Model-Informed Decision Making from Drug Discovery to Reimbursement: A Roadmap for Clinical Pharmacology in...

The transition to value-based healthcare demands more efficient, evidence-driven approaches to drug discovery, development and medical use. Clinical pharmacology is uniquely positioned to support this shift through model-informed drug development (MIDD) decision-making, integrating quantitative methods across the product lifecycle. This commentary outlines a roadmap for embedding MIDD from early drug discovery through regulatory evaluation and ultimately into reimbursement and real-world use. We highlight how pharmacokinetic/pharmacodynamic modeling, mechanistic modeling, disease progression models, patient-relevant endpoints, and real-world data will inform dose selection, optimize trial design, and reduce uncertainty in benefit-risk and cost-effectiveness assessments. Emphasis is placed on cross-stakeholder alignment, including regulators, payers, clinicians, and industry, to ensure that model-informed evidence translates into demonstrable patient and economic value. Key challenges, such as data integration, model transparency, and acceptance by decision-makers, are discussed alongside practical solutions. By advancing a lifecycle-based, quantitatively informed framework, clinical pharmacology can play a central role in delivering therapies that are not only safe and effective but also aligned with the principles of value-based healthcare.