Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries.

ABSTRACT:

Objectives

The aim of the study was to assess the reimbursement policy for orphan drugs (ODs) in selected European countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedures and reimbursement decision-making.

Materials and methods

A list of authorized ODs was extracted from a web-based registry of the European Medicines Agency, including information on active substance, Anatomical Therapeutic Chemical (ATC) classification code, and therapeutic area. A country-based questionnaire survey was conducted between September 2022 and September 2023 among selected experts from 12 European countries. A descriptive and statistical analysis was performed to identify correlations between country characteristic, HTA procedures,

SUBMITTER: Jakubowski S 

PROVIDER: S-EPMC11611580 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature