Beyond RCTs: The Role of Patient Perspectives and Nontraditional Evidence in HTA Decisions for Breast Cancer (BC) and Non-Small Cell Lung Cancer (NSCLC)
Author(s)
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: Appraisals increasingly include non-traditional evidence (NTE), such as RWE and ITCs, yet face scrutiny. Patient input may play help legitimise the use of NTE. This study aims to evaluate the association between patient input and the use and acceptance of NTE, as well as use of surrogate endpoints, and PROs/QoL data.
METHODS: A retrospective comparative analysis examined 162 appraisals for BC and NSCLC (2020-2024), listed in HTA-Hive database across four agencies (G-BA, HAS, NICE, PBAC). Variables captured were: inclusion and acceptance of (1) patient input, (2) clinical vs surrogate primary endpoints, (3) PROs/QoL endpoints, (4) ITCs or RWE, and (5) associated agency judgements. Associations were tested with χ² or Fisher’s exact statistics; agency-specific tests were run whenever pooled analyses masked agency differences.
RESULTS: Patient input was not associated with whether dossiers included ITCs, RWE, or PRO/QoL data, but it was strongly linked to selecting surrogate primary endpoints (pooled p<0.001), particularly for NICE (p=0.0002). When agencies were combined, patient input correlated with acceptance of PFS. Agency-stratified analyses showed pronounced effects: patient input strongly shaped judgements on RWE, ITCs, PFS, and PRO/QoL (all p<0.05). NICE and PBAC shifted toward more positive or neutral views of NTE when patient input was present, HAS remained largely neutral, and G-BA stayed predominantly negative.
CONCLUSIONS: Patient input appears to play a reinforcing role in enabling evidentiary flexibility. Its presence is significantly associated with greater use of surrogate endpoints but not PROs/QoL measures, RWE or ITCs. While pooled analyses showed no significant link with the acceptance of NTE, it was significantly associated with increased acceptance of PFS. Notably, in cost-effectiveness-driven systems such as NICE/PBAC, appraisals that included patient input were more likely to result in positive or neutral judgments of NTE. Patient perspectives may help legitimise the use of NTE.
METHODS: A retrospective comparative analysis examined 162 appraisals for BC and NSCLC (2020-2024), listed in HTA-Hive database across four agencies (G-BA, HAS, NICE, PBAC). Variables captured were: inclusion and acceptance of (1) patient input, (2) clinical vs surrogate primary endpoints, (3) PROs/QoL endpoints, (4) ITCs or RWE, and (5) associated agency judgements. Associations were tested with χ² or Fisher’s exact statistics; agency-specific tests were run whenever pooled analyses masked agency differences.
RESULTS: Patient input was not associated with whether dossiers included ITCs, RWE, or PRO/QoL data, but it was strongly linked to selecting surrogate primary endpoints (pooled p<0.001), particularly for NICE (p=0.0002). When agencies were combined, patient input correlated with acceptance of PFS. Agency-stratified analyses showed pronounced effects: patient input strongly shaped judgements on RWE, ITCs, PFS, and PRO/QoL (all p<0.05). NICE and PBAC shifted toward more positive or neutral views of NTE when patient input was present, HAS remained largely neutral, and G-BA stayed predominantly negative.
CONCLUSIONS: Patient input appears to play a reinforcing role in enabling evidentiary flexibility. Its presence is significantly associated with greater use of surrogate endpoints but not PROs/QoL measures, RWE or ITCs. While pooled analyses showed no significant link with the acceptance of NTE, it was significantly associated with increased acceptance of PFS. Notably, in cost-effectiveness-driven systems such as NICE/PBAC, appraisals that included patient input were more likely to result in positive or neutral judgments of NTE. Patient perspectives may help legitimise the use of NTE.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA54
Topic
Health Technology Assessment, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure, Value Frameworks & Dossier Format
Disease
Oncology