Regulatory Affairs

Transforming the Biopharmaceutical Development Roadmap

Biopharmaceutical analytical methods have improved significantly since the early 1990s. Techniques and fundamental mechanisms largely have remained the same, but modern analytical tools are delivering better insights into drug-product characteristics thanks to improved reagent quality, method intensification, and automation. Harmonized regulations and the revolutionized role of contract development and manufacturing organizations (CDMOs) also continue to support bioprocess transformations. Improved Analytics and Standardized Single-Use Equipment Today’s analytical methods provide high resolutions and deliver reliable results quickly. For example, sodium dodecyl sulfate–polyacrylamide…

20 Years of Chemistry, Manufacturing, and Controls Strategy. A Conversation About the CASSS CMC Forum Series

Formerly known as the California Separation Science Society, the CASSS organization has maintained a close relationship with BioProcess International since its inaugural year. In particular, the CMC Strategy Forum series began the same year that BPI’s original staff joined Informa to develop a new international trade publication covering biopharmaceutical development and manufacturing. Beginning with our February 2004 issue, BPI has been proud to publish dozens of reports from this influential series of meetings over the years. Their success around the…

Surveying the Biosimilars Regulatory Landscape

BPI’s history coincides with that of biosimilars development. Although nonpeptide biosimilar products did not begin receiving commercial authorization until the 2010s, health authorities and drug makers already had been exploring the complex concept of biosimilarity. In the May issue of BPI’s first volume, Theresa L. Gerrard (then an independent consultant who also had been director of the Division of Cytokine Biology at the US Food and Drug Administration Center for Biologics Evaluation and Research, FDA CBER) wrote: The potential for…

From PDFs to Digital Submissions The Rapid Pace of Regulatory Change

The pace of regulatory change in the pharmaceutical industry over the past 20 years has been rapid. Although heightened emphasis on patient safety, data transparency, and harmonization has prompted new guidance’s and requirements, perhaps what is most notable is the rate at which technology has changed how processes are documented and regulated. Today, digitization is entrenched in day-to-day life and is steadily becoming the norm among health authorities and at pharmaceutical companies. But just two decades ago, incorporating technology-led regulatory…

eBook: Biopharmaceutical Training — Designing Effective Adult-Learning–Based Programs

Biopharmaceutical industry training is imperative not only to ensure patient safety, but also to develop a skilled workforce that can support successful businesses. Current good manufacturing practice (CGMP) skills and behaviors are needed so that products being developed, manufactured, and delivered are compliant and within specifications. Individuals who fulfill roles within development, manufacturing, and delivery processes need some level of education to develop all the necessary skills and behaviors. Adult earners have unique needs and requirements that must be incorporated…

CMC Strategies for Expedited Program Development — Regulatory Perspectives: Session 1 of a CMC Strategy Forum

In response to increasing demands for expedited availability of biotherapeutics around the world — and with the ultimate goal of patient benefit — health authorities have developed accelerated regulatory pathways to reduce development timelines to product licensure. Because of the complexity and unique nature of each program and product modality, some solutions must be worked out case by case between sponsors and regulatory agencies. On 13 October 2020, CASSS–Sharing Science Solutions presented a virtual session overviewing currently available regulatory pathways…

Managing Host-Cell Proteins: Robust Risk-Assessment Frameworks for Process-Related Impurities in Biological Products

Although biomanufacturing processes are designed to generate highly pure drug substances, some host-cell proteins (HCPs) copurify with target proteins and thus remain in finished drug products. Biopharmaceutical developers are keenly aware that such impurities must be minimized to protect patients. HCPs can activate several kinds of immune responses in treated patients, including production of antidrug antibodies and induction of cross-reactivity with therapeutic proteins (1–5). HCPs also can diminish drug efficacy, potency, and/or stability (6, 7). Thus, regulatory guidances such as…

Hurdles Ahead for Cell and Gene Therapy Makers

Significant growth of the cell and gene therapy (CGT) pipeline in recent years demonstrates the enormous potential of these modalities to treat or even cure otherwise intractable diseases. Several CGT products have been approved for clinical use over the past five years. More than 75 such products have come to the market around the world so far. They include chimeric antigen receptor (CAR) T-cell therapies that involve genetic engineering of patient cells ex vivo as well as in vivo gene therapies…

The CRISPR Saga (So Far)

Since January 2016 (with a brief interlude as described below), the Patent Trial and Appeal Board has been attempting to adjudicate the proper inventorship of CRISPR technology in (to date) six separate patent-interference proceedings. (Scientific “priority has been decided, for now, by the awarding of the Nobel Prize in Chemistry to Jennifer Doudna and Emmanuelle Charpentier in 2020; see the “Priority Claims” box). CRISPR-based gene editing was hailed as the “Breakthrough of the Year” in 2015 (1), and the scientific…

Orphan Drug Designation: Securing the Significant Benefits

When preparing an application for orphan drug designation (ODD) in the European Union (EU), you need to consider key eligibility criteria that are different from those required by the US Food and Drug Administration (FDA). One factor is justifying the “significant benefit” (defined below) of your drug product over existing therapies for an orphan condition. Significant benefit contributes considerably in securing a successful ODD application if the correct approach is taken — as described in two European Medicines Agency (EMA)…