Bringing a new pharmaceutical product to market is a unique process based on a number of requirements for supporting a product launch. For a research and development (R&D) company, launching a product into market may seem to be an issue for someone else to handle in the far-distant future and at a much later time. But even at laboratory or early development stages, biotechnology companies should understand the language of pharmaceutical companies and know how that industry operates. Doing so helps biotech companies make early decisions regarding their investigational products that could lead to future success.
Here we focus on medical affairs, a critical part of the pharmaceutical industry. There is no real equivalent to medical affairs in biotechnology, yet it plays an ever-increasing and important role in that industry as well. After describing the role of medical affairs and its key functions, we present a case that highlights its importance both in prelaunch and during launch. The case demonstrates a ...
For decades, innovations in research and production techniques have been driving forces in the biopharmaceutical industry. But market conditions fueled by the economic downturn over the past five years have increased regulatory burdens in the United States and Europe. Rising costs and risks associated with new drug development now require that biopharmaceutical companies manufacture their products more quickly and cost-effectively than ever before. To this end, companies are looking for new ways to reduce expenditures, increase profitability, speed research, enhance their portfolios of pipeline products, reduce pharmaceutical product development timelines, and move products from laboratory to market in record time.
Trends from mega-mergers to a tendency among regulators and payers to deny or only cautiously accept me-too products — have resulted in a shift toward higher-value and higher-risk products. Pressured by regulators and payers, biopharmaceutical companies are now increasingly looking toward unmet...
Polymerases are natural enzymes that are vital to nucleic acid synthesis: DNA polymerase for replication of deoxyribonucleic acid and RNA polymerase for replication of ribonucleic acid. Thus all living things make and use polymerases of their own. But in 1969, the University of Wisconsin’s Thomas D. Brock and Hudson Freeze identified a new species of extremophilic bacterium thriving at 160 °F (70 °C) in a hot spring in Yellowstone National Park. In time, heat-tolerant polymerase isolated from
Thermus aquaticus
(
Taq
) would lead to an amazingly simple laboratory technique with hundreds of valuable uses in molecular biology: the polymerase chain reaction (PCR).
PCR uses in vitro enzymatic synthesis to amplify specific DNA sequences, producing as much as 100 billion copies of a single molecule in just a few hours. It has “revolutionized research in the biological sciences and medicine, and has influenced criminology and law” (
1
). The technique finds utility in many aspects of biological research. It is ...
Since the 1985 approval of the first recombinant human growth hormone (hGH, such as Protropin/somatrem human growth hormone from Genentech, now Roche), the number of clinical indications for therapy with hGH has steadily increased (
1
). That led to a highly successful drug with more than US$3 billion sales in 2011 (
2
). Even so, hGH shares a common problem with most other first-generation protein therapeutics: a very short plasma half-life of just about two hours in humans. Because such biologics are relatively small molecules, they are rapidly eliminated by renal filtration (
3
). So they usually have to be injected daily to accomplish the desired therapeutic effect. The same holds true for antibody fragments (
4
) and for the growing class of alternative protein scaffolds (
5
). Thus, a technology is needed to prolong their plasma half-lives to meet clinical demands. Indeed, resulting “biobetters” could promise lower dosing with longer time intervals, leading to in improved tolerability and enhanced p...
+2Cell therapy promises revolutionary new therapeutic treatments for cancer and other serious diseases and injuries. For example, T-cell therapy response rates of >50% and durable complete response rates of 20% have been reported in patients with metastatic melanoma who had failed other therapies (
1
). In another example, sustained remissions of up to a year were achieved among a small group of advanced chronic lymphocytic leukemia patients upon treatment with autologous T-cells expressing an anti-CD19 chimeric antigen receptor (
2
). Numerous other examples use cell therapy for cardiac repair, bone or cartilage regeneration, organ repair (pancreas or liver), neurological repair (spinal cord or brain injury), correcting genetic defects, and treating infectious diseases such as human immunodeficiency virus (HIV) (
3
).
Such therapies involve the ex vivo expansion and manipulation of different cell types including stem cells and T-cells. Because many are still in early clinical research phases, often their m...
+1 Traditionally, the CaSSS CMC Strategy Forum meetings have provided a scientific focus on the development of biotech drug substances and their manufacture and characterization, leaving the development of drug product formulation and filling, understanding primary containers, and considering novel delivery systems somewhat out of scope. Over recent years, however, the importance of investing more science and technology into drug product development has become evident as different product types, higher protein concentrations, and doses and requirements for improved delivery of biological drug products have increased. The need to give patients larger and more concentrated doses has challenged formulation scientists, who now collaborate with early protein scientists to develop sequences at the earliest stages of development with final drug products in mind. Increasing such volumes and concentrations of drug products is driving the need for development of new technologies that can deliver high doses. Delivery d...
Today’s biomanufacturing operations require constant management of biopharmaceutical process attributes throughout process development and production. Continuous online measurements of pH, dissolved oxygen (DO), oxidation–reduction potential (ORP), and conductivity (Figure 1) allow real-time industrial process monitoring and adjustment. These functions are crucial to process improvement studies and accurate, reliable manufacturing of high-quality products.
“In the pharmaceutical industry, it is extremely valuable to see how an attribute changes with time and correlate that change with parts of the process,” says L. Harry Lam, PhD, a biopharmaceutical manufacturing industry expert. “It is imperative to have effective equipment that provides reliable measurements.”
PRODUCT:
ALL BIOLOGICALS
PROCESS FOCUS:
Production
WHO SHOULD READ:
Process development, facilities, and manufacturing
KEYWORDS:
QUALITY SYSTEMS, MICROBIAL FERMENTATION, PROCESS MONITORING AND CONTROL, WIRELESS TECHNOLOGY, OPTICAL SENSORS
LEV...
Organized by CASSS, an International Separation Science Society, the seventh annual CMC Strategy Forum Europe will focus on improving quality in development and manufacturing of biopharmaceutical products. Led by experts from global regulatory agencies, academia, and industry, this event series explores emerging aspects of chemistry, manufacturing, and controls (CMC) technology and regulation. The forums are designed to maximize dialog among participants. Relatively short and focused presentations set the agenda for panel discussions that engage all who have experience and expertise to share.
Monday, 6 May 2013
The morning workshop on process validation is sponsored by European Biopharmaceutical Enterprises (EBE), an organization that represents biopharmaceutical companies operating in Europe. The afternoon session covers industry modernization with a regulatory perspectives plenary session.
Tuesday, 7 May 2013
Morning Session — Beyond Specifications (chaired by Brendan Hughes and Karin Sewerin):
Specifi...
How can we empower women to advance their own careers? How do we encourage entrepreneurship for more female scientists? What will get more girls excited about science? Those are questions that the Women In Bio (WIB) organization seeks to address as it creates programs and networking events across the country. WIB is an organization of biotechnology professionals whose mission is to promote careers, leadership, and entrepreneurship for women involved in life sciences. Started in 2002 as a small support network by a group of visionary women in Washington, DC, WIB now boasts 10 chapters across the United States, Canada, and India. Membership is soaring as female professionals sign on to this congenial organization of career women dedicated to helping them reach their own goals. WIB’s chapters in North America cover many key metropolitan areas: Atlanta, Boston, Chicago, Montreal, Pittsburgh, San Francisco, Seattle, and the Washington, DC/Baltimore area. Although each chapter reflects its own locale’s characte...
