Advocating From the Margins: How Patient Input Aligns With Broader Evidence Tolerance in HTA Within Oncology
Author(s)
Ahmad Hecham Alani, PharmD1, Abhinaw Sharma, MSc1, Lehar Chellani, MSc1, Fiona Tolkmitt, BSc1, Mackenzie Mills, PhD1, Panos Kanavos, BSc, MSc, PhD2.
1HTA-Hive (Hive Health Optimum Ltd.), London, United Kingdom, 2Department of Health Policy, London School of Economics and Political Science, London, United Kingdom.
1HTA-Hive (Hive Health Optimum Ltd.), London, United Kingdom, 2Department of Health Policy, London School of Economics and Political Science, London, United Kingdom.
OBJECTIVES: HTA bodies vary in how they integrate patient perspectives. NICE (England), PBAC (Australia), and HAS (France) maintain defined channels for patient input, while Germany’s G-BA limits patients to non-voting observer roles. This study aims to assess and quantify the influence of patient input on recommendations for breast cancer and non-small-cell lung cancer across these four jurisdictions.
METHODS: This was a retrospective analysis of 162 appraisals (2020-2024). Key variables and analyses focused on: (1) characterising the presence and type of patient input; (2) examining the association between patient input and favourable outcomes; and (3) assessing the influence of patient input on the acceptance of key endpoints. Statistical associations were evaluated using chi-squared tests, Fisher’s exact tests, and effect size measured by Cramér’s V.
RESULTS: Patient input was reported in 96% of PBAC (43/45), 70% of NICE (30/43), and 44% of HAS (18/41) appraisals. Most input came from patient organisations (69%), followed by patient experts (31%) and individuals (26%). Across all agencies, the presence of patient input was not significantly associated with a positive recommendation (p > 0.60). However, patient input was significantly more common in appraisals using surrogate endpoints—especially in NICE (27/30) and PBAC (37/43), with a strong association overall (p<0.001; V = 0.37, medium-to-large effect), and only a suggestive trend for HAS. Notably, no patient input was considered in submissions where clinical endpoints were the primary outcomes in NICE (9/13), HAS (6/19), or PBAC (0/2).
CONCLUSIONS: Agencies diverge considerably in how they incorporate and weigh patient input, with significant variability in both frequency and influence. While patient contributions are more commonly present in appraisals using surrogate endpoints, their overall impact on recommendations appears to be limited. These findings suggest that although patient voices are increasingly acknowledged, their practical influence on decision-making remains muted and inconsistent across jurisdictions.
METHODS: This was a retrospective analysis of 162 appraisals (2020-2024). Key variables and analyses focused on: (1) characterising the presence and type of patient input; (2) examining the association between patient input and favourable outcomes; and (3) assessing the influence of patient input on the acceptance of key endpoints. Statistical associations were evaluated using chi-squared tests, Fisher’s exact tests, and effect size measured by Cramér’s V.
RESULTS: Patient input was reported in 96% of PBAC (43/45), 70% of NICE (30/43), and 44% of HAS (18/41) appraisals. Most input came from patient organisations (69%), followed by patient experts (31%) and individuals (26%). Across all agencies, the presence of patient input was not significantly associated with a positive recommendation (p > 0.60). However, patient input was significantly more common in appraisals using surrogate endpoints—especially in NICE (27/30) and PBAC (37/43), with a strong association overall (p<0.001; V = 0.37, medium-to-large effect), and only a suggestive trend for HAS. Notably, no patient input was considered in submissions where clinical endpoints were the primary outcomes in NICE (9/13), HAS (6/19), or PBAC (0/2).
CONCLUSIONS: Agencies diverge considerably in how they incorporate and weigh patient input, with significant variability in both frequency and influence. While patient contributions are more commonly present in appraisals using surrogate endpoints, their overall impact on recommendations appears to be limited. These findings suggest that although patient voices are increasingly acknowledged, their practical influence on decision-making remains muted and inconsistent across jurisdictions.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA27
Topic
Clinical Outcomes, Health Technology Assessment, Organizational Practices
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
Oncology, Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)