Concerns for safety in administration of injectable drug products have escalated in recent years. As a result, scrutiny of administration practices has increased. Pharmaceutical manufacturers are placing greater emphasis on providing the best patient and caregiver experience as well as improving the convenience of drug administration. In fact, many drugs that are regularly administered for chronic conditions are now being offered for at-home preparation and administration. These trends highlight the importance of providing therapies that are not only effective, but…
Manufacturing
Addressing Business Models, Reimbursement, and Cost of Goods
The early ISCT organization provided a powerful forum for sharing solutions, developing standards, and moving the emerging concepts in cell therapy forward as the field grew up and out of academia. Currently, the ISCT organization is uniquely positioned to facilitate sharing of best practices, standards, and strategies across the for- profit cell therapy industry through its Commercialization committee. The Business Models, Reimbursement and CoGS (cost of goods sold) subcommittee of the ISCT Commercialization committee was formed to address several key…
Industry Educational Platforms Drive Commercialization Objectives
Within the International Society for Cellular Therapy’s (ISCT’s) Industry Commercialization committee, Tracey Lodie, director of immunology and stem cell biology at Genzyme, chairs the Industry Education subcommittee, which was established in May 2010. In an interview with BPI, she described the subcommittee’s objectives and how they tie into the manufacturing, testing, and commercialization challenges for cellular therapies. Reducing the Risk “ISCT is working toward becoming an informational hub, acting as a resource to de-risk cell therapy…
Building from the Ground Up
New treatment modalities — as transformative as they may be of our approaches to human healthcare — still need to be profitable for their developers, provide the sorts of returns desired by investors, and be accessible to patients financially. As many industry experts have told us, the venture capital climate these days is much different from that of the early, giddier days of monoclonal antibodies. And with criteria still-emerging around the world for how regenerative medicines are and…
Working Together for the Future
Most individuals who choose to pursue a career in healthcare would say they do so because they are driven by a fundamental desire to help people. If you ask people why they decided to work in the field of regenerative medicine, many will tell you it’s because they believe it is the most exciting area of medical research and that it holds the greatest potential to transform medicine as we know it. The transformational potential of stem cells and regenerative…
Questions to Consider When Thinking About Companion Tests
Personalized medicine is a catch phrase of the 21st century — and with good reason. Advances in genetics and biochemistry promise to tease apart factors that explain why some patients benefit dramatically from a therapy whereas others receive no benefit at all. They also help explain side-effect profiles. To accomplish such lofty goals, drug makers are increasingly partnering with diagnostics companies to develop companion biomarkers. But these companies operate in very different business and regulatory environments, so partnerships can be…
Combination Products for Biotherapeutics
Combination products (see the “Definition†box) are experiencing steady growth in the pharmaceutical industry. According to one report, about 30% of products currently in development are combination products (1). Expanding interest in such products can be attributed to manufacturers’ need to generate new market value for current products that will soon lose patent, requirements for long-term patient care, pressure to reduce healthcare costs, and consumer interest in localized drug delivery with improved therapeutic effectiveness (2). During the 2008…
Efficient Aggregate Removal from Impure Pharmaceutical Active Antibodies
Polishing with membrane chromatography (MC) has achieved acceptance as state-of- the-art technology for charged impurities. Traditionally, anion-exchange (AEX) and cation-exchange (CEX) membrane chromatography have been used to remove charged contaminants such as host-cell proteins (HCPs), recombinant DNA, protein A, endotoxins, and viruses. In monoclonal antibody (MAb) processes, polishing steps usually follow a protein A affinity column step. In some cases, CEX capture is applied, either with at least one AEX or a combined AEX and CEX step. The latter may…
Review, Approval, and Marketing of Biosimilars in the United States
Development, testing, review, approval, and marketing of biosimilar drugs in the United States presents unique safety and regulatory challenges (1). By contrast with traditional small-molecule drugs, biologics derive from sources such as genetically engineered cell lines, plasma, and tissue specimens (2,3). Patents on first-generation biologics approved and marketed in the United States have begun to expire, thereby opening the market to less-expensive “generic†versions. The complexity of biologic drugs and the specificity of their source materials, however, make…
Stability Considerations for Biopharmaceuticals: Overview of Protein and Peptide Degradation Pathways
To ensure product safety and efficacy, protein therapeutics must meet defined quality characteristics immediately after manufacture as well at the end of their designated shelf lives. Many physical and chemical factors can affect the quality and stability of biopharmaceutical products, particularly after long-term storage in a container–closure system likely to be subject to variations in temperature, light, and agitation with shipping and handling. Compared with traditional chemical pharmaceuticals, proteins are considerably larger molecular entities with inherent physiochemical complexities, from their…