USING SHORTFALL METRICS TO PRIORITIZE THERAPEUTIC INNOVATION IN ONCOLOGY
Author(s)
Ariel Sun, MA1, Neda Aminnejad, PhD2, Sonja Sorensen, MPH3;
1Thermo Fisher Scientific, San Francisco, CA, USA, 2Thermo Fisher Scientific, Toronto, ON, Canada, 3Thermo Fisher Scientific, Bethesda, MD, USA
1Thermo Fisher Scientific, San Francisco, CA, USA, 2Thermo Fisher Scientific, Toronto, ON, Canada, 3Thermo Fisher Scientific, Bethesda, MD, USA
OBJECTIVES: To apply a quality-adjusted life-year (QALY) shortfall-based portfolio modeling approach to oncology indications to quantify unmet need and assess potential health gains of hypothetical pipeline assets across cancers, disease stages, and lines of therapy relative to standard of care (SoC).
METHODS: A QALY shortfall-based portfolio model was applied across oncology indications, analyzed separately for solid tumors and hematologic malignancies. QALY shortfall represents the difference between QALYs expected under SoC and those expected for an age- and sex-matched general population, estimated as absolute and proportional measures. Indication-specific survival and utility inputs for SoC were sourced from published Kaplan-Meier data in pivotal trials, supplemented by HTA reports and clinical guidelines. Hypothetical pipeline assets were modeled by applying assumed hazard ratios (HRs) to extrapolated SoC survival curves, with HRs derived from median or landmark survival outcomes specified in target product profiles. Incremental QALY gains versus SoC were estimated for each indication.
RESULTS: Among hematologic malignancies, QALY shortfall varied substantially, with higher shortfalls observed in aggressive disease with limited effective treatment options (e.g., relapsed/refractory acute myeloid leukemia), and lower shortfalls in indications with more favorable prognosis and established therapeutic options (e.g., newly diagnosed multiple myeloma). Absolute shortfalls varied by approximately eight-fold, with proportional shortfall ranging from 10%-80%. Among solid tumor indications, QALY shortfall similarly varied by disease stage and availability of curative or disease-modifying therapies. For example, QALY shortfall increased by approximately five-fold from perioperative cutaneous squamous cell carcinoma to metastatic non-small cell lung cancer, with proportional shortfall ranging from 20%-90%. Overall, indications with greater QALY shortfall may offer greater opportunity for meaningful health gains from new interventions.
CONCLUSIONS: QALY shortfall-based portfolio analysis quantifies unmet need across oncology indications and identifies settings where new intervention may yield the greatest health impact. This approach supports evidence-based prioritization of development opportunities in early portfolio decision making.
METHODS: A QALY shortfall-based portfolio model was applied across oncology indications, analyzed separately for solid tumors and hematologic malignancies. QALY shortfall represents the difference between QALYs expected under SoC and those expected for an age- and sex-matched general population, estimated as absolute and proportional measures. Indication-specific survival and utility inputs for SoC were sourced from published Kaplan-Meier data in pivotal trials, supplemented by HTA reports and clinical guidelines. Hypothetical pipeline assets were modeled by applying assumed hazard ratios (HRs) to extrapolated SoC survival curves, with HRs derived from median or landmark survival outcomes specified in target product profiles. Incremental QALY gains versus SoC were estimated for each indication.
RESULTS: Among hematologic malignancies, QALY shortfall varied substantially, with higher shortfalls observed in aggressive disease with limited effective treatment options (e.g., relapsed/refractory acute myeloid leukemia), and lower shortfalls in indications with more favorable prognosis and established therapeutic options (e.g., newly diagnosed multiple myeloma). Absolute shortfalls varied by approximately eight-fold, with proportional shortfall ranging from 10%-80%. Among solid tumor indications, QALY shortfall similarly varied by disease stage and availability of curative or disease-modifying therapies. For example, QALY shortfall increased by approximately five-fold from perioperative cutaneous squamous cell carcinoma to metastatic non-small cell lung cancer, with proportional shortfall ranging from 20%-90%. Overall, indications with greater QALY shortfall may offer greater opportunity for meaningful health gains from new interventions.
CONCLUSIONS: QALY shortfall-based portfolio analysis quantifies unmet need across oncology indications and identifies settings where new intervention may yield the greatest health impact. This approach supports evidence-based prioritization of development opportunities in early portfolio decision making.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE141
Topic
Economic Evaluation
Disease
SDC: Oncology