Keynote abstract
Traditional pharmaceutical biotechnology that includes biofermentation, bioproductionand biotransormation of antibiotics, steroids, vitamins and other natural compounds,has a long tradition and is rooted in the last century. Today, modern pharmaceutical biotechnology still use the knowledge and basic methodology of bioengineering and bioprocessing, obtained from traditional biotechnology, but the paradigmatic change, due to the introduction of modern biomolecular techniques, redefining the definition.Pharmaceutical biotechnology is defined as a science covering all technologies required for the developing, production, manufacturing and registration of biopharmaceuticals.
Biopharmaceuticals, including recombinant therapeutic proteins, monoclonal antibodies and nucleic acid-based medicinal products now represent approximately one in every four genuinely new pharmaceuticals coming on the market. The overall market for this class of pharmaceutics is valued over 40 billion USD annually, with approximately 10 blockbusters, commanding multi-billion annual revenue. The future development is mainly focused on the design of second generation of biopharmaceuticals, including PEG-derived or lipo-derived biologics, as well as on the development of gene therapy. One major problem is that drugs often do not have expected effect. In estimation, 100 million USD are wasted every year in the US alone because patients take therapeutics that are ineffective or have serious side effects. This finding is extremely important in the biopharmaceutical field, due to the high cost of biomedicines, as well as due to the more specific and more selective target action of biopharmaceuticals. Thus, a personalised drug therapy with pharmaco-metabolomics is going to be introduced. In the pipeline, over 500 biopharmaceuticals are undergoingsecond or third phase of clinical trials. Most of these are protein-based although 11 represent gene therapy products and 14 antisense DNA-based or RNAi- based products. Of the overall putative products, 154 aim to treat cancer, second the largest group is composed of autoimmune disorders, than infectious diseases, and cardiovascular diseases.
In the last part of the paper presented, the regulatory situation ob the introduction, development and registration of similar biological products will be presented. Namely, first patents of original biopharmaceuticals have already expired and some “biogenerics” will be introduced into the EU and USA market. The vast differences between biological and small synthetic molecules have stimulated a debate about how much preclinical and clinical testing should be required. It is evidenced that the regulatory requirements for market approval of biosimilars, based on the fundamental criteria of quality, safety and efficacy, must be developed beyond those for medicinal low-molecular-weight generic drugs.
