Analysis of Pros Included in the HTA Assessment of Conditionally Approved Oncology Drugs in the UK, Italy, and Germany
Author(s)
Mark Chalmers, PhD1, Vidhyasagari Sundaram, MPH2, PRATEEK KANADE, M. Pharm2, Mansi Deshpande, M. Pharm2, Rory Dunne, MPH3.
1Partner, EY, Dublin, Ireland, 2Ernst & Young, Bangalore, India, 3Ernst & Young, Dublin, Ireland.
1Partner, EY, Dublin, Ireland, 2Ernst & Young, Bangalore, India, 3Ernst & Young, Dublin, Ireland.
OBJECTIVES: To compare and analyze the PROs (patient-reported outcomes) used in HTA assessments of all conditional approvals in oncology from 2015 to 2025 in Germany, France, and the UK
METHODS: Oncology conditional approvals granted between 2015 and 2024 were identified from the EMA website. For these drugs, HTAs published by HAS in France, G-BA in Germany, and NICE in the UK were assessed for the indications for which the conditional approval was granted. The HTA documents were then evaluated for the inclusion of different patient-reported outcomes (PROs), commentary regarding deficiencies, and the impact of PROs on the HTA outcome
RESULTS: A total of 36 conditional approvals were identified. Based on this, a total of 98 HTA assessments were identified for further analysis. ~16% of assessments did not include any PROs, while 53% of them included disease agnostic PROs, and 40% included disease specific ones. A total of 20 unique indications were identified, for which disease-specific (e.g., FACT-LymS, NSCLC SAQ) and disease-agnostic PROs (e.g., EORTC-QLQ-C30, EQ-5D-5L etc.,) were identified. Common challenges with the included PROs highlighted by various HTA agencies include a lack of comparative data, small sample sizes, and poor response rates. The impact of PROs on HTA outcomes was, however, uncertain in almost all cases.
CONCLUSIONS: PROs have gained significant importance in recent years and are increasingly included in various trials, including those approved via the conditional approval pathway. However, there is no direct correlation between the inclusion of PROs and HTA outcomes, considering that other factors such as efficacy, safety, amongst other parameters also play a significant role. Understanding the opinions of HTA bodies regarding the shortcomings in the included PROs would be important for manufacturers to design and include better PROs in the future.
METHODS: Oncology conditional approvals granted between 2015 and 2024 were identified from the EMA website. For these drugs, HTAs published by HAS in France, G-BA in Germany, and NICE in the UK were assessed for the indications for which the conditional approval was granted. The HTA documents were then evaluated for the inclusion of different patient-reported outcomes (PROs), commentary regarding deficiencies, and the impact of PROs on the HTA outcome
RESULTS: A total of 36 conditional approvals were identified. Based on this, a total of 98 HTA assessments were identified for further analysis. ~16% of assessments did not include any PROs, while 53% of them included disease agnostic PROs, and 40% included disease specific ones. A total of 20 unique indications were identified, for which disease-specific (e.g., FACT-LymS, NSCLC SAQ) and disease-agnostic PROs (e.g., EORTC-QLQ-C30, EQ-5D-5L etc.,) were identified. Common challenges with the included PROs highlighted by various HTA agencies include a lack of comparative data, small sample sizes, and poor response rates. The impact of PROs on HTA outcomes was, however, uncertain in almost all cases.
CONCLUSIONS: PROs have gained significant importance in recent years and are increasingly included in various trials, including those approved via the conditional approval pathway. However, there is no direct correlation between the inclusion of PROs and HTA outcomes, considering that other factors such as efficacy, safety, amongst other parameters also play a significant role. Understanding the opinions of HTA bodies regarding the shortcomings in the included PROs would be important for manufacturers to design and include better PROs in the future.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA32
Topic
Health Technology Assessment
Topic Subcategory
Systems & Structure
Disease
Oncology