Hardly any industry combines as wide a variety of expert knowledge as the pharmaceutical industry. Hardly any industry invests as much in research and development as the pharmaceutical industry. Hardly any industry involves such a high amount of entrepreneurial risk. And hardly any industry has to fight so hard to gain public recognition for its achievements.
High risks – low prospects of success
The development of innovative medicines is risky, time-consuming and expensive. On an average, ten to twelve years pass before a new substance is approved for marketing. The chances of a substance that is in phase 1 clinical trials to become a success at the time of its approval many years later, are only about ten per cent on average. When the new drug is finally approved, it has to recoup the costs of its own development, the costs of the 90 per cent of failed developments, plus the costs of future medicines. As with an inter-generational contract: Today’s sales will ensure tomorrow’s innovations. Just an example: In oncology, only three in ten approved drugs are successful enough after approval to earn their own costs of research and development accrued so far.
Target identification and target validation
To find a new agent for the treatment of an illness, it is important, in the first place, to understand which processes in the human body are not functioning properly.
Once a so-called target has been identified, that is, a molecule or a biochemical structure whose manipulation might help to alleviate the symptoms of or even cure an illness, it will be validated for pharmacological use. Only when its molecular biological and biochemical functions have been understood is it possible to find out which effects that manipulation may be expected to have on the condition. Testing approaches help researchers to measure and visualize the biochemical processes in cells that are to be investigated. They are specifically developed for the identified target and enable in vitro analysis in test tubes.
Lead identification and lead optimization
After this, complete classes of agents (leads), which are available in libraries, are tested to determine if and how they interact with the target.
Those classes that have shown to have a significant impact on the target are then further analyzed in vitro.
Preclinical and clinical phases
Only very few agents, whose effectiveness and safety have been tested on animals, enter the preclinical phase. If the test results in that phase are positive the selected agents will subsequently be tested on humans during clinical trials. Such trials have to meet the highest legal requirements that are laid down in national and international regulations and laws. In addition, clinical trials are reviewed by the relevant approval authorities and independent ethics committees.
It is not until the results of the clinical trials have shown that the new agent is both effective and safe that an application for approval can be submitted. National and international authorities decide if the accumulated data is convincing enough to grant the pharmaceutical company the approval that is necessary to start marketing the new substance.
Pharmaceutical research and development enters new territories, day after day. This makes it expensive and risky. However, a risk usually holds a chance. For instance, the decreasing cancer mortality rates in first-world countries are mainly due to innovative therapeutic strategies which pharmaceutical companies have been developing and continue to develop in cooperation with leading scientific institutions.
Celgene is a globally active biopharmaceutical company founded in New Jersey, USA, in 1986. Celgene engages in the research, development and commercialization of innovative medicines, mainly for the treatment of various forms of cancer and diseases of the immune system. The German subsidiary of the company, Celgene GmbH, was established in Munich-Riem in 2006.
The author studied molecular biology at Ludwig-Maximilians University in Munich. From 1991 to 2002, he held leading positions at AMGEN GmbH in Munich and AMGEN International in Switzerland. In 2006, Mr Pehl joined Celgene and set up the company’s German subsidiary in Munich. Since 2009, he has been the Regional Vice President of Celgene International with responsibility for the Central European operations.