Milestone 44 - Updated review of HTA appraisal approaches for OMPs (WS1 of IMPACT HTA WP10)
| Main Authors: | Elena Nicod, Amanda Whittal, Michael Drummond, Karen Facey |
|---|---|
| Format: | info Lainnya Journal |
| Bahasa: | eng |
| Terbitan: |
, 2020
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| Subjects: | |
| Online Access: |
https://zenodo.org/record/5019109 |
Daftar Isi:
- IMPACT HTA Work Package 10 (WP10) is developing guidance on novel approaches to appraising medicines for rare diseases to support robust, accountable decision-making across Europe on high-cost products that have a limited evidence base with high uncertainty and intense stakeholder scrutiny. WP10 is exploring how different forms of evidence obtained from a range of data sources and stakeholders can be integrated with economic modelling to inform robust, accountable decisions about OMPs and ultra-OMPs. The first step was to document HTA appraisal/reimbursement processes for medicines for rare diseases in all EU Member States, Canada, New Zealand and Australia in WP10 workstream 1. This information was used to explore whether there is a for need supplemental HTA appraisal processes for rare disease treatments (RDTs), and if so, what form these could take. This work was done in several stages: (1) Development of individual country vignettes providing an overview of HTA appraisal processes for rare disease treatments. This was done in close collaboration with HTA/reimbursement experts in each country (via a questionnaire & emails with follow-up questions and validation) and additional desk research. These vignettes are now publicly available on the IMPACT-HTA website https://www.impact-hta.eu/country-vignettes. (2) Peer review publication providing an overview of existing supplemental processes for rare disease treatments in the study countries (soon to be submitted to Orphanet Journal of Rare Diseases). This work characterises the different types of supplemental processes identified for rare disease treatments, and their features and potential impact on decision-making. This was used to further the debate about whether there is a need for supplemental processes for appraisal/reimbursement of rare disease treatments, and what form these could take. (3) Additional work is being done illustrate the impact of these supplemental processes in practice. This is being done through case study analyses, which aim to identify and contrast the impact of these supplemental processes across a number of countries with and without supplemental processes. This report is a H2020 project deliverable outlining what has been done and summarising key results.