Full Description
Tackling translational medicine with a focus on the drug discovery development-interface, this book integrates approaches and tactics from multiple disciplines, rather than just the pharmaceutical aspect of the field. The authors of each chapter address the paradox between the molecular understanding of diseases, drug discovery, and drug development. Laying out the detailed trends from various fields, different chapters are dedicated to target engagement, toxicological safety assessments, and the compelling relationship of optimizing early clinical studies with design strategies. The book also highlights the importance of balancing the three pillars: sufficient efficacy, acceptable safety and appropriate pharmacokinetics, all of which are crucial to successful efforts in discovery and development. With discussions regarding the combined approaches of molecular research, personalized medicine, pre-clinical and clinical development, as well as targeted therapies—this compendium is a flexible fit, perfect for professionals in the pharmaceutical industry and related academic fields.
Contents
Pharmaceutical Industry Performance.- Scope: productivity, success rates and costs.- Competitive landscape and regulatory requirements across different geographies. Drivers for increased efficiency, innovative approaches, and outlook for the next decade.- Unmet needs, major therapeutic areas of R&D focus and investment.- New Product Planning: Scope: General overview of patient profiles from different therapeutic areas, competitive intelligence, standard of care considerations, and impact of this information on the definition of NCE and product attributes and timelines.- Discovery Chemistry and Technologies: Scope: The druggable chemistry space, current targets of interest, new discovery technologies to define the binding pockets and compound attributes for a druggable target.- Challenges, examples of innovation and success.- Chemistry (Authors: TBD).- Biology, genomics, target validation.- In silico tools.- Early Discovery: Target of Interest through Lead: Scope: Effective and dynamic cross-functional collaboration between in vitro biology, chemistry, pharmacology, ADME, preformulation and toxicology to achieve Druggable Leads.- Application of in silico, in vitro and in vivo tools and translational approaches and strategies for integrated assessment and creation of druggable and developable lead scaffolds and assessing affinity and druggability at the scaffold level.- FBDD, druggable scaffolds, hit screening and making compounds.- Integrated assessment of Lead readiness, SRA.- Lead Optimization: Scope: Effective and dynamic cross-functional collaboration between in vitro biology, chemistry, pharmacology, ADME, preformulation and toxicology to achieve target engagement with a druggable candidate.- Candidate Selection.- Application of in silico, in vitro and in vivo tools and translational approaches and strategies for integrated assessment and identification of druggable and developable Clinical Candidate(s) with line of sight for clinical development.- Include description of tools, examples of application/case studies.- Developability including formulations and bio-relevant dissolution.- Chemistry scale up.- Early Clinical Development: Scope: Strategies for clinical trial study designs in the context of different therapeutic areas, clinical formulations, safety margins, biomarkers, tailored therapeutics, PK/PD modeling, controlled release formulations.- John Morrison working out the details of this section.- Alternative Drug Delivery: Scope: non-oral formulations based on patient/caregiver/marketing considerations.- Design, definition of compound attributes and development.- Regulatory aspects.- Industry Academia Collaboration.- Outlook for the Future.
