Associations of Product-Specific and Clinical Evidence Quality Characteristics With Health Technology Assessment Outcomes: A Quantitative Assessment of Appraisals for Drugs for Rare Diseases in England and Germany
Author(s)
Lea Wiedmann, MSc1, Orlagh Carroll, PhD2, Ellen Nolte, PhD1, John Cairns, MPhil1.
1Department of Health Services Research and Policy, London School of Hygiene & Tropical Medicine, London, United Kingdom, 2Department of Infectious Disease Epidemiology and International Health, London School of Hygiene & Tropical Medicine, London, United Kingdom.
1Department of Health Services Research and Policy, London School of Hygiene & Tropical Medicine, London, United Kingdom, 2Department of Infectious Disease Epidemiology and International Health, London School of Hygiene & Tropical Medicine, London, United Kingdom.
OBJECTIVES: Health technology assessment (HTA) outcomes for rare disease treatments (RDTs) vary across countries but how product-specific and clinical evidence characteristics influence HTA outcomes remains unclear. This study explored the association of product-specific and clinical evidence quality characteristics with HTA outcomes in RDT appraisals issued in England and Germany.
METHODS: We analysed RDT appraisals published between 2011-2023 and extracted data for product-specific characteristics (advanced therapeutic medicinal product status, patient age, alternative treatment, and therapeutic area) and clinical evidence quality characteristics (RCT, risk of bias, maturity of survival, and applicability) using a published protocol. In our primary analysis, we defined a positive HTA outcome as either unrestricted recommendation for routine use in England or as major/considerable/minor clinical benefit in Germany. We reported associations with the HTA outcome using logistic regression, controlling for the HTA process. We conducted a secondary analysis using a broader definition of a positive HTA outcome.
RESULTS: We identified 102 medicine-indication pairs. We found that the odds of a positive HTA outcome where higher in Germany when the main study was an RCT (AOR=13.12, 95% CI 2.18, 78.88, p=<0.001). This association was smaller and not significant at the 5% level in England. However, in England, the odds of a positive HTA outcome were higher for RDTs appraised under the highly specialised technology guidance process (AOR=12.26, 95% CI 2.03, 74.08, p=0.006). In Germany, limited appraisals were not significantly associated with a positive HTA outcome. No other variables were significantly associated with the outcome in our primary analysis. Some results of the secondary analysis differ from the primary analysis, showing sensitivity to the choice of definition of a ‘positive’ HTA outcome.
CONCLUSIONS: The findings improve our understanding of the characteristics associated with HTA outcomes, providing insights into HTA systems in England and Germany. Understanding how countries differ can also inform strategies to improve HTA.
METHODS: We analysed RDT appraisals published between 2011-2023 and extracted data for product-specific characteristics (advanced therapeutic medicinal product status, patient age, alternative treatment, and therapeutic area) and clinical evidence quality characteristics (RCT, risk of bias, maturity of survival, and applicability) using a published protocol. In our primary analysis, we defined a positive HTA outcome as either unrestricted recommendation for routine use in England or as major/considerable/minor clinical benefit in Germany. We reported associations with the HTA outcome using logistic regression, controlling for the HTA process. We conducted a secondary analysis using a broader definition of a positive HTA outcome.
RESULTS: We identified 102 medicine-indication pairs. We found that the odds of a positive HTA outcome where higher in Germany when the main study was an RCT (AOR=13.12, 95% CI 2.18, 78.88, p=<0.001). This association was smaller and not significant at the 5% level in England. However, in England, the odds of a positive HTA outcome were higher for RDTs appraised under the highly specialised technology guidance process (AOR=12.26, 95% CI 2.03, 74.08, p=0.006). In Germany, limited appraisals were not significantly associated with a positive HTA outcome. No other variables were significantly associated with the outcome in our primary analysis. Some results of the secondary analysis differ from the primary analysis, showing sensitivity to the choice of definition of a ‘positive’ HTA outcome.
CONCLUSIONS: The findings improve our understanding of the characteristics associated with HTA outcomes, providing insights into HTA systems in England and Germany. Understanding how countries differ can also inform strategies to improve HTA.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA49
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Rare & Orphan Diseases