Patients have been found to be sharing information on side-effects, dosage etc on sites like PatientsLikeMe. How useful is this data? Can pharma learn from it? Should pharma be monitoring it and responding to it? Is it their responsibility to reach out to these people? What they are doing is potentially dangerous but when people have nothing to loose you can see why they would be willing to give it a shot.

How close are we to accurately predicting through a simple blood test for the risk of #breastcancer?

Last week there was quite a media flurry about the possibility of a blood test for breast cancer after a press release from the Breast Cancer Campaign. It all steamed from a paper published by Cancer Research in February which highlighted that epigenetic changes could be associated with this form of cancer. The study found that in blood samples from 1,380 women at a high risk of breast cancer there was an association of DNA methylation in white blood cells and the chance of cancer development. The samples were taken at an average of 3 years before diagnosis.

It is still far too early to say we have one simple blood test to predict the risk of breast cancer, indeed this research only looked at a single gene and breast cancer can be triggered by many genetic changes. Hopefully this will lead to larger, genome-wide testing for breast cancer and the same methodology being applied to other cancers such as leukemias and lymphomas. Unfortunately these future applications are some time away, no matter what the media would like to claim.

You would be joining some of the world’s best pharmaceutical companies. Those attending so far include:

Abbott, Actelion Pharmaceuticals, Aderans Research Institute, Aptiv Solutions, AstraZeneca, AstraZeneca R&D, Bayer Healthcare, Bayer Pharma AG , Bayer Technology Services GmbH, Biocartis SA, BioMartin Pharmaceutical, Bristol Myers Squibb, Cancer Research UK, Celgene, Chiesi Farmaceutici, Clinical Reference Laboratory, Daiichi Sankyo Pharma, Debiopharm SA, DUCK FLATS Pharma, Eisai, Eli Lilly, European Commission, Everest Biotech, F. Hoffmann- La Roche, Federal Agency for Medicines and Health Products, Galapagos Research Center, Gilead Sciences, GlaxoSmithKline, GlaxoSmithKline R&D, Hoffmann-La Roche, Industrial Technology Research Institute, J&J Pharmaceutical R&D, Janssen Pharmaceuticals, Java Clinical Research, Johnson & Johnson, Leo Pharma, London Genetics, MedImmune, Merck, Merck Research Laboratories, Merck Serono SA, Merck Sharp & Dohme Europe, Novartis, Novartis Institutes for BioMedical Research, Novartis Pharmaceutical R&D, Nuvisan GmbH, Nycomed, Orion Corporation, Oxford Biodynamics, Pfizer, Quality Assistance, Roche China, Roche/Genentech, Sanofi R&D, Sanofi-Aventis R&D, Selcia, Selventa, Shire Pharmaceuticals, Sunovian Pharmaceuticals, Takeda Pharmaceuticals, Technology Strategy Board, Telormedix SA, UCB Pharma S.A, UCD Conway Institute of Biomolecular & Biomedical Research and more.

Make sure your register now  to ensure you hear the latest clinical development strategies in phase I from the industry’s best experts.

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Check out the agenda

Last month an international research team led by University of California Davis made a vital step in an effective vaccine against Salmonella

The discovery of the 8 common antigens will allow the development of a vaccine in the lab which can be applied to clinical trials at a later stage. This comes at a vital time. Salmonella bacteria is becoming increasingly resistant to antibiotic treatments and with no new, effective antibiotics coming, the 1.4million cases that occur annually in the US alone could get alot more sever. Already 580 lives are lost to this bacteria.

We wanted to know what the current pre clinical drug development challenges were so we asked Phil Birch, Corporate Brand Manager at Aptiv Solutions what his thoughts were. Here is what he had to say.

The Key Challenges in Clinical Development are:

• - R&D productivity continues to decline
• - Development costs continue to rise
• - Late-stage Phase III terminations remain a major issue
• - Health-economic pressures mount
• - The justification of true cost/benefit is becoming more challenging
• - Revenue forecasts remain flat or shoe minimal growth

Strategies to Expedite Drug Development

Adaptive Clinical Trials

The design and implementation of Adaptive Clinical Trials (ACTs) offers significant potential to improve the efficiency of product development programmes. ACTs rely on the timely collection of data in defined interim analysis steps providing the opportunity for the trial to adapt to emerging data (i.e. real time learning). For example, following an interim analysis, a decision may be made to re-estimate the size of the trial, stop the overall trial for futility, drop ineffective treatments, or change the randomization allocation in favour of more effective treatments in defined populations of patients. Importantly, these are not ad-hoc changes but are design changes that are pre-specified and planned in advance. Adaptive design concepts are applicable from phase I through phase IV, and importantly regulatory agencies in US and Europe have published specific guidance documents on adaptive trials which support their use in exploratory Phase I to Phase IIb (“Learn”) trials and in adequate and well controlled Phase III (“Confirm”) trials.

Although adaptive design concepts have been available for some time, only recently has implementation (especially for complex design trials) been achievable. This breakthrough has been possible because of the development of integrated technologies specifically designed to support the execution of ACTs. The utilization of adaptive execution technologies alongside specific adaptive processes governing biostatistics, data management, project management and monitoring has opened up the potential for wider use of adaptive designs in clinical development. Importantly, these processes have to meet the requirements specified in agency guidance and hence an intimate knowledge of these guidelines and their interpretation is essential to regulatory success.

