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Witness Testimony
Mr. Hal Barron M.D.
NIH: Re-engineering Clinical Research.
Good morning, Mr. Chairman, and thank you for the opportunity to testify before the Subcommittee on the most important issue of clinical trials. My name is Dr. Hal Barron. I am Chief Medical Officer for Genentech, one of the nation's leading biotechnology companies headquartered in South San Francisco, California. As you are no doubt aware, Genentech was founded in 1976 by Herb Boyer and Bob Swanson, and has the unique distinction of being the very first biotech company. Since 1976, a robust and productive industry has grown from the foresight, innovation and risk-taking of Dr. Boyer and Mr. Swanson. Genentech alone has discovered, developed and currently manufacture 13 therapies targeted at such unmet medical needs as cardiovascular disease, Cystic Fibrosis and cancer. Last month, the Food and Drug Administration (FDA) approved Avastin, a groundbreaking therapy that reduces blood supply to tumors. This product was approved for the treatment of colorectal cancer and we are in the process of studying whether the drug is active in a number of different cancers. Ours is a terrific - and unusual - success story. We are a soup-to-nuts company, doing everything from basic research to clinical development to manufacturing to marketing. My written testimony describes for you in detail the myriad steps and challenges present in the discovery, development and manufacturing of breakthrough biologics. My presentation today will focus on one critical piece of the development process absolutely essential to our success - the clinical trials we conduct with our patients. I am delighted to have the chance to discuss with you Genentech's rich experience in drug discovery and development, our experience with clinical trials, and our valuable interactions with the National Institutes of Health (NIH). Also for your edification, I have attached a presentation that provides comprehensive review of our development organization. Genentech has one of the biotechnology industry's most extensive track record in all phases of bringing new disease treatments to patients - from discovery research through development, commercialization and product operations. With 13 protein-based products on the market for serious or life-threatening medical conditions, Genentech has experience taking a drug from A to Z, transforming the seed of an idea in a lab into a novel therapy for a patient in need. Such a fully integrated approach differentiates Genentech from many other biotechnology companies. Although I won't be focusing on how we partner with other biotech/pharmaceutical companies, Genentech has worked closely with such companies as Xoma, Novartis, OSI pharmaceutical, Amgen, and Roche Discovery Research Research is the wellspring of potential products, and Genentech's research organization is among the worlds finest. Genentech scientists are the most prolific in the biotechnology industry, publishing at a rate of 250 to 300 scientific papers a year, and are among the top one percent of researchers in the world in terms of total citations. In addition, Genentech's scientists have secured more than 4,300 patents worldwide and have another 5,000 pending. Discovery research at Genentech focuses primarily on three areas of medicine where there is a strong need for safer, more efficacious therapies: oncology, immunology and vascular biology. In addition, Genentech remains open to other projects where the company has significant opportunities to fill a therapeutic void in important areas of medicine. To ensure continued scientific excellence, Genentech opened the Founders Research center, a 275,000 square-foot, $85 million research facility devoted solely to biotechnology, in October 1992. It was dedicated to Bob Swanson and Dr. Herbert Boyer in honor of their pursuit of the promise of biotechnology when they established Genentech 28 years ago in 1976. In April 2001, the company celebrated its 25th anniversary by breaking ground on the 280,000 square foot expansion of the Founders Research Center. The complex - comprising the existing facility and the new expansion - is the single largest biotechnology research facility in the world, with more than 500,000 square feet of research space containing specialized laboratories and state-of-the-science equipment in several interconnected buildings. Clinical Development Genentech uses a rigorous set of criteria, including scientific factors, medical need and market potential, to determine which projects to move from discovery research into development. The physicians, scientists and medical professionals in Development play the essential role of translating basic science into patient benefit. They help Genentech determine which potential new drugs are tested against specific diseases in the clinic and determine how the chosen drug candidates should move through the many phases of clinical testing. Since these therapeutic proteins must be delivered into the body safely, and their effectiveness must be measured and documented in order to secure marketing approval. These scientists leverage their expertise in clinical medicine, clinical study design, epidemiology, bio-statistics and health care economics to design these trials. They incorporate some of the newest technologies such as molecular diagnostics, imaging studies (such as CT and MRI scans) as well as novel biomarkers into these trials as well. Genentech's development pipeline has both breadth and depth, with projects targeting a range of disease areas across all phases of clinical development. This very broad pipeline requires leadership from the best experts. Our MDs and PhDs come from many prestigious academic institutions such as Harvard, Yale, Stanford, University of California, San Francisco (UCSF) and many other top institutions. Commercialization Commercial translates research and development innovations into changes in medical practice that enhance and extend patients' lives. The Commercial team introduces multiple products into new and different markets, directs pre-launch commercial development activities, and utilizes cutting-edge sales approaches. The Commercial organization is also involved with development activities that bring