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Article History

Received: 3 July 2024

Accepted: 17 January 2025

First Online: 13 February 2025

Declarations

:

: “The Research Ethics Committee UZ/KU Leuven hereby grants favorable advice to the proposed study, as it was described in the protocol. The Commission is of the opinion that from an ethical standpoint there are no objections to the proposed study. The study was approved on 04-10-2022.” Moreover, this approval certifies that the study was performed in accordance with the ICH-GCP principles (International Conference on Harmonization Guidelines on Good Clinical Practice), the latest version of the Declaration of Helsinki, the Oviedo Convention on Human Rights and Biomedicine and applicable laws and regulations. Committee’s reference number: MP021468. Informed consents to participate in this study were obtained from all participants.

: Informed consents to publish study findings were obtained from all participants.

: Khadidja Abdallah (KA) does not report any conflict of interest. Steven Simoens (SS) has previously conducted research about market access of orphan drugs sponsored by the Belgian Health Care Knowledge Centre and by Genzyme (now Sanofi), and he has participated in an orphan drug roundtable sponsored by Celgene. SS is a member of the ISPOR Rare Disease Special Interest Group’s Challenges in Research and Health Technology Assessment of Rare Disease Technologies Working Group, the International Working Group on Orphan Drugs, and the Innoval Working Group on Ultra-Rare Disorders. Isabelle Huys (IH) is currently partly seconded to the cabinet of Deputy Prime Minister and Minister for Social Affairs and Public Health of Belgium. She has worked in collaboration with RIZIV/INAMI on the reformation of the Drug Reimbursement procedures in Belgium. Kathleen J. Claes (KC) has received consultancy fees from Alexion, Astellas, Astra Zeneca and Sanofi.