Chapter 4. Biopharmaceutical Technologies and Processes in Drug Development

Summary

The process from conception into introducing a bio-therapeutic product for medical treatment can be divided into (1) protein drug candidate discovery and development, (2) large-scale protein production, and (3) product development and approval. Therefore, this chapter is organized in three sections, and summarized as follows:

A. Application of Biotechnologies in Drug Discovery and Development: Text in this section focuses primarily on discovery and development of recombinant proteins. Discussion topics in this section include (1) data mining, molecular cloning and characterization of protein drug candidates, (2) optimization of cell expression systems to maximize protein production, (3) optimization of molecular characteristics to maximize pharmacokinetic and therapeutic potentials, and (4) application of proteins and genes as targets to accelerate drug discovery and development. The accompanying story boxes are included to provide readers with learning insight on the topics.

B. Large-scale Production of Recombinant Proteins: A key to successful production of recombinant proteins on the pharmaceutical scale is to identify host cells with maximum efficiency in expressing candidate proteins that are safe and effective at a reasonable cost. In this context, the topics in this section include (1) optimization of genetic constructs and host cells for recombinant protein expression, (2) large-scale cultivation methods and procedures, (3) downstream processing or purification of recombinant proteins on the pharmaceutical scale, and (4) quality assurance and quality control procedures essential to produce products that meet and exceed pharmaceutical quality and safety standards.

C. Biologic Drug Development and Approval: All pharmaceuticals marketed in the United States, including biotechnology products must be proven safe and effective for their intended use. The US Food and Drug Administration (FDA) evaluates the safety and efficacy data collected as part of the preclinical and clinical studies. While there are multiple pathways to accelerate the FDA review process, the final approval decision is based on whether the benefits outweigh the risks associated with the drug candidate. In addition, to highlight the similarity and differences between the review processes of biologic and chemical products, this section also discusses changes taking place within the two divisions—the Center for Drug Evaluation and Research, and Center for Biologics Evaluation and Research—and globalization of the drug approval process.