancer develops, and it saves thousands of lives in the UK each year.”

A new NICE guideline on treating blood clots in hospital patients could mark a significant advance in the prevention of an estimated 25,000 avoidable deaths.

The guideline sets out simple steps to help prevent venous thromboembolism (VTE), which covers both deep vein thrombosis (DVT) and its possible consequence pulmonary embolism (PE).

An estimated 25,000 people who are admitted to hospital die from preventable venous thromboembolism each year. PE is the immediate cause of 10% of all hospital deaths, a rate greater than the combined total from breast cancer, AIDS and traffic accidents.

The NICE guideline, jointly developed with the National Clinical Guideline Centre for Acute and Chronic Conditions, recommends that all patients should be assessed for risk of developing blood clots on admission to hospital and then given preventative treatment that suits their individual needs.

These options include blood-thinning drugs such as heparin, anti-embolism stockings and foot impulse or pneumatic devices. The advice covers all patients admitted to hospital – including those having day-case procedures – and not just those patients having surgery.

Professor Tom Treasure, Chair of the NICE Guideline Development Group and Professor of Cardio-Thoracic Surgery, said: “Put simply, all patients without exception should be assessed on being admitted to hospital for risk of developing a blood clot, and then given preventative treatment that is appropriate for them. VTE is a silent killer – so it’s the responsibility of medical professionals to take the very simple steps set out in the guideline, which can help prevent unnecessary deaths and long-term illnesses.”

Publication of new NICE guidelines follow a recent DH initiative that will further encourage hospitals to implement risk assessment and prevention of hospital-acquired VTE by offering financial incentives for compliance. This initiative will operate through the CQUIN (Commissioning for Quality and Innovation) payment framework.

Professor Beverley Hunt, Medical Director of Lifeblood: the thrombosis charity and a member of the NICE Guideline Development Group, said: “Having the Trusts penalised if they don’t adequately risk-assess is great news for patients. The NICE Guidelines clearly outline what protection patients at risk from VTE should expect. With the new CQUIN targets in place, hospital managers have now been forced to make this a priority, with those Trusts that fail to meet the new targets facing significant fines.”

In response to the guidelines, Bayer Schering Pharma has launched a Thromboprophylaxis Assessment and Implementation Toolkit Route Map for anyone who is involved in the provision, management and commissioning of VTE thromboprophylaxis services.

The European Medicines Agency has recommended additional measures to better manage the risk of progressive multifocal leukoencephalopathy (PML) with Elan Pharma’s Tysabri (natalizumab).

The CHMP has concluded that the risk of developing PML increases after two years of Tysabri use, though this risk remains low.

However, the benefits of the medicine continue to outweigh its risks for patients with highly active relapsing-remitting multiple sclerosis, for whom there are few treatment options available.

Because it is important that PML is detected early, the Committee recommended that a number of measures be put in place to ensure that patients and doctors are fully aware of the risk of PML.

These include an updating of the product information to include the increased risk of PML after two years of treatment and forms to be signed by patients at the beginning of treatment, and again after two years, after in-depth discussion about the risk of PML with their doctor.

Measures to minimise the risk of PML were part of the initial marketing authorisation for Tysabri, issued in June 2006. Since then, they have been continuously updated and strengthened to increase awareness of the risk of PML.

The CHMP has recommended the granting of a conditional marketing authorisation for a fourth pandemic vaccine, Arepanrix from GlaxoSmithKline Biologicals.

The recommendation was made using an emergency procedure that fast-tracks evaluation of new vaccines developed during a pandemic.

Further clinical studies in children, adolescents and adults are ongoing and results will become available from March 2010 onwards.

The Committee also reviewed further data on the three centrally-authorised pandemic influenza vaccines Celvapan, Focetria and Pandemrix and the antiviral Tamiflu.

Following a review of Celvapan studies in both children and adults, the Committee agreed that the studies did not support a change to a single-dose vaccination schedule. The Committee will continue to discuss this issue with experts and has requested further data from the manufacturer.

The Committee supported an update to the product information of Pandemrix to include additional data on the immunogenicity and safety in three- to nine-year-old children. Data is still being assessed on Focetria.

The Committee also recommended that safety data in immunocompromised subjects be added to the product information for Tamiflu.

The CHMP has issued a positive opinion for GlaxoSmithKline and Genmab’s Arzerra (ofatumumab) for the treatment of refractory chronic lymphocytic leukaemia (CLL).

The CHMP has recommended that the drug is granted a conditional marketing authorisation in the EU for the treatment of patients who are refractory to fludarabine and alemtuzumab, the standard therapies currently used to treat CLL.

CLL is a cancer of the blood and bone marrow. The disease is termed refractory when patients do not respond to treatment, and at this stage there are limited treatment options and poor outcomes with existing treatments.

A conditional marketing authorisation is granted to a drug with a positive benefit/risk assessment that fulfils an unmet medical need. These are granted when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required.

As part of the conditions of the conditional marketing authorisation for ofatumumab, GSK will be required to provide further data.

Cimzia has become the first RA treatment to receive positive guidance from NICE in more than two years due to an innovative patient access scheme.

Certolizumab pegol, or Cimzia, has been recommended for approval for the treatment of adults with severe active RA, on the condition that the patient access scheme is implemented and the drug is prescribed in accordance with NICE’s specifications.

The drug’s registered UK indication is for use in combination with methotrexate (MTX) for the treatment of moderate to severe active rheumatoid arthritis (RA) in adult patients when the response to disease-modifying antirheumatic drugs (DMARDs), including methotrexate, has been inadequate.

In a novel move for drugs in RA, manufacturer UCB has worked with the DH to provide a clear, cost-effective alternative for people with RA through a patient access scheme.

In clinical studies, the new drug has demonstrated significant results as early as the first week of treatment, and the majority of patients have responded within the first 12 weeks. UCB and the DH have agreed a patient access scheme to ensure that treatment decisions are based on patient need rather than cost. UCB will make the new treatment available to all eligible RA patients free of charge for the first 12 weeks.

Ailsa Bosworth, Chief Executive of the National Rheumatoid Arthritis Society, comments: “This is the first time a new NICE-recommended RA treatment has been made available in over two years – and it could make a big difference to patients’ day-to-day lives.”

“Certolizumab pegol is an important new treatment option for people with rheumatoid arthritis, and it’s exciting that it is now available on the NHS,” commented Professor Peter Taylor, investigator and Professor in Experimental Rheumatology, Imperial College London NHS Trust.

“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.”