DOES UNMET NEED SHAPE THE INDIRECT COMPARATIVE EVIDENCE SUBMITTED FOR HTA? EVIDENCE FROM PRECISION ONCOLOGY ACROSS SEVEN COUNTRIES
Author(s)
Diana Rebeca Acosta Focil, MD1, Ahmad Hecham Alani, PharmD1, Mackenzie Mills, PhD1, Panos Kanavos, BSc, MSc, PhD2.
1HTA-Hive (Hive Health Optimum Ltd.), London, United Kingdom, 2London School of Economics and Political Science, London, United Kingdom.
1HTA-Hive (Hive Health Optimum Ltd.), London, United Kingdom, 2London School of Economics and Political Science, London, United Kingdom.
OBJECTIVES: Indirect treatment comparisons (ITCs) are frequently used in health technology assessment (HTA) when direct comparative evidence is unavailable. This study evaluated whether the presence and type of unmet need described in precision oncology HTA reports were associated with ITC use and the choice of ITC method.
METHODS: Public HTA reports evaluating precision oncology therapies published between 2020 and 2024 were identified across seven countries. Unmet need statements were classified as treatment availability, clinical benefit, or patient-centred unmet need. ITC use was identified at the report level. Multivariable logistic regression examined the association between each unmet need category and ITC use after adjustment for HTA decision-making country group (cost-effectiveness, clinical-benefit, or budget-impact) and HTA outcome. Among reports including an ITC, the distribution of ITC methods was summarised by unmet need category.
RESULTS: Of 676 reports included, 250 (37.0%) incorporated an ITC and 435 (64.3%) reported at least one unmet need. Reports describing unmet need were more likely to include an ITC than those reporting none (45.5% vs 21.6%). ITC use was highest among reports describing patient-centred unmet need (71.4%), although this category was uncommon (n=14), followed by treatment availability (n=221, 55.2%) and clinical benefit (n= 218, 38.1%). After adjustment, treatment availability (OR 3.46; 95% CI 2.19-5.48), clinical benefit (OR 2.14; 95% CI 1.39-3.29), and patient-centred unmet need (OR 4.70; 95% CI 1.36-16.21) were associated with significantly greater odds of ITC use. Treatment availability unmet need was most frequently accompanied by matching-adjusted indirect comparisons (MAICs) and naïve comparisons, whereas clinical benefit unmet need was most frequently accompanied by MAICs and network meta-analyses.
CONCLUSIONS: Precision oncology HTA reports describing unmet need were more likely to include indirect comparative evidence. Modest variation in ITC methods across categories suggests the type of unmet need described may be associated with the comparative evidence submitted to HTA.
METHODS: Public HTA reports evaluating precision oncology therapies published between 2020 and 2024 were identified across seven countries. Unmet need statements were classified as treatment availability, clinical benefit, or patient-centred unmet need. ITC use was identified at the report level. Multivariable logistic regression examined the association between each unmet need category and ITC use after adjustment for HTA decision-making country group (cost-effectiveness, clinical-benefit, or budget-impact) and HTA outcome. Among reports including an ITC, the distribution of ITC methods was summarised by unmet need category.
RESULTS: Of 676 reports included, 250 (37.0%) incorporated an ITC and 435 (64.3%) reported at least one unmet need. Reports describing unmet need were more likely to include an ITC than those reporting none (45.5% vs 21.6%). ITC use was highest among reports describing patient-centred unmet need (71.4%), although this category was uncommon (n=14), followed by treatment availability (n=221, 55.2%) and clinical benefit (n= 218, 38.1%). After adjustment, treatment availability (OR 3.46; 95% CI 2.19-5.48), clinical benefit (OR 2.14; 95% CI 1.39-3.29), and patient-centred unmet need (OR 4.70; 95% CI 1.36-16.21) were associated with significantly greater odds of ITC use. Treatment availability unmet need was most frequently accompanied by matching-adjusted indirect comparisons (MAICs) and naïve comparisons, whereas clinical benefit unmet need was most frequently accompanied by MAICs and network meta-analyses.
CONCLUSIONS: Precision oncology HTA reports describing unmet need were more likely to include indirect comparative evidence. Modest variation in ITC methods across categories suggests the type of unmet need described may be associated with the comparative evidence submitted to HTA.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA263
Topic
Health Technology Assessment, Methodological & Statistical Research, Study Approaches
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology