Assessability of Single-Arm Studies (SAT) Under Joint Clinical Assessment (JCA): Whether Drugs Assessed With SAT in the Last Years Would Fit Under the New EU HTA Framework?
Author(s)
Krzysztof Kloc, MSc1, Mondher Toumi, PhD2, Elzbieta Lukomska, MSc1, Malwina Kowalska, MSc1, Inez Tyrala-Chowaniec, MSc1.
1Clever-Access, Krakow, Poland, 2Inovintell, Krakow, Poland.
1Clever-Access, Krakow, Poland, 2Inovintell, Krakow, Poland.
OBJECTIVES: The Joint Clinical Assessment (JCA) aims to assess the degree of certainty of relative effectiveness (RE) of an intervention over a comparator. While randomized control trials are state-of-the-art, single-arm trials (SATs) are inadequate for estimating RE unless they are paired with an external control. Our objective was to review the French Health Technology Assessment (HTA) opinions driven by SATs and simulate the JCA assessment of these products.
METHODS: SAT-based HTA opinions issued between 2019 and 2024 were identified in the NaviHTA database and described. JCA clinical trial validity guidance was applied to assess identified products.
RESULTS: Out of 883 opinions, 141 (16%) were SATs driven, representing 116 reports and 96 medications. 49% of these opinions were for oncology indications, of which 48% were haematological cancers. 8/141 opinions (6%) included a control group allowing RE assessment. Among those, 4 opinions included unadjusted comparisons and the same number, matching-adjusted approach. SAT-based opinions that included a control, received higher additional benefit ratings (ASMR) compared to those without a control. Despite lacking a control group, 98 opinions (74%) recommended reimbursement. 36 (27%) indicated ASMR ratings from II to IV and 6 (46%), ASMR V. Under JCA, 82% of analysed cases would not qualify for assessment and no report would be issued. 8% would qualify for JCA for a likely limited number of PICOs (Population-Intervention-Comparator-Outcome) but the certainty rating would be low.
CONCLUSIONS: Most of the products developed with SAT and accepted in France would not qualify for JCA review. A minority would be reviewed for a limited number of PICOs, but RE would be rated of low certainty. Contextualisation and deliberative process are instrumental, and would almost systematically reverse JCA conclusions. It is questionable if products with SATs as the primary evidence should be eligible for JCA because it will poorly inform Member States' HTA assessment.
METHODS: SAT-based HTA opinions issued between 2019 and 2024 were identified in the NaviHTA database and described. JCA clinical trial validity guidance was applied to assess identified products.
RESULTS: Out of 883 opinions, 141 (16%) were SATs driven, representing 116 reports and 96 medications. 49% of these opinions were for oncology indications, of which 48% were haematological cancers. 8/141 opinions (6%) included a control group allowing RE assessment. Among those, 4 opinions included unadjusted comparisons and the same number, matching-adjusted approach. SAT-based opinions that included a control, received higher additional benefit ratings (ASMR) compared to those without a control. Despite lacking a control group, 98 opinions (74%) recommended reimbursement. 36 (27%) indicated ASMR ratings from II to IV and 6 (46%), ASMR V. Under JCA, 82% of analysed cases would not qualify for assessment and no report would be issued. 8% would qualify for JCA for a likely limited number of PICOs (Population-Intervention-Comparator-Outcome) but the certainty rating would be low.
CONCLUSIONS: Most of the products developed with SAT and accepted in France would not qualify for JCA review. A minority would be reviewed for a limited number of PICOs, but RE would be rated of low certainty. Contextualisation and deliberative process are instrumental, and would almost systematically reverse JCA conclusions. It is questionable if products with SATs as the primary evidence should be eligible for JCA because it will poorly inform Member States' HTA assessment.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HTA34
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas