WHEN THERAPIES ARE TARGETED BUT METHODS ARE GUESSING: CLOSING THE GAP IN PRECISION ONCOLOGY EVALUATION
Author(s)
Aishee Ghatak, MSc1, Syed Salleh, PhD2, Thaison Tong, PhD2, Raju Gautam, PhD2, Shilpi Swami, MSc2;
1ConnectHEOR, Delhi, India, 2ConnectHEOR, London, United Kingdom
1ConnectHEOR, Delhi, India, 2ConnectHEOR, London, United Kingdom
OBJECTIVES: Precision oncology (PO) has progressed rapidly with advances in genomic-testing, biomarkers, and companion-diagnostics (CDs), yet health technology assessment (HTA) economic evaluations (EE) remain inconsistent and often inadequately capture genomic heterogeneity, diagnostic value, and uncertainty in small biomarker-defined populations. We reviewed recent policy documents, framework/methodological reviews to identify recommendations and limitations for improving consistency in PO EE.
METHODS: A targeted review was conducted to identify reviews and key papers on PO-related EE frameworks/methodologies (2023-2025). Searches were conducted in PubMed, supplemented by targeted searches of key health economics journals, including Value in Health and Pharmacoeconomics. An Institute for Clinical and Economic Review whitepaper on gene therapy was included to provide complementary policy-level insights into the evaluation of complex, high-cost precision-technologies. Data was extracted on framework characteristics, scope, value dimensions, and methodological features.
RESULTS: Included publications (total=11, predominantly multi-country) fell into three categories. (1) Conceptual reviews (n=3) emphasized diagnostic innovation, adaptive trials, and system-level coordination, highlighting misalignment between scientific advances and reimbursement, and the need to move beyond isolated drug evaluations toward dynamic strategies capturing testing/treatment, sequencing, alternative pathways, and operational realities of PO implementation. (2) Health economic and HTA-reviews (n=4) highlighted evidence immaturity, small biomarker-defined populations, limited integration of CDs and real-world evidence, requiring analytic approaches suited to evolving, heterogeneous data. (3) Policy-oriented documents (n=4) emphasized high upfront costs, long-term uncertainty, and sustainability challenges. A methodological paper proposed biomarker-centric EE framework using basket trials. Across categories, evaluations were fragmented and overly reliant on a single incremental cost-effectiveness ratio, prompting calls for broader decision outputs including cost-consequences, uncertainty, scenarios, and multi-criteria frameworks, alongside adaptive reimbursement, and stronger regulator-payer coordination.
CONCLUSIONS: The literature indicates that traditional EE methods are insufficient for PO, with few operational HTA frameworks available. This underscores the need for flexible evaluation and reimbursement frameworks that support early-access and reassessment across decision makers.
METHODS: A targeted review was conducted to identify reviews and key papers on PO-related EE frameworks/methodologies (2023-2025). Searches were conducted in PubMed, supplemented by targeted searches of key health economics journals, including Value in Health and Pharmacoeconomics. An Institute for Clinical and Economic Review whitepaper on gene therapy was included to provide complementary policy-level insights into the evaluation of complex, high-cost precision-technologies. Data was extracted on framework characteristics, scope, value dimensions, and methodological features.
RESULTS: Included publications (total=11, predominantly multi-country) fell into three categories. (1) Conceptual reviews (n=3) emphasized diagnostic innovation, adaptive trials, and system-level coordination, highlighting misalignment between scientific advances and reimbursement, and the need to move beyond isolated drug evaluations toward dynamic strategies capturing testing/treatment, sequencing, alternative pathways, and operational realities of PO implementation. (2) Health economic and HTA-reviews (n=4) highlighted evidence immaturity, small biomarker-defined populations, limited integration of CDs and real-world evidence, requiring analytic approaches suited to evolving, heterogeneous data. (3) Policy-oriented documents (n=4) emphasized high upfront costs, long-term uncertainty, and sustainability challenges. A methodological paper proposed biomarker-centric EE framework using basket trials. Across categories, evaluations were fragmented and overly reliant on a single incremental cost-effectiveness ratio, prompting calls for broader decision outputs including cost-consequences, uncertainty, scenarios, and multi-criteria frameworks, alongside adaptive reimbursement, and stronger regulator-payer coordination.
CONCLUSIONS: The literature indicates that traditional EE methods are insufficient for PO, with few operational HTA frameworks available. This underscores the need for flexible evaluation and reimbursement frameworks that support early-access and reassessment across decision makers.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PT42
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Trial-Based Economic Evaluation
Disease
STA: Personalized & Precision Medicine