Abstract for: An integrated framework for biopharmaceutical drug design, development, and implementation
Biopharmaceutical research and development (R&D) is a high-risk, costly, and very complex process. Quality by Design (QbD), put forward by the International Council on Harmonisation to guide pharmaceutical R&D, is defined from a Chemistry, Manufacturing, and Control perspective to assure quality, i.e. safety and efficacy. However, it does not systematically consider patient perspectives or user requirements. Growing pressure on the pharmaceutical industry to increase efficiency while closing gaps in access to life-saving medicines has led to the use of implementation science as a way to reduce commercial risk and better address patient needs. This research paper defines societal product utility for biopharmaceutical products and draws on a novel practical framework to assess the effectiveness of implementation. It incorporates systematically user perspectives and requirements in the early stages of drug development, while linking to the QbD process. A causal-loop diagram, applicable to a broad range of biopharmaceutical products, captures the internal causal structure for drug design, development, and implementation to assess societal product utility and the effectiveness of implementation. The assessment accounts for contextual factors and shows the broader value of biopharmaceuticals to society. This integrated framework leverages dynamic modeling to proactively assess health technologies from early R&D stages to delivery.