The essential requirements for successful design, implementation and execution of ACTs are multi-fold and include:

• Expertise to develop innovative adaptive methodologies including new validated design software (e.g. ADDPLAN®, FACTS™)

• Expertise and experience in developing robust solutions to address implementation challenges

• Integrated technologies designed specifically to manage the interim analysis steps central to the adaptive trial process (e.g. AptivAdvantage™)

• SOPs, processes & work practices designed specifically to support adaptive trial execution

Expertise in this area has grown steadily over the last decade not only within the pharma companies but also through the emergence of certain service provides who have specific expertise in adaptive trial design and execution. These service providers are enabling sponsor companies implement complex design trials that were once thought too difficult to achieve. The integrated execution environment required to support adaptive designs will also increase the number and complexity of designs available to industry, leading to trials that more robustly identify critical components of success.

Some of the key benefits will include:

• Improved decision-making driven by significant increases in the value of information gained per $ invested
• Increased R&D productivity in which ‘winning’ treatments will be selected over treatments that are destined to fail – and a dramatic decrease in the need to repeat trials that just fall short of significance
• More effective dose selection at Phase II leading to a much higher success rate at phase III
• The application of enrichment designs to robustly identify specific patient sub-populations to treat with targeted therapies – leading to much improved health economic measures and stronger arguments for successful pricing and reimbursement

These factors will change the landscape of R&D and contribute significantly to pharma industry recovery and sustained future success.

Aptiv Solutions are sponsors of Exploratory Clinical Development World Europe 2012.

Early diagnosis of clinical depression

The researchers used the potential biomarkers from the rat models to screen the blood of 15 to 19 year olds with untreated clinical depression or without depression. They refined the biomarker panel and were able to spot the difference between the 2 groups including those with anxiety or maltreatment. This is the first biomarker test to distinguish between the subtypes.

This is great news for those suffering from this illness and will hopefully lead to an improvement in treatment soon.

If you are involved in Biomarkers then check out the programme for Biomarkers World Europe 2012.

Strategic alliances can be powerful things. But, like everything in life, it has its downsides. Here’s a look at the advantages and disadvantages of such alliances.

Advantages:

• Get instant/quicker market access into a new market.
• Increase sales.
• Widen your distribution channels.
• Strengthen your position in a market where you have minimal traction.
• Gain new skills and technology. 
• Share fixed costs and resources.
• Broaden your business contacts.
• Gain greater knowledge of international customs and culture.
• Strengthen your brand awareness in the world marketplace.

Disadvantages:

• Weaker management involvement
• Less equity stake.
• Loss of control on important issues.
• Fear of market insulation due to local partner’s presence.
• Less efficient communication.
• Poor resource allocation.
• Difficult to keep objectives on target.

All these things are worth considering before you decide to go down this route.

After Merck KGaA was forced to pull the Cladribine program it has partnered with Japan’s Ono Pharmaceuticals. A new release showed the oral MS drug in the partnership, ONO-4641, eliminated a large percentage of brain lesions which are associated with the disease. The study recruited 407 patients and after 26 weeks of a daily dose, investigators found that the mid-range dose had eliminated 92% of the brain lesions compared to the placebo. Although there appears to be cardiovascular issues as well as liver enzyme elevation, the MS drug showed some promising results and now faces the stiff competition from other developers Biogen Idec’s BG-12 – among them and Teva Pharmaceuticals laquinimod pill.

Phase II clinical data on Agenus’ HSPPC-96 vaccine (also known as vitespen) was just announced at the 80^th American Association of Neurological Surgeons Annual Scientific Meeting. The study focused on more than 40 patients suffering from Glioblastoma multiforme (GBM) the most common and most aggressive of the brain cancer known as gliomas. The cancer is difficult to treat and most patients die within a year. In the study, 93% of the patients were alive at 9 months compared to only 68% with standard treatments. Further studies will look at a combination of the vaccine and bevacizumab.

At the beginning of the month, Galeterone, a product of Tokai Pharmaceuticals, showed efficacy and was well tolerated in a Phase I study. In the trial of 49 patients, nearly half of the patients had a reduction of prostrate specific antigen (PSA) levels, whilst others had a reduction in tumour size.

And finally a ‘universal’ cancer vaccine in Phase I/II trials for 15 patients with multiple myeloma recurring after remission. This ‘universal’ vaccine, known as ImMucin, works by targeting the MUC1 antigen on the surface of the cells and is being developed by Israeli comoany Vaxil BioTherapeutics. In ongoing trials, the 7 patients which have completed treatment have shown a fall in the number of plasma cells, with 3 of those now back in remission. The story has been hailed in the news worldwide as a miracle cure but at present this can only really be counted as interesting interim Phase I/II results.

The researchers have now formed a company, ReXceptor, and are looking to raise funding for a phase I clinical trial. The jump from mice to humans is one of the most difficult steps in drug development but the results could be amazing!