rare (orphan) diseases
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Horizon Scanning and Early Assessment of Health Technologies for the Treatment of Orphan Diseases
Abstract: The theoretical foundations and regulatory framework of the processes of the formation of an effective health technology assessment (HTA) system at the early stages of the life cycle of medicines are analyzed in the article. Particular attention is paid to horizon scanning (HS) and early assessment of expensive innovative drugs utilized for treating rare diseases. In order to inform policymakers, purchasers, and providers (to prioritize MT research, financial, and operational …
Published in International Journal of Brain Sciences · Vol. 1, Issue 1, 2024 · pp. 32–39 Read article