Innovation, Precision Medicine, and Future Challenges in Clinical Development

Advancements in basic biology, genetics, analytic and information technologies are revolutionizing drug discovery and development, and the manner in which we conduct clinical research trials. The drive for precision/individualized medicine requires that with the innovative, potent agents entering clinical research, we focus our efforts to ensure patient safety and to select patients with the most opportunity to benefit. Pharmaceutical scientists play an increasingly important role in ensuring that we understand the pharmacologic properties of our new drugs, that we have formulations flexible enough to allow more individualized dose administration and that we have fast, precise and accurate analytical methods to allow rapid decision making. An example of these innovative agents is the development of bi-specific antibodies that target more than one disease pathway. These types of proteins are being explored in therapeutic areas such as immunology and oncology and present unique challenges from a drug development perspective. Associated with these proteins is the challenge to develop biomarkers allowing for early go/no go decisions prior to investing in the large and expensive outcome trials required for regulatory approval and patient access. This issue is highlighted by the fact that even today very well researched endpoints such as LDL-C lowering are still being debated. Finally there is an increased focus on “Real World Data” requiring that we continue to adapt to information acquired long after initial regulatory approvals. Despite these challenges the opportunity to bring advanced treatment to patients that will positively change their lives is entirely within our reach.