Pharmaceutical Science Priorities: Discovery, PBPK, and Continuous Manufacturing

Abstract

This comprehensive analysis maps near-term priorities for pharmaceutical sciences across multiple domains to accelerate the development of safe and effective therapies worldwide. Priority discovery themes encompass nextgeneration anti-infectives targeting resistant pathogens, advanced central nervous system agents for neurodegenerative diseases, allosteric and bitopic G-protein-coupled receptor ligands, protein-protein interaction modulators, and epigenetic inhibitors to expand first-in-class therapeutic mechanisms. Translational research engines emphasize physiologically based pharmacokinetic-pharmacodynamic modeling and systems pharmacology integrated with organ-on-chip technologies and increased utilization of human tissue systems to predict variability, optimize dosing regimens, and enable individualized patient care. Manufacturing innovations focus on greener process chemistry, continuous flow reactors, fully integrated end-toend production systems, scalable nanoscale delivery platforms, nonparenteral formulations for macromolecules, and on-demand personalized dosing enabled by advanced devices and printing technologies. Analytical and quality priorities include high-throughput miniaturized screening platforms, real-time aptamer-based bioanalysis, global harmonization of bioanalytical method validation, and biorelevant dissolution tools for enhanced in vivo predictivity. Biotechnology and regulatory science address comparability and biosimilarity frameworks, device-enabled administration systems, Biopharmaceutical Classification System harmonization, refined bioequivalence limits, optimized global clinical trial design, and convergence of biosimilar regulatory pathways for timely patient access to quality medicines.