Regulatory Affairs

Virtual Audits: A New Reality in the World of COVID-19

The novel coronavirus disease 2019 (COVID-19) pandemic has every industry seeking out ways to accomplish time-sensitive activities using a number of virtual approaches. This is certainly true in the biopharmaceutical sector, in which good manufacturing practice (GMP) audits are required to manufacture medicinal drug products for human use. Examples include supplier/vendor audits, mock inspections, and preapproval and prelicense inspections (PAIs and PLIs) conducted by sponsors and regulatory authorities. Auditors usually perform such activities on site and only sometimes remotely. In…

Cell and Gene Therapies Get a Reality Check: A Conversation with Anthony Davies of Dark Horse Consulting Group

As founder of cell and gene therapy (CGT) specialist firm Dark Horse Consulting Group in California, Anthony Davies speaks from a quarter century of experience including former positions at Onyx Pharmaceuticals, Syrrx, ZymeQuest, Serologicals, Geron Corporation, Capricor, and 4D Molecular Therapeutics — and he currently serves on the board of directors for TrakCel and the scientific advisory boards for Akron Biotech and BioLife Solutions. In his plenary address at the Phacilitate 2020 Leaders World conference (part of Advanced Therapies Week…

Brazilian JBS Federally Inspected Fetal Bovine Serum

Serum is the most commonly used supplement in cell culture. Fetal bovine serum (FBS) is the common choice because it contains high concentrations of growth factors and other important signaling molecules (e.g., adhesion proteins, nutrients, carrier proteins, cytokines, and hormones) required for cell survival and differentiation together with its buffering capabilities. FBS production begins with the collection of whole blood from bovine fetuses under aseptic conditions. Once collected, the blood is allowed to clot, and the serum is mechanically separated.…

Using Blockchain Technology to Ensure Data Integrity: Applying Hyperledger Fabric to Biomanufacturing

Digitalization of manufacturing operations is a major challenge that many industries face. With the advent of smart equipment, automation of unit operations and complete processes, and digitalization of batch documentation, more data are generated now than ever before. The information must remain manageable, and data integrity needs to be ensured. The challenge for biomanufacturers will be to ensure that their entire large output of data will be attributable, legible, contemporaneous, original, and accurate (ALCOA) as defined by the US Food…

Evaluating Biosimilars: A View from the Small-Molecule World

For many years the pharmaceutical industry was dominated by small (usually synthetic) molecules, mixed with a number of nonactive materials and encapsulated or (in the really old days) rolled into pills or pressed into tablets. Although synthesizing the active pharmaceutical ingredients (APIs), formulating the dosage forms, and analyzing the materials at every stage of a product life cycle were not always trivial activities, they were relatively straightforward. Most of the tools needed for analyzing/controlling each step of the manufacturing process…

Bioassay Evolution: Finding Best Practices for Biopharmaceutical Quality Systems

Bioassays help drug developers determine the biological activity (potency) of their products, which has been a biopharmaceutical critical quality attribute (CQA) since long before that concept had a name. Because of their complex nature, bioassays are among of the most challenging experiments to perform reliably with dependably accurate results. Consistent assay performance requires a controlled environment and qualified reagents; skilled analysts who understand cell physiology, regulatory requirements, and the latest techniques; and assay protocols that are intelligently developed, characterized, and…

Cell Banking for Cell and Gene Therapy: Regulatory, Ethical, and Scientific Considerations

Regenerative medicine holds great potential for human disease management, with hundreds of cell and gene therapy (CGT) products for tissue/organ reconstitution or replacement in different stages of development and clinical testing for toxicity, safety, and efficacy. For example, currently more than 60 CGTs have marketing authorization (although many with only conditional approval) from central regulatory agencies worldwide (1). Those products are treating conditions such as hematopoietic malignancies, immunological disorders, and cartilage disorders. Most of those treatments use culture-expanded autologous or…

eBook: Biologics Stability — Lifecycle Management of Drug Products

The biomanufacturing industry’s increasing attention to risk mitigation through quality by design (QbD) and the emergence of complex therapeutic modalities have driven the need for a lifetime-management approach to assuring drug product stability. To that end, industry guidelines have been (or are being) developed to guide the industry toward a “holistic approach” to conducting stability assessments. However, not all methods are stability indicating, and many more industry concerns need to be addressed. This BPI eBook offers perspectives on ICH Q12…

eBook: Speed to IND — Balancing Risk and Reward

With so many biopharmaceuticals obtaining breakthrough or fast-track designations, companies that use accelerated strategies to be first in human studies can be left with significant quality and manufacturing challenges that must be solved later on. Despite regulatory encouragement to create solid design spaces and define parameters according to quality by design (QbD), those may go by the wayside given the pressures of speed. The reward is the investigational new drug (IND) application itself — but if companies lock in subpar…

Addressing Regulatory Requirements for Filter Integrity Testing

Filter integrity is a fundamental element of sterility assurance during production of biopharmaceutical and vaccine products. Integrity test results are a key foundation for drug lot release, so any external element that could affect their reliability must be viewed as a critical issue. But when should a filter integrity test be performed? This article highlights the Sartocheck 5 Plus filter integrity tester as a means to address regulatory requirements. Please fill out the form below to read the full article…