Presentation: KP01

In 1953, the DNA structure was elucidated by Crick and Watson, but it was not until the 1980s that this discovery marked a turning point for the understanding of human pathophysiology as well as for the pharmaceutical industry. With the ability to produce human proteins using modern techniques of biotechnology instead of classical extraction and purification methods, the door to what we today refer to as 'molecular medicine' was opened.

The concomitant transfer of financing instruments (Venture Capital) from IT-related technological developments into molecular medicine, and the founding of many start-up companies in this field in fact created a paradigm shift, not only in thinking and approaching biological problems, but also in the way of working within big pharma towards hypotheses-driven research.

The elucidation of the human genome and the human proteome gave access to a dramatically increased number of potential targets, and also led to a much better understanding of human pathophysiologies. At the same time, it increased the methodological armamentarium which, in turn, revolutionized research, development, and even marketing of new drug opportunities. Above all, it enabled biology to take the lead in the pharmaceutical industry, and at the same time led to a revival of the chemistry of small molecules. Concept research in biology and chemistry was no longer solely the domain of academic research. Industrial researchers also had to invest more time and resources into early research such as target identification, and especially into target validation. Intellectual property was no longer relevant only for patenting new licenses for drug candidates; it also became important to patent targets at the genetic and at the protein level. Nowadays, intellectual property is exercised in a much broader sense than in earlier years.

In the biomedical century, the dependency on and necessity of cooperation among academic institutions, start-up companies and big industry corporations culminated in a revival of the 'public-private partnership.' This new world of molecular medicine enabled us to take new diagnostic principles, both in vitro and in vivo, into consideration. It also resulted in a clear bridging concept between molecular diagnostics on the one side, and therapeutic outcomes on the other side, which consequently led to a renewed need for biomarker research, pharmacogenetics, and to the definition of new clinical endpoints. This development was kindled by the fact that diseases that beforehand could not be treated -- causally or symptomatically-- could now be approached. This development also led to a completely new ranking list of pharmaceutical industries, to an expansion of the pharmaceutical market, and, simultaneously, to a higher degree of competition. Therefore, the time interval between the first product on the market and its follow-up compound by a competitor is becoming shorter and shorter, while at the same time the pressure on follow-up compounds to be superior is increasing steadily. Success rates - even in phase III - are decreasing to such an extent that R&D spending has increased considerably over time. Today, it is estimated that the development of a new medical entity costs about 800 million Euro, which is an enormous burden and a long-term investment for pharmaceutical companies, demonstrating the immense necessity to introduce new controlling instruments for research and development.

There is no doubt that molecular medicine has opened up a variety of new opportunities. This makes molecular medicine not only a buzz word, but a real new paradigm in the way we think, plan, and perform our research and development activities. In the end, it is the patient who really benefits from this development.

Thus, regarding cost containment as a major hurdle to further exploit what molecular medicine is going to offer to us, it is our joint obligation to convince governments, payers within medical systems, doctors and patients about the enormous opportunities ahead of us. It also is our obligation to create awareness that we can only exploit those opportunities if societies are prepared to invest in this new field. Only then will a new world of opportunities to diagnose, treat, and also prevent diseases become a reality. Above all - with the help of regenerative medicine - diseases will not only be stopped, but physiological functions that have been lost due to the disease will be brought back.

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