Abstract
ObjectiveThe transferability of the EU joint clinical assessment (JCA) reports for pharmaceuticals for the German benefit assessment was evaluated by systematically comparing EU JCA and German clinical assessments (CA) based on established assessment elements for HTA and assessing the potential impact of differences on Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) ability to derive the therapeutic added value.MethodsIdentification of all pharmaceuticals undergoing both, EU JCA and German CA between January 2016–June 2020. Qualitative review and data extraction from the assessments, assessment of methodological differences using a hierarchical model. Recommendations for harmonisation were developed and consented with pharmaceutical industry stakeholders.ResultsDifferences with potentially major impact: (1) View on differing treatment algorithms and definition of corresponding subpopulations/respective comparators. (2) Clinical relevance of surrogate/intermediate endpoints. Inclusion of different/surrogate morbidity endpoints resulting in different relative effectiveness conclusions. (3) Tolerance of study interventions not used according to marketing authorisation. (4) Different operationalisation and/or weighting of individual safety endpoints leading to differing relative safety conclusions. Differences with potentially minor impact: (1) Disagreement in risk of bias assessment for overall survival and its robustness against study limitations. (2) Use of patient-reported outcome symptom scales as measurements for health-related quality of life instruments.ConclusionWhile many synergies between EU JCA and German CA exist, we identified several aspects in HTA methodology that would benefit of harmonisation and ensure the transferability of future EU JCA to the German HTA process without duplicated evaluation requirements. For those, a set of recommendations was developed.
Highlights
Over the past decades, Health Technology Assessments (HTAs) for new medicines have become a standard feature in many European countries as part of their reimbursement decision-making processes [1, 2]
While many synergies between EU joint clinical assessments (JCA) and German clinical assessments (CA) exist, we identified several aspects in HTA methodology that would benefit of harmonisation and ensure the transferability of future EU JCA to the German HTA process without duplicated evaluation requirements
JA3 EU JCA were included in the analysis to reflect the most current state of EUnetHTA methodology, which has evolved over the three Joint Action (JA)
Summary
Health Technology Assessments (HTAs) for new medicines have become a standard feature in many European countries as part of their reimbursement decision-making processes [1, 2]. Differences in HTA methodologies across European countries are well known [2–5] and may lead to delayed and unequal access by patients to medicines in Europe [6, 7]. Since 2006 and until 2021, the European network for Health Technology Assessment (EUnetHTA) developed and piloted methods and processes for cross-border collaboration on HTA in Europe, including > 20 joint clinical assessments (JCA) for pharmaceuticals and > 30 for medical devices, on a EU-funded project basis [8–11]. The European Commission, Council and the Parliament have very recently (June 2021) reached a compromise on the EU HTA Regulation, expected to come into force in 2024. A main objective of the regulation is to establish common rules and methodologies for JCA, to further promote convergence and to reduce duplication of submissions across the EU [12, 15]
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