Speaker: Scott Gottlieb, MD, Senior Advisor, Medical Technology and Regulatory Affairs, Food and Drug Administration, Rockville, Maryland.
One cause of the slow approval process at the FDA is the existence of too many review cycles because of safety and efficacy concerns. The FDA is committed to reducing its review time by 10% in 2004.
For biotechnology products, pharmacogenomic data should be filed. A quality system approach by the FDA encourages new metrics to evaluate a product’s performance. An innovation proposed by the FDA is to use clinical outcomes along with surrogate outcomes (i.e., patient survival versus shrinkage of tumors). Biomarkers can be used as clinical outcomes and better measures of toxicity. Measuring biological agents for impurities slows down drug approval, and better science is needed.
Imaging technology has been proposed to achieve improved clinical outcomes for such illnesses as Alzheimer’s disease, depression, schizophrenia, and dementia. Molecular technology would be used to measure the penetration of a given product into tissues (for example, bone), to determine tumor shrinkage with x-rays, and to evaluate glioblastomas with functional magnetic resonance imaging (fMRI).
Although generic biological agents are not available, they would probably be only slightly less expensive than the brand-name products.
Keynote Address: Translating Genomic Knowledge into Health Benefits: Beyond the Human Genome
Speaker: Eric Green, MD, PhD, Scientific Director, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland.
The human genome was sequenced in 2003. The current major areas of genomic research are the need to identify (1) all functional elements in the human genome and (2) the genes that make proteins from one generation to the next. A large part of biological complexity is not in the genes; rather, it has to do with regulatory elements and noncoding functional elements. As a result, it is important to find these sequences and to be able to recognize them when new ones are found.
Gene-prediction software is marvelously advanced for finding genes but woefully inadequate for finding noncoding functional elements. The problem is that there are no good gold standards. There is not a single region in the genome where all the functional elements are known. Many people thought that by sequencing the mouse genome, all the important regions would be found. Clearly, that is not true.
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Computer-aided sequence analysis may be used to compare human and mouse genomes. It is known that approximately 40% of human and mouse [genomes] are aligned and that 5% of that similar sequence is functionally important (e.g., for encoding proteins). But how does one extract that 5%? There must be some way to discriminate between regions that are similar (but not necessarily significant) and those that are important.
Part of the problem in studying the genome is that it is very large and full of “noise” (or what used to be called “junk” DNA). It is sometimes extremely difficult, at this point, to distinguish what is important from what is not. There is also a need to generate a genome parts list.
Virtually all diseases (e.g., cystic fibrosis, diabetes, arthritis, cardiovascular disease, asthma) have a genetic component. By translating genomic knowledge to medicine, we might be able to improve human health. In addition, diagnostics in the genomic era are exciting because they can improve our understanding of genetic defects. Ideally, pharmacogenomics will lead to matching an individual’s drug therapy to his or her genes.
Biotechnology Health Care: A Market Poised for Explosive Growth
Keynote Address Speaker: Francoise Simon, PhD, Professor of Business, Columbia University, New York, New York.
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The focus of R&D in the post-genomics era is shifting from populations to individuals. This change presents a challenge to the traditional blockbuster brand drugs, which must make way for the targeted brand therapeutic agents, marketed globally to genotype-specific segments. Despite the increasing importance of the targeted model, an absolute shift to this model is unlikely. However, big business factors will continue to drive mass-market blockbuster drugs. One factor is that the patents on several drugs, worth $100 billion in this decade, are scheduled to expire, and companies will thus feel the pressure to develop new blockbusters.
It must be realized that post-genomic technologies have a long-term payoff and are expected to raise R&D costs and risks in the short term (i.e., in the next three to five years) as the number of drug targets rises from the current 500 to more than 5,000. Mergers of large drug companies have increased the scale and the need for growth. Marketing costs have risen since the late 1990s and have, in the U.S., been driven by such factors as the use of direct-to-consumer advertising that have not been successful. kamagra soft tablets
Targeted drugs have a higher return on marketing investment than mass-market products because the focused model is much more cost-effective. The physician audience for a targeted brand might be as small as 5,000 specialists worldwide who are reachable with a small, highly trained sales force; in contrast, a 3,000-person sales force is necessary to promote primary care brands in the U.S.
Patient pools for biotechnology drugs tend to be small and highly motivated, and these patients are more effectively reached via the Internet than by expensive mass media. Targeted therapies are often supplied directly to physicians or hospitals, and the wholesaler share of profits is thereby eliminated. Because customized drugs are largely breakthrough products, they can command high prices.
Bioscience is driving innovation across industry sectors, and the result is the emergence of “biobrands,” which range from bioengineered tissue to molecular diagnostics and drug-device combinations. These new biobrands will increasingly rely on evidence-based medicine, although experience-based marketing will continue to play a role. Products that address critical therapeutic areas, such as oncology, will require evidence-based marketing, whereas products that address primary care diseases, such as allergies, may benefit from a combination of evidence-based and experience-based marketing.
The growth of biotechnology products is explosive. Boundaries are fading between the biotechnology and pharmaceutical sectors as top-tier biotechnology companies have turned into full-fledged biopharmaceutical companies: Amgen’s market capitalization now tops those of Roche and AstraZeneca. In 2002, eight biological agents were in the billion-dollar club, and many have a pattern of sustained high growth, given that they target critical therapeutic areas and have limited competition. Advances in genomics have opened the door for the development of medications designed to target specific genotypes, resulting in more rational drug designs.