Qualification Criteria of Gene Therapy for Haemophilia-Opinion of the EAHAD Gene Therapy Working Group.

ABSTRACT:

Background

Following the approval of the first gene therapies for haemophilia, it is essential to develop an optimal infrastructure for the administration of gene therapy. This can be ensured by identifying the criteria for the definition of treatment centres (hub centres) and follow-up centres (spoke centres), as well as establishing effective cooperation between them.

Methods

The interdisciplinary members of the EAHAD Gene Therapy Working Group answered a survey to define requirements for centres participating in gene therapy care, addressing aspects such as product administration, coagulation parameter monitoring, and long-term safety surveillance.

Results

The majority support the implementation of standardised protocols. Hub centres are expected to maintain high s

SUBMITTER: Miesbach W 

PROVIDER: S-EPMC12462593 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature