Balancing Rigor and Feasibility of Real-World Data Demands in Germany Within the EU HTA Context: How Fit for Purpose Is the Routine Practice Data Collection (Anwendungsbegleitende Datenerhebung, AbD)?
Author(s)
Ulrike Kuchenbecker, PhD1, Renata Majewska, MSc2, Anna Danuta Kuciara, MPH2, Clement Francois, PhD3, Simon Pannett, PhD4.
1Principal HTA&Market Access, Putnam, Mannheim, Germany, 2Putnam, Krakow, Poland, 3Putnam, Paris, France, 4Putnam, London, United Kingdom.
1Principal HTA&Market Access, Putnam, Mannheim, Germany, 2Putnam, Krakow, Poland, 3Putnam, Paris, France, 4Putnam, London, United Kingdom.
OBJECTIVES: Since 2021, G-BA can initiate routine practice data collection (AbD) for drugs for which only limited meaningful data is available at the time of approval. Aim of this study was to evaluate the usability of AbD-data to identify critical success factors regarding implementation and their relevance in re-assessments.
METHODS: A structured review of IQWiG´s conceptual reports and G-BA decisions was conducted to identify barriers and enabling factors across study design, operational implementation, and methodological acceptability.
RESULTS: 21 indications in high-need areas were assessed to date: 7 proceedings were initiated, 6 were discontinued, 5 AbD are still ongoing, 3 were not started. The first re-assessment using AbD-data is planned for April 2028. Key challenges identified include delays in protocol finalization, limited fit of existing registries, and difficulty achieving adequate confounder control in non-randomized settings. Central hurdle was to identify enough patients in Germany for inclusion or manufacturer´s decision not to submit a study protocol. Registries with clearly aligned endpoints, governance, and stakeholder coordination were more likely to meet evidentiary needs. In contrast, using existing registries to set up an AbD was proved as inefficient because they often don´t meet the methodological requirements of IQWiG and G-BA.
CONCLUSIONS: Since 2021, number of AbD-initiatives is limited and often fail due to low patient numbers. With no re-assessment to date and within the full data package at time of review, impact of these data remains questionable. Early dialogue, registry planning, and methodological alignment can help transforming AbD from a formality into a strategic tool for HTA. Within the new EU HTA procedure, a European-wide registry should be advocated to address the challenges and ensure the investment is spread across multiple geographies. This may require more initial coordination. However, if the evidence is essential to securing access, then the upfront investment would be worthwhile.
METHODS: A structured review of IQWiG´s conceptual reports and G-BA decisions was conducted to identify barriers and enabling factors across study design, operational implementation, and methodological acceptability.
RESULTS: 21 indications in high-need areas were assessed to date: 7 proceedings were initiated, 6 were discontinued, 5 AbD are still ongoing, 3 were not started. The first re-assessment using AbD-data is planned for April 2028. Key challenges identified include delays in protocol finalization, limited fit of existing registries, and difficulty achieving adequate confounder control in non-randomized settings. Central hurdle was to identify enough patients in Germany for inclusion or manufacturer´s decision not to submit a study protocol. Registries with clearly aligned endpoints, governance, and stakeholder coordination were more likely to meet evidentiary needs. In contrast, using existing registries to set up an AbD was proved as inefficient because they often don´t meet the methodological requirements of IQWiG and G-BA.
CONCLUSIONS: Since 2021, number of AbD-initiatives is limited and often fail due to low patient numbers. With no re-assessment to date and within the full data package at time of review, impact of these data remains questionable. Early dialogue, registry planning, and methodological alignment can help transforming AbD from a formality into a strategic tool for HTA. Within the new EU HTA procedure, a European-wide registry should be advocated to address the challenges and ensure the investment is spread across multiple geographies. This may require more initial coordination. However, if the evidence is essential to securing access, then the upfront investment would be worthwhile.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
SA15
Topic
Health Policy & Regulatory, Real World Data & Information Systems, Study Approaches
Topic Subcategory
Registries
Disease
Neurological Disorders, No Additional Disease & Conditions/Specialized Treatment Areas, Oncology, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)