Description
This study was carried out to provide practical guidance for professionals and health services worldwide on vosoritide use in the treatment of achondroplasia. A total of 16 leading experts and patient advocates representing nine countries convened to form a guideline development group. This group developed statements to provide recommendations across the treatment pathway, from treatment initiation, ongoing monitoring and evaluation, to stopping vosoritide treatment and the continued follow up after cessation. These guidelines recommend a minimum set of requirements and a practical framework for professionals and health services worldwide.
Savarirayan et al. developed the guideline scope and topics at a hybrid meeting in November 2023. Prior to the meeting, a literature search was carried out to identify existing guidelines and to inform the scope. An anonymous, virtual Delphi voting process was carried out. Consensus was defined when >80% of the group either agreed/strongly agreed or disagreed/strongly disagreed. A total of 62 statements were proposed, with 56 reaching a positive consensus following Round 1 voting. Statements that did not reach consensus were reviewed and amended, and three new statements proposed. There were 12 statements that reached consensus at Round 2 voting. A total of 64 guidance statements reached consensus.
