Organised by SciCom
Co-Organised by: Thomas Hartung, Johns Hopkins University
14 – 18 February, 2013, Boston USA

Annual Meeting, American Association for the Advancement of Science (AAAS):
The Beauty and the Benefits of Science

Independent clinical trials are fundamental to improving the results, safety, and cost-effectiveness of health care. Because of costs, the scope of these trials is often limited to new products. Important clinical interventions, surgery, physiotherapy, and rehabilitation are of less interest to commercial sponsors seeking primarily drug and device authorization. Whereas investigator-driven studies now find support in the United States and European Union, they cannot match the resources available for commercial clinical trials. Public and charitable funding typically backs health care–orientated trials independently of manufacturers, but is often much smaller in size. Results matter. When we do get such new evidence, it allows us to look back: how good was the preclinical research to start with? While evidence-based medicine is the health care imperative of the 21st century, it remains difficult to generate the evidence base for pressing public health challenges. This session unites key stages and world authorities in the clinical trials process to explain why and what must change. With expertise on leading animal and human studies to end products, speakers will provide new insights into how clinical trials are conducted. Their common cause will be to argue that greater international cooperation and standards are required. That said, effective communication of clinical and societal benefits must be better coordinated to citizens, patients, health-care professionals, researchers, policy-makers, and society as a whole.

Scroll down or click here to see photos of the event Moderator, Discussants and Speakers

Danish Flag Moderator:
Klaus Bock,
Danish Ministry of Science, Technology & Innovation
US Flag Discussant:
Wilson M. Compton,
U.S. Department of Health and Human Services
EU Flag Discussant:
Anne Glover,
European Commission


Presentation Title: Look Back in Anger? What Clinical Trials Tell Us About Preclinical Research

German Flag Speaker:
Thomas Hartung,

Johns Hopkins University, Baltimore, MD
Friday, 15 February, 2013, Room 309 (Hynes Convention Center)

Talk Description:

Misled by animal studies? Wherever we give a closer look to the outcome of clinical trials in a field such as recently stroke or septic shock, we see how limited the value of our preclinical models was. For all indications, 92% of drugs, which enter clinical trials will not make it to the market, despite all promise of the animal models used to develop them. Drug development already starts to decrease its reliance on animal models: In Europe for example, despite increasing R&D expenditure animal use by pharmaceutical companies dropped from 2005 to 2008 by more than 25%.

What does this tell us for areas, where we have no or few clinical trials to correct? Toxicology is a prime example, where regulatory decisions for products traded at $10 trillion per year are taken only on the basis of such testing. Are we sorting out the wrong candidate substances? Aspirin would today likely fail the preclinical stage. Rats and mice predict each other for complex endpoints with only 60% and predicted together only 43% of clinical toxicities observed later. New approaches under the name Toxicology for the 21st Century are currently emerging, which rely on molecular pathways of human toxicity.

For drug efficacy testing, doubt as to animal models is also increasing: A National Academy of Science panel analyzed recently the suitability of animal models to assess the human efficacy of countermeasures to bioterrorism: It could neither identify suitable models nor did it recommend their development, but called for the establishment of other human relevant tools. In line, about $200 million have been made available by NIH, FDA and DoD agencies over the last year to start developing a human-on-a-chip approach.

There is no reason to assume that other preclinical animal research is more predictive than the one carried out in drug industry. Begley and Ellis reported recently that only 6 out of 53 landmark studies in cancer could be reproduced by industry and similarly Bayer one year earlier reported only about 25% reproducibility. The conclusion: Publish less but of better quality and do not rely on the face value of animal studies.

Presentation Title: Optimizing the Value to Society of Industry-Sponsored Clinical Research

US Flag Speaker:
Jay Siegel,
Janssen Research and Development, Raritan, NJ
Friday, 15 February, 2013, Room 309 (Hynes Convention Center)

Talk Description:

Clinical research, due to its costs and timelines, often stands as the rate limiting step in the translation of the extraordinary advances being made in biology and medicine into health benefits. A substantial portion of clinical research is funded by the pharmaceutical and medical device industries to support market access, market penetration, and reimbursement of products. While private and government funded research supplements this research, there is much that can be done to ensure the industry-funded, product-directed research addresses societal needs optimally. In this talk I will compare the clinical information needs and value equations of regulators, payers, policymakers, providers, and patients. Several aspects of clinical research design that play a role in ensuring efficient addressing of critical needs will be discussed, with examples. Some public policy measures that can impact the efficiency and utility of industry-sponsored trials will be identified.

Presentation Title: The Latest Clinical Trial Research on Brain Reward Systems

Irish Flag Speaker:
Paul J. Kenny,
The Scripps Research Institute, Jupiter, FL
Friday, 15 February, 2013, Room 309 (Hynes Convention Center)

Talk Description:

Wouldn't it be wonderful to attribute our compulsion for addictive damaging activities and the leading causes of preventable death, such as overeating, smoking or taking illicit drugs, wholly to our genetic make-up and parents? In this presentation, I will discuss the latest clinical and preclinical findings behind compulsive behaviour, made in conjunction with teams of global researchers. Personalized medicine provides plenty of research linking genetics and disease. But establishing a relationship between genetic variation and behavior is trickier. I will discuss what the most recent clinical trials tell us about gene and environment interactions. How does over-consumption of high-fat food trigger addiction-like neuro adaptive responses in our brain-reward circuitry? How does nicotine work as the principal reinforcing component in tobacco smoke responsible for addiction? Why are less than 25% of heroin users proven to be dependent, while other addictive substances need one try for a permanent susceptibility to addiction to occur? I will prove that genetics plays but one part. My talk will demonstrate that compulsive behaviour usually comes about after extended access and shed new light on how the three-legged stool of biological, psychological and social elements work together.

Link to sessions:

Ann GloverPaul Kenny

Klaus BockJay Siegel

Thomas HartungWilson Compton