Publications by authors named "W G Goettsch"
Roadmap to Innovation of HTA Methods (IHTAM): insights from three case studies of quantitative methods.
Int J Technol Assess Health Care
November 2024
Article Synopsis
- The Innovation of Health Technology Assessment Methods (IHTAM) framework was developed to improve how health technology assessments (HTA) are conducted but needed real-world evaluation.
- The framework was applied to three case studies, and feedback from stakeholders indicated it improved collaboration but suggested the need for a structured roadmap for better applicability.
- A 48-item roadmap was created to offer tailored guidance and actionable steps, encouraging future use of the IHTAM framework among stakeholders for method innovation in HTA.
Objective: To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions.
Methods: A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population.
Objectives: This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.
Methods: A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).
Article Synopsis
- Progress in genetic diagnosis and orphan drug legislation has led to new therapies for rare neurogenetic diseases (RNDs), but challenges remain in academia, regulation, and finances.
- The study aims to create a practical framework for developing patient registries that address these challenges and enhance outcomes in care, research, and drug development for RNDs.
- A comprehensive approach combining literature review, interviews with existing registries, and feedback from various stakeholders was used to ensure the framework meets diverse needs and emphasizes key principles like accessible, independent, and trustworthy data governance.
Background: This work aimed to determine the role and action points for the involvement of medical societies in the European Health Technology Assessment (EU HTA) Methods: An online pre-convention survey was developed addressing four areas related to the EU HTA: (i) medical societies' role; (ii) role of clinical guidelines; (iii) interface with the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS); and (iv) approaching 'best-available evidence' (BAE). A descriptive analysis of questionnaire outcomes was conducted to inform the European Access Academy (EAA) Fall Convention 2023. Within the working groups (WGs), action points were identified and prioritised.
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