forward products in the pipeline in the most efficient way to meet the demands of the market and the healthcare community - directing market research, sponsoring medical education efforts, and developing a leading patient reimbursement program. The Commercial team's unique consultative education, sales, marketing, and distribution models have resulted in 13 successfully marketed products to date and have made Genentech a valuable and sought-after partner. Product Operations Biotech's rich promise is only truly fulfilled when its scientific breakthroughs are transformed into safe, effective therapies and made available in quantities sufficient to treat all those in need. This extremely complex and demanding task is the responsibility of various product operations groups in the company, including Process Sciences, Engineering, Quality and Manufacturing. Process Sciences At Genentech, the transition from laboratory production to full-scale manufacturing is the work of the Process Sciences group. This group is made up of five divisions: Cell Culture & Fermentation R&D, Recovery Sciences, Analytical Chemistry, Pharmaceutical R&D, and Manufacturing Sciences. The Cell Culture group grows increasingly larger and more efficient cultures of cells that produce the desired protein. Recovery Science extracts and purifies the protein molecules from the cell cultures, with the goal of both high yield and high purity. Analytical Chemistry is the function that checks to ensure the purified protein is the right one, and that it is active and able to be made into a medicine. And Pharmaceutical R&D determines the formulation-or recipe-for the final medicine, how it should be administered and its packaging. Finally, Genentech's Process Sciences group works closely with the FDA to ensure an approved manufacturing process is in place and pure product is available to patients upon approval of new pharmaceuticals. Quality The Quality group is comprised of two main areas: Quality Control, which executes the many different procedures for testing Genentech's products; and Quality Assurance, which evaluates all documentation to determine whether each procedure was completed correctly. Genentech strictly adheres to federal requirements for quality and collaborates with the FDA to ensure its processes are of the highest standards. Every medicine that leaves Genentech has been subjected to stringent standards and procedures to ensure its quality and purity. Manufacturing Genentech was the first biotechnology company to scale up protein manufacturing successfully from the small quantities used for research to the much larger quantities needed for clinical trials and marketing. With state-of-the-art facilities in the United States and Europe, the company continues to be a world leader in the manufacture of human bio-therapeutics, processing approximately three million liters of product annually for clinical research and the marketplace through a variety of fermentation and proprietary purification processes. In 1998, Genentech completed its second manufacturing facility in Vacaville, California. The largest multi-product biotechnology manufacturing facility in the world, the Vacaville plant occupies 420,000 square feet on 100 acres. It became operational in 1999 and received FDA licensure in April 2000. Also in April 2000, Genentech further expanded its manufacturing capacity with the purchase of a cell culture manufacturing facility in Porriņo, Spain. When renovated and licensed, the facility will supplement Genentech's existing bulk cell culture production capacity. The NIH and Drug Discovery and Development It is these many complex and interrelated steps that explain why the vast majority of drugs approved for patients have been discovered and developed by companies like Genentech. As a research-intensive company, we learn a great deal from the basic research conducted by the NIH. The NIH performs this function extremely well and we support and appreciate the continued increases in funding you have given the NIH over the past several years. In addition, we partner with the NIH on clinical trials particularly in areas outside our area of expertise or where trials could not be conducted without NIH support. This collaboration is extremely important as Genentech or any company for that matter, cannot do everything alone and we greatly benefit from the expertise of the NIH and academia in general. The opportunities for industry/government collaboration have been fruitful and could be even more substantial. One area in which the NIH is particularly well suited to make important advances is that of molecular diagnostics and discovery of novel "bio-markers"- markers that identify which patients with a given disease have the worst prognosis. Advances in identifying biomarkers presents a real opportunity to benefit patients at a much faster pace than today's R&D efforts, and is an area that could enable industry to be more successful in their endeavors to bring targeted therapeutics to market. In addition, there is an opportunity for industry to work with the NIH in developing drugs for indications that the company decides not to pursue. It is clear that a company such as Genentech cannot design and implement all the necessary clinical trials to maximize the benefit of their new therapeutics. Thus, certain patient populations may not always be examined. Whether it is because the trials will take too long, represent too small a population or fall outside a company's focus area, providing the NIH access to our novel therapeutics can and has been invaluable. We hope such activities continue to be funded as they have the opportunity to make a significant difference in patient care. I hope that this presentation has given you a better understanding of the processes and challenges we face in bringing new, breakthrough biologic to market for patients. I hope you also have a better understanding of the relationship between the NIH and private industry. We look forward to exploring these and other partnership opportunities with Dr. Zerhouni and this Subcommittee. Thank you again, Mr. Chairman, for this opportunity to testify before you and the Subcommittee. I am happy to answer any questions you may have. Related Documents |
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