Getting Innovative Therapies Faster to Patients at the Right Dose: Impact of Quantitative Pharmacology Towards First Registration and Expanding Therapeutic Use

Abstract

Quantitative pharmacology (QP) applications in translational medicine, drug-development, and therapeutic use were crowd-sourced by the ASCPT Impact and Influence initiative. Highlighted QP case studies demonstrated faster access to innovative therapies for patients through 1) rational dose selection for pivotal trials; 2) reduced trial-burden for vulnerable populations; or 3) simplified posology. Critical success factors were proactive stakeholder engagement, alignment on the value of model-informed approaches, and utilizing foundational clinical pharmacology understanding of the therapy.

Figure 1

Quantitative pharmacology (QP) is an enabler for faster access to innovative medicines as described through case studies. For the first registration of a novel therapy, QP can accelerate patients' access through rational dose regimen selection for the pivotal registration trials by a self‐estimated ∼0.5–1 year. Moreover, QP can facilitate reducing trial burden for vulnerable populations through effective translation of efficacious concentrations between adult and children and bringing the treatment options ∼1–4 years earlier to children. Finally, QP can enable simplification of dosing (posology) during therapeutic use expansion of a novel therapy. Solid understanding of the clinical pharmacology of the novel therapy, integrated into model‐informed knowledge, effective communication of QP learnings and results to project teams and stakeholders, and alignment on the value of model‐informed learnings by decision makers are all critical factors for QP to impact and influence the drug development process and ultimately bringing novel therapies faster to patients at the right dose.