Implementing High-Cost Precision Medicine: Health Economic Evaluation, Advanced Modelling, and Equitable System Design

Precision medicine is changing how healthcare is planned and delivered. Comprehensive genomic profiling, targeted therapies, cell and gene therapies, liquid biopsies, and data informed diagnostic pathways are creating new opportunities to improve patient outcomes. At the same time, these developments are placing greater pressure on healthcare budgets, payment systems, clinical services, and health data infrastructure.High treatment and diagnostic costs, uncertain long term outcomes, fragmented reimbursement arrangements, limited evidence at the time of approval, and unequal access can make it difficult for health systems to determine which technologies should be adopted and how they should be delivered in practice. These challenges are particularly important for technologies that require specialised infrastructure, highly trained staff, complex referral pathways, or long term monitoring.Economic evidence in precision medicine has often focused on individual technologies or clinical interventions. This includes important work in cancer care, where genomic testing, targeted therapies, immunotherapies, cell and gene therapies, and complex diagnostic pathways are changing treatment decisions and resource requirements. However, decision makers also need to understand the wider effects on health systems, including changes in service capacity, workforce requirements, diagnostic pathways, resource use, patient costs, and access across different populations.This Research Topic will examine the economic, financial, and health system implications of precision medicine. It will bring together research on health economic evaluation, health technology assessment, real world evidence, financing and reimbursement, service delivery, implementation, and equity.The Research Topic will cover the following areas:- Health economic evaluation and health technology assessment of precision medicine technologies and services, including applications in cancer care. - Relevant areas may include comprehensive genomic profiling, liquid biopsies, multi gene panels, targeted therapies, immunotherapies, cell and gene therapies, biomarker guided care, and other advanced diagnostics and treatments.- Cost effectiveness, cost utility, budget impact, resource use, value of information, and other forms of economic evaluation for precision medicine.- Approaches for addressing uncertainty in economic and clinical evidence, including the use of surrogate outcomes, immature survival data, small patient populations, changing treatment pathways, and evidence generated after market access.- Evaluation of precision medicine across the full technology lifecycle, from development and adoption to implementation and longer term use in routine care.- Financing, pricing, and reimbursement models for high cost precision medicine technologies, including value based payment, outcomes based agreements, managed entry arrangements, subscription models, annuity based financing, risk sharing, and coverage with evidence development.- Payer coverage decisions, funding thresholds, reimbursement timelines, and international comparisons of health technology assessment and pricing systems across single payer and multi payer healthcare systems.- Real world resource utilisation and cost mapping using electronic health records, claims data, disease registries, linked datasets, and other sources of real world evidence.- Discrete event simulation, agent based modelling, system dynamics, microsimulation, decision analytic modelling, and other quantitative methods applied to precision medicine.- Simulation and modelling studies addressing diagnostic pathway redesign, service capacity planning, genomic testing, laboratory services, treatment delivery, waiting times, workforce requirements, and the organisation of care.- Methods for using real world data to estimate costs, address missing data, link datasets, assess variation in care delivery, and evaluate the transferability of findings between healthcare systems.- Health equity and access to precision medicine, including socioeconomic, geographic, demographic, and health system inequalities.- Distributional cost effectiveness analysis and other approaches for assessing the effects of precision medicine across different population groups.- Patient and family financial burden, out of pocket costs, financial toxicity, and the wider economic consequences of precision medicine for adult and paediatric populations.- Implementation and delivery of precision medicine in resource constrained settings and low and middle income countries.- Workforce readiness, clinical training, laboratory capacity, bioinformatics, data infrastructure, procurement, referral pathways, and organisational changes required to deliver precision medicine effectively.- Patient and public preferences, stakeholder perspectives, and approaches to involving patients, clinicians, payers, health system leaders, and communities in decisions about the funding and prioritisation of specialised technologies.- Policy research that provides practical recommendations for coverage decisions, health technology assessment, financing, implementation, and the sustainable delivery of precision medicine.- Research focused on cancer care and precision oncology is explicitly within the scope of this Research Topic when it addresses questions related to health economics, financing, reimbursement, resource utilisation, equity, service delivery, or implementation. This includes research on genomic testing, biomarker guided treatment, targeted therapies, immunotherapies, cell and gene therapies, cancer prevention, diagnosis, treatment, survivorship, and supportive care.The collection also welcomes studies examining precision medicine in other disease areas and healthcare settings, including rare diseases, inherited disorders, cardiovascular disease, neurological conditions, and other fields where advanced diagnostics or therapies create significant economic and health system challenges.We welcome all article types, including Original Research, Systematic Reviews, Methods, Policy and Practice Reviews, Policy Briefs, Perspectives, Brief Research Reports, and Study Protocols.All submissions must be within the scope of the journal and section to which they are submitted. Topic editors: Hadi Akbarzadeh Khorshidi, Sam Pollard, Adam J N Raymakers Source: https://www.frontiersin.org/research-topics/86641/implementing-high-cost-precision-medicine-health-economic-evaluation-advanced-modelling-and-equitable-system-design