Today, many if not all of the major pharmaceutical companies have specific budgets for large research and development pipelines for new biologics including vaccines, monoclonal antibodies, recombinant proteins and even gene therapy projects. In addition, small innovative companies are continually presenting us with novel treatment opportunities which hold great promise for the future. The reason for such huge optimism in the field of biologics is that there are now a number of âblockbusterâ drugs for which the safety profile has been very successfully managed. This success has been achieved following a considerable amount of time and financial investment by both the innovators and the regulators…
Category: <span>Risk Analysis and Management</span>
A patent permits the patent holder to keep others out. That is, a patent holder can prevent others from making, using, selling, offering to sell, or importing an invention within the United States and its territories and possessions. In the United States, these exclusionary rights generally last for 20 years from the date on which the patent application was filed. What the patent holder can keep others out of is defined by the âclaimsâ of the patent. Patent claims describe the boundaries of the patent, much like a real property deed outlines the borders of a parcel of land. However, having a patent does not guarantee that the patent holder is free to use the claimed inventionâŠ
Many precautions are taken in a typical research lab to ensure the integrity of biological specimens. Temperature, storage, and personnel access, among others, are all tightly controlled, and codified into standard operating procedures (SOPs), if not almost biblical law. And no wonder â companies have millions invested in biotech solutions whose progress is often measured in years and decades. Scientists have their lifeâs work on the line. So, it is with some surprise that the diligence most companies exercise during the research and development process is not always maintained during specimen transport. Every time a specimen leaves the lab, be it for further analytical testing or investigational purposes, it runs a heightened risk of contamination, especially from fluctuating temperatures. Ensuring that this does not happen should be the responsibility and concern of everyone with a stake in a biological productâs successâŠ
Acute Renal Failure (ARF) is a severe inflammatory disease state often accompanied by multiple organ failure (MOF). ARF is precipitated by many factors such as blood loss, surgery, sepsis, toxins, trauma, and is most often linked to the loss of kidney tubule function. Proximal tubule cells are specifically injured in acute renal failure. Current therapies for ARF involve conventional kidney support with hemodialysis or hemofiltration. These therapies offer replacement of normal renal functions such as waste removal, fluid, and electrolyte balance, but they cannot provide vital endocrinological and metabolic functions of a healthy kidney. Despite advances in synthetic materials and extracorporeal circuits for hemodialysis and hemofiltration, ARF is associated with a high mortality rate ranging between 55â70 percent…
One of the fundamental foundations of bioprocess system design is the use of âclosedâ systems for production. Closed systems and equipment are utilized to prevent contamination of the product. They are also used as a means of containment to protect not only the product, but also workers and the facility environment. The concept of a closed processing system makes common sense. What is surprising is how closed systems are defined and referenced within the body of regulatory guidance documents and how companies differ in their implementation of a âclosed manufacturingâ concept. What is not surprising is the impact that closed systems have on facility design…
Regulations and common sense require that a manufacturer qualify both the raw materials and the suppliers the firm will use to produce GMP supplies of a pharmaceutical or biopharmaceutical. Because the firm is accepting legal liability for the quality of all raw materials, it makes good business sense to devote resources to these activities. This article explores how to assure that qualification is complete, efficient, and compliant with current good manufacturing practice (cGMP), and discusses the challenges faced by today’s manufacturers…
From an intellectual property (IP) standpoint, probably the two biggest problems I encounter in my practice for early-to-middle stage technology companies are: (1) their failure to fully understand and keep abreast of the competitive intellectual property environment , and (2) their failure to institute procedures that will permit and encourage development of a strategic intellectual property portfolio. By “strategic,” I mean an intellectual property portfolio that focuses on both an offensive and defensive position â a portfolio that not only covers the product and all aspects of its manufacture, production, and applications (defensive portfolio development), but also provides significant blocking positions with respect to competitors’ efforts…
The United States Pharmacopeia (USP) is a 184-year-old organization that has been in the forefront of technology since its inception. From publishing a manual about how to prepare therapeutic potions, USP has evolved into a compendium of standards and information on manufactured pharmaceutical products, with more than 4,000 monographs covering drug substances and biologics, and their dosage forms, excipients, and nutritional supplements. It is not surprising that the USP initiative in cell and gene therapy and tissue engineering has closely followed the emergence of these technologies…
Long-term growth of the biopharmaceutical industry is increasingly relying on outsourcing to overcome the current capacity constraints, especially for monoclonal antibody production. Companies are often reluctant to commit to building multimillion dollar manufacturing facilities for potential products with no guarantee of approval. Therefore to offset risks, companies will enter into contract manufacturing arrangements…
Xenotransplantation has been defined by the US Public Health Service (PHS) as any procedure that involves the transplantation, implantation, or infusion into a human recipient of either (a) live cells, tissues, or organs from a nonhuman animal source, or (b) human body fluids, cells, tissues, or organs that have had ex vivo contact with live, nonhuman animal cells, tissues, or organs (PHS Guideline on Infectious Disease Issues in Xenotransplantation). In the US, several xenotransplantation clinical trials are in progress under US Food and Drug Administration (FDA) oversight. The PHS and the FDA have issued guidelines and guidance documents to address the potential for cross-species infection posed by the use of xenotransplantation products in humans. To minimize the infectious disease risk, these documents provide recommendations on how to screen and maintain source herds, individual source animals, and when possible, xenotransplantation products themselves. However, while precautions can be put in place to remove exogenous infectious agents, the endogenous retroviruses that a source species may carry cannot be removed…