Outlook for the Future

Abstract

The nature of the pharmaceutical industry is constantly evolving. There is currently a strong demand for new therapeutics to be simultaneously safer, more effective, and less expensive. These seemingly incompatible expectations will likely increase over time, and balancing them will require continuous innovation, such as novel technologies to expand the so-called “druggable” chemical space and new insights to reduce the number and severity of costly clinical failures. As a result, the industry has begun pivoting away from the traditional therapeutic product profile: once-a-day, orally administered small molecule drugs intended for large patient populations. Instead, there is a renewed focus toward more niche or specialty areas, using alternative molecular therapeutic modalities administered with novel delivery technologies, often via non-oral routes to smaller and more specific patient populations.

The shift from traditional to specialty drug candidates has also resulted in larger organizations focusing less on establishing new internal drug discovery expertise and more on leveraging the existing clinical expertise toward new therapies discovered by external partners. Such partners include specialized smaller pharmaceutical organizations or academic groups with novel molecular assets, targeting capabilities or even whole drug discovery platforms. The size and lack of experience of these small entities often necessitate that their technologies be developed with the assistance of larger, more established pharmaceutical organizations. The successful execution of this approach requires a collaborative mind-set to collectively overcome the interconnected drug discovery and development challenges. This includes assessing the progressability and developability of promising drug candidates as well as ensuring pertinent clinical information is translated upstream into continuing discovery efforts.

Despite implementing significant changes, a number of challenges continue to impede the industry’s productivity improvement efforts. These include significant knowledge gaps, effective decision-making in an uncertain environment, and competing stakeholder interests. This chapter explores how these issues were, are, and may be addressed in the past, present, and future.