Market Access 2030: AI-Driven Dossier Transformation and Therapeutic Value Communication Across Global Health Systems
Author(s)
Stefan Walzer, MA, PhD1, Helmut Butscher, MSc2, Lutz Michael Vollmer, MSc1, Douglas Robertson Niven, PhD3, Catherine Bacon, MSc4, Elaine J. Wright, MSc5, Mel Dean Walker, PhD6.
1MArS Market Access & Pricing Strategy GmbH, Weil am Rhein, Germany, 2Patinova, Merzhausen, Germany, 3Niven Biopharma Y8499447H, Valencia, Spain, 4Fingerpost Consulting Ltd, Cheadle, United Kingdom, 5WRIGHT Pharma Partnering ltd, Dunboyne, Ireland, 6Healthcare BioPharma Futures Limited, Wexham, United Kingdom.
1MArS Market Access & Pricing Strategy GmbH, Weil am Rhein, Germany, 2Patinova, Merzhausen, Germany, 3Niven Biopharma Y8499447H, Valencia, Spain, 4Fingerpost Consulting Ltd, Cheadle, United Kingdom, 5WRIGHT Pharma Partnering ltd, Dunboyne, Ireland, 6Healthcare BioPharma Futures Limited, Wexham, United Kingdom.
OBJECTIVES: The FDA’s Enterprise-level Structured Application (ELSA) initiative represents a structural turning point in how clinical evidence, particularly for novel and high-impact therapies, will be submitted and evaluated. It introduces machine-readable dossiers and AI-compatible formats, laying the groundwork for intelligent regulatory and payer interfaces. As high-cost therapies in oncology, neurology, and rare diseases become increasingly complex, the role of AI in translating real-world outcomes into structured value propositions becomes central to future access.
METHODS: A multidisciplinary analysis was conducted combining policy review, organizational change theory (Kotter, Lewin), and historical analogues from regulatory science (eCTD adoption), precision oncology (genomic-driven submissions), and adaptive licensing models. Use cases from gene therapy, diabetes digital therapeutics, and multi-arm oncology trials were analyzed to project AI’s role in shaping evidence generation, dossier structure, and HTA alignment.
RESULTS: By 2030, value dossiers for complex therapies are expected to evolve into living, AI-generated assets that integrate:
(1) Real-time real-world data (RWD) pipelines from wearable, genomic, and biomarker platforms;
(2) AI-based simulation models to optimize pricing and risk-sharing scenarios for subpopulations;
(3) Dynamic, versioned dossier components enabling adaptive reimbursement aligned with clinical updates.
Structured HTA systems (e.g., NICE, G-BA) are likely to adopt modular appraisal frameworks supported by AI to address therapeutic heterogeneity and uncertainty. Historical analogues show similar transformation patterns: the eCTD shift in 1997 reduced regulatory friction; genomics integration in oncology created new evidentiary norms; and adaptive licensing pilots in Europe initiated cross-agency collaboration. Europe’s policy alignment via EHDS, the AI Act, and JCA provides the infrastructure for scaling these changes.
CONCLUSIONS: To ensure access and sustainability, stakeholders must co-develop AI-compatible standards that reflect the therapeutic and evidentiary complexity of modern medicine. Adoption will be accelerated with the use of health technology specific frameworks that address risks of bias, transparency and reproducibility.
METHODS: A multidisciplinary analysis was conducted combining policy review, organizational change theory (Kotter, Lewin), and historical analogues from regulatory science (eCTD adoption), precision oncology (genomic-driven submissions), and adaptive licensing models. Use cases from gene therapy, diabetes digital therapeutics, and multi-arm oncology trials were analyzed to project AI’s role in shaping evidence generation, dossier structure, and HTA alignment.
RESULTS: By 2030, value dossiers for complex therapies are expected to evolve into living, AI-generated assets that integrate:
(1) Real-time real-world data (RWD) pipelines from wearable, genomic, and biomarker platforms;
(2) AI-based simulation models to optimize pricing and risk-sharing scenarios for subpopulations;
(3) Dynamic, versioned dossier components enabling adaptive reimbursement aligned with clinical updates.
Structured HTA systems (e.g., NICE, G-BA) are likely to adopt modular appraisal frameworks supported by AI to address therapeutic heterogeneity and uncertainty. Historical analogues show similar transformation patterns: the eCTD shift in 1997 reduced regulatory friction; genomics integration in oncology created new evidentiary norms; and adaptive licensing pilots in Europe initiated cross-agency collaboration. Europe’s policy alignment via EHDS, the AI Act, and JCA provides the infrastructure for scaling these changes.
CONCLUSIONS: To ensure access and sustainability, stakeholders must co-develop AI-compatible standards that reflect the therapeutic and evidentiary complexity of modern medicine. Adoption will be accelerated with the use of health technology specific frameworks that address risks of bias, transparency and reproducibility.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA228
Topic
Health Policy & Regulatory, Health Service Delivery & Process of Care, Health Technology Assessment
Topic Subcategory
Systems & Structure, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas