In this course, we focus exclusively on the dominant role of biologic therapies in modern medicine. In 2020, six of the top ten drugs by revenue were molecules of biologic origin, namely those manufactured primarily by biosynthetic rather than chemical means, with sales of the top selling therapy, the anti-TNF-alpha monoclonal antibody adalimumab, falling just shy of the US$20 billion mark. The lucrative preeminence of biologics is set to continue, bolstered by the introduction of innovative molecular delivery strategies, such as antibody-targeted conjugates, fragments and fusions, as well as by the robust staying power of market leaders. The latter phenomenon is an inevitable consequence of the higher-than-usual regulatory hurdles faced by conventional generic manufacturers seeking to make biosimilars: intended copies of off-patent biologics that, having undergone a strict comparability exercise, are approved by regulatory agencies such as the EMA and the FDA.

This course will survey this changing landscape within an historical framework and will highlight critical scientific and process parameters unique to biologics, that set them aside from conventional small-molecule medicines, including their molecular architecture and mechanisms of action, manufacturing considerations, analytical and functional lot release assays and clinical trial design. We will explore some of the pitfalls by examining a roster of clinical case studies. The capacity of payers to afford these increasingly high-cost therapies in the face of current economic trends will be discussed.

The broad goals of the course are as follows: a detailed understanding of the complexities associated with biologic drugs; a broad familiarity with biologics manufacturing and its inherent variability; a critical understanding of the aspects of biosimilarity; and a familiarity with the clinical implications emerging from the use of biologics.