Government policies affecting intellectual property rights and the review of food and health care products dramatically influence investments in research leading to the development and sale of products that serve unmet medical needs or provide consumers with safe sources of food and drug products at a low cost. When statutes that affect several regulatory agencies are revised within a short time period, institutions that rely on exclusive rights offered by those agencies in exchange for obligations of disclosure and compliance must alter their business plans to adjust to new rules leading to the benefit conferred by the government. In 2011, the Leahy-Smith America Invents Act (AIA) was passed, changing many aspects of the federal statutes relating to the United States Patent and Trademark Office (PTO), and in 2010, the Patient Protection and Affordable Care Act (PPACA) was passed, which included the Biologics Price Competition and Innovation Act (BPCIA), requiring the United States Food and Drug Administration (FDA) to establish an abbreviated regulatory approval pathway for complex macromolecules produced in living cells or organisms. This series of articles briefly reviews key aspects of the AIA and the BPCIA, plus recent court cases relating to complementary periods of exclusivity offered by the FDA and the PTO, which should be of great interest to academic and corporate institutions having an interest in the life sciences. Important aspects of the AIA will be discussed in the first article in this series…
Tag: <span>FDA</span>
Traditionally, the Six Sigma framework has underpinned quality improvement and assurance in biopharmaceutical manufacturing process management. This paper proposes a neural network (NN) approach to vaccine yield classification and compares it to an existing multiple linear regression approach. As part of the Six Sigma process, this paper shows how a data mining framework can be used to extract further value and insight from the data gathered during the manufacturing process, and how insights into yield classification can be used in the quality improvement process.
Manufacturers of biological products have come to accept that it makes sense, from both a business as well as a regulatory perspective, to address GMP compliance issues with bioprocessing methods as early as possible in product development. Logically, this same reasoning would also apply to the associated analytical methods used to characterize the product; however, companies still frequently leave methods optimization and validation until later in the developmental timeline which can expose them to unexpected regulatory challenges. In addition, as therapeutics increase in complexity (e.g., cell therapies, transgenics), it raises the likelihood that product characterization will be assessed by novel and increasingly intricate assays—making it difficult to follow a “one size fits all” approach to method selection, development and validation…
Recombinant DNA-transduced cellular products encounter the product development and regulatory issues of both gene therapy and cellular therapy products. The characterization of recombinant DNA-transduced cellular products remains highly challenging for both sponsors and regulatory agencies. The regulatory concerns and product testing for such cellular products are similar to those for all biologicals. These concerns include the demonstration of product safety, identity, purity, and potency; the control of the manufacturing process to ensure the consistency of product manufacturing under a proper quality control program; and the demonstration of reproducibility and consistency of product lots by means of defined product lot release testing criteria…
Since the first gene therapy trials were conducted 25 years ago, there have been high expectations from the public, and much attention from investors, that previously incurable diseases would be cured by gene therapy. Still, despite numerous gene therapy clinical trials for many different indications, there are no approved gene therapy drugs in the United States. In 1999, one gene therapy patient died during clinical trials, the first ever. This highly publicized event led to heightened regulatory scrutiny over all such trials. Then in 2003 and 2005, three subjects developed leukemia as a direct consequence of gene therapy; one of them eventually passed away. The regulatory response stemming from these incidents led to greater regulatory oversight in gene therapy, as compared to other investigational drugs and biologics…
New regulatory initiatives often produce paranoid responses. These over-reactions are often a result of initial rumors fueled by less-than scrupulous consultants or by misinterpreted statements reported out of context from unscripted regulators. The “remote monitoring capability” incorporated into the emerging Process Analytical Technology (www.fda.gov/cder/OPS/PAT.htm) initiative is a prime example. Put the fear back in the closet: remote monitoring will not lead to unannounced or secret FDA electronic visits, unscheduled remote audits, or regulatory spying on industry processing activities…