Biomolecules Versus Smaller Chemicals in Toxicology: ICH, EU, and US Recommendations

Abstract

For many life science professionals, biological products represent the cutting edge of medical research and are the smartest means to target and treat a variety of disease and conditions for which the current treatments are still unsatisfactory. In contrast to small molecule drugs, including new chemical entities (NCEs), biomolecules (also called biologics, biologicals, biopharmaceuticals, or biotechnology-derived pharmaceuticals) are complex macromolecules, sometimes occurring as mixtures that are not easily identified or fully characterized. Nevertheless, due to the rapid development of biotechnology in the last three decades, the number of approved biomolecules is increasing at a faster rate than it is the case for new chemical entities. Biologicals are usually highly specific for a target, are more heat sensitive and susceptible to microbial contamination, and are likely antigenic. Thus, the quality and security testing of biologicals is becoming increasingly important. This updated chapter compares the evolving regulatory environment relevant for biomolecules, with a typical “case-by-case” development program versus NCEs, which are generally developed according to a more standard “classical” manner.