Regulatory Affairs

Bioindustry Researchers Take Strides in Preventing Diseases: Developments in Genetic Diagnosis and Therapy

Johnson & Johnson’s pharmaceutical research arm recently launched a drug-development project that will intercept diseases before patients even realize they’re sick. In the company’s words: “The future of healthcare will increasingly depend on identifying and correctly interpreting the earliest signals of disease susceptibility, preventing or intercepting disease before it even begins.” (1). The first targets include Alzheimer’s, some forms of cancer, heart disease, rheumatoid arthritis, multiple sclerosis, and type 1 diabetes. The company’s Disease Interception Accelerator group will search for…

Elucidation: Centers for the Innovation in Advanced Development and Manufacturing

The 2009 influenza pandemic was the main driver for the Centers for the Innovation in Advanced Development and Manufacturing (CIADM). As a new federal government authority, the Biomedical Advanced Research and Development Authority (BARDA), directed by Dr. Robin Robinson, assembled a team of scientists and technicians with a vast array of knowledge and experience in chemical, biological, radiation, and nuclear fields. BARDA’s directive was to enhance preparedness for catastrophic challenges such as an influenza pandemic or other serious threats to…

Special Report on Assays, Test Methods, and Comparability: The CMC Strategy Forum Series, Part 4, Introduction

The CMC Strategy Forums focus on relevant chemistry, manufacturing, and controls (CMC) issues throughout the life cycle of a therapeutic and thereby foster collaborative technical and regulatory interaction. Forum chairs share information with regulatory agencies to help them merge good scientific and regulatory practices. Outcomes of the forum meetings are published in BioProcess International and on the CASSS website (www.casss.org). This process is meant to help ensure that biopharmaceutical products manufactured with advancing technologies in a regulated environment will continue…

Special Report on Assays, Test Methods, and Comparability The CMC Strategy Forum Series, Part 4, Biosimilar Products: Scientific Principles, Challenges, and Opportunities

The Chemistry, Manufacturing, and Controls (CMC) Strategy Forum held on 22 January 2012 in San Francisco, CA, focused on selected scientific and regulatory aspects in the development of biosimilar products. Such products are an increasingly important area of interest for both the biopharmaceutical industry and its regulatory agencies. Biosimilars are highly complex, so scientists have been unable to demonstrate identity to a level typically possible for small molecules. Consequently, specific scientific and regulatory approaches are required to ensure the high…

Special Report on Assays, Test Methods, and Comparability The CMC Strategy Forum Series, Part 4, The Role of Higher-Order Structure in Defining Biopharmaceutical Quality

Cosponsored by CASSS (an International Separation Science Society) and the US FDA, the 17th CMC Strategy Forum was designed to explore the relationships between higher-order molecular structure and quality of therapeutic proteins and peptides, vaccines, and blood-derived products. Understanding those relationships is important to defining and controlling the critical quality attributes (CQAs) of biopharmaceutical products. The forum program highlighted the current state of the art for analytical tools used to monitor higher-order structure. Case studies demonstrating the effects of changes…

Special Report on Assays, Test Methods, and Comparability The CMC Strategy Forum Series, Part 4, Demonstrating Comparability for Well-Characterized Biotechnology Products: Early Phase, Late Phase, and Postapproval

Challenges and approaches in demonstrating comparability of a well-characterized biotechnology product after manufacturing changes can be as varied and complex as the products themselves. Participants at the January 2005 CMC Strategy Forum sought to discuss and agree on common implementation strategies for different manufacturing change scenarios (1). Development of flexible, comprehensive approaches in strategy development addressed evaluation of critical product characteristics, appropriate process steps to test, numbers of lots and levels of testing required, and assessment of product comparability. The…

Special Report on Assays, Test Methods, and Comparability The CMC Strategy Forum Series, Part 4, The Roles of Bioactivity Assays in Lot Release and Stability Testing

A January 2007 CMC Strategy Forum on the roles of bioactivity assays in lot release and stability testing was held in Washington, DC (1). Its purpose was to promote an understanding of the design and utility of bioassays throughout product development and to delineate the conditions under which surrogate assays could be used to determine product potency. Topics of discussion included appropriate assay selection at each stage of product development, the potential use of binding assays for potency testing, and…

Effective Management of Contract Organizations: Keeping the Product Pipeline Moving, Compliant, and Available

Both small and large biopharmaceutical companies are increasingly pursuing the outsourcing of manufacturing and testing throughout the product lifecycle. The growing use of contract manufacturing organizations (CMOs) and contract testing organizations (CTOs) has led to increasing complexity within the biopharmaceutical industry as more third-party sites are leveraged to support global markets. To address those issues, a CASSS Chemistry, Manufacturing, and Controls (CMC) Strategy Forum was held in Washington, DC, 27–28 July 2014. The title was “Effective Management of Contract Organizations: Sponsors, Contract Organizations, Health Authorities and Patients — Keeping the Product Pipeline Moving, Compliant, and Available.” The CMC Strategy Forum is…

Management, Notification, and Documentation of Single-Use Systems Change Orders: Challenges and Opportunities

Single-use systems (SUS) consist of numerous plastic components derived from different suppliers. As such, they are prone to changes, including alterations in construction materials or modifications in manufacturing processes. Such changes may originate at the immediate supplier or farther back in the supply chain as a result of product improvements, process improvements, part discontinuation, or even business decisions such as manufacturing site relocation. Whether those changes are major or minor, managing their impact on biopharmaceutial processes and product quality often…

Planning for Commercial Scale of Cell Therapy and Regenerative Medicine Products, Part 2: Clinical Efficacy, Reimbursement, and Needle-to-Needle Logistics

Cell therapy is an emerging pillar in healthcare with the potential to provide curative solutions to a wide range of indications. The biological complexities through which cell technologies exert their clinical impact (especially those used in immunotherapies for cancer) provide opportunities for novel modes of immune regulation, cell targeting, and payload delivery. Cells also can serve as vehicles for genetic content, which the gene therapy industry is now investigating. Since early 2004, Invetech has worked with organizations dedicated to cell…