The current draft of ICH Q12 appears to have taken several steps backward in the pursuit of the manufacturing excellence initiated by ICH Q8 (R2) pharmaceutical development and expanded by FDAās 2011 process validation guidelines…
Category: <span>Regulatory</span>
The same sensor instrumentation can now be used in process development laboratories, clinical trials, pilot plants, and large-scale manufacturing ā thus simplifying product development, scale-up, and regulatory record-keeping…
A considerable clinical need exists for alternatives to the autologous vein and artery tissues used for vascular reconstructive surgeries such as coronary artery bypass graft (CABG), lower limb bypass, arteriovenous shunts, and repair of congenital defects to coronary circulation. So far, devices made from synthetic materials have not matched the Āefficacy of native grafts, particularly in small diameter applications. Recent advances in cell biology and tissue engineering have introduced the possibility of a living, biological graft that might mimic the functional properties of native Āvessels…
Regulatory agencies routinely announce changes in the GMP (Good Manufacturing Practices) norms and quality standards designed to guarantee that every product retains characteristics required for its pharmaceutical use.The alignment with emerging regulatory requirements is mandatory, therefore, to assure production process consistency, as well as the safety and efficacy of the finished product…
In order to move product development forward, the majority of biotech companies and academic institutions involved in cell-based therapies need new facilities in order to scale up production capabilities and comply with evolving regulatory requirements. Some institutions choose to use a contract manufacturing organization (CMO) to benefit from established expertise while others support their clinical development programs with their own dedicated production facility. The main challenges in establishing a dedicated pilot-scale production facility are described hereafterā¦
The analytical characterization of recombinant protein therapeutic drug products has broadened to include the use of more sophisticated technologies. The expansion of technical abilities has translated into increasing the depth and breadth of our knowledge and understanding of the drug product intended for commercialization. With the availability of more precise methods, the regulatory expectations for understanding the characteristics of a protein therapeutic drug product are increasing. A thorough understanding of a therapeutic proteinās biochemical and biophysical characteristics is necessary to support investigational new drug (IND) applications and other drug regulatory filingsā¦
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 assaysāmaking it difficult to follow a āone size fits allā approach to method selection, development and validationā¦
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 criteriaā¦
As pharmaceutical and biopharmaceutical companies become more global in nature with products that have the potential to reach into the worldwide marketplace, a special understanding is needed of the requirements that are specific to varying geographical areas. Specifically, the regions for worldwide pharmaceutical distribution can be broken into America, Europe, and Asia-Pacific, with each region presenting its own regulation and technical challenges. There are many issues that are common among these regions, but each regionās focus may be different. Typically, an issue arises in one region and then migrates to another as people become aware of the issues and concerns. For example, the use of prefilled syringe systems in Europe and Asia has migrated to the American marketplace, amounting to a more significant volumeā¦
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…