Mussert, Caroline MA;
Miesbach, Wolfgang;
Chowdary, Pratima;
Lillicrap, David;
Mahlangu, Johnny;
Peyvandi, Flora;
Pipe, Steven W;
... Leebeek, Frank WG; + view all
(2025)
A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and Summary of Product Characteristics (SMPCs); Communication from the ISTH SSC Working Group on Gene Therapy.
Journal of Thrombosis and Haemostasis
10.1016/j.jtha.2025.09.041.
(In press).
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Abstract
BACKGROUND: Gene therapy for hemophilia has recently been implemented as standard clinical care, requiring organizational and multi-stakeholder preparedness and clear guidelines. In addition to pharmaceutical Summaries of Product Characteristics (SMPCs), various (inter)national guidance documents have been published. However, no guidance document or SMPC covers the entire gene therapy care pathway. STUDY OBJECTIVES: To provide a complete and comprehensive overview of current guidance documents and SMPCs to develop a comprehensive care pathway for hemophilia gene therapy delivery. METHODS: Published gene therapy guidance documents and collected SMPCs were complemented by a selective search in online databases, including Pubmed and scientific societies' websites. Reference lists were checked for additional relevant articles. RESULTS: Four SMPCs and eleven (inter)national guidance documents and recommendations were collected. The documents were focused on either the intervention or the care pathway, and none were comprehensive covering all aspects of hemophilia gene therapy delivery. Considerable differences were found between the two approved gene therapy products and between the SMPCs issued by the two regulatory authorities, the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). (Inter)national guidance documents provided additional information and recommendations not covered in SMPCs. CONCLUSION: Based on SMPCs and (inter) national guidance documents and recommendations a care pathway has been developed and visualized in a Metro Map. This provides a clear and comprehensive overview of all activities, contact moments and responsibilities within the longitudinal gene therapy treatment process. This comprehensive care pathway may help navigate gene therapy implementation, providing guidance to clinicians, patients and caregivers.
| Type: | Article |
|---|---|
| Title: | A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and Summary of Product Characteristics (SMPCs); Communication from the ISTH SSC Working Group on Gene Therapy |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.jtha.2025.09.041 |
| Publisher version: | https://doi.org/10.1016/j.jtha.2025.09.041 |
| Language: | English |
| Additional information: | Under a Creative Commons license https://creativecommons.org/licenses/by/4.0/ |
| Keywords: | Hemophilia, care pathway, gene therapy, guideline, implementation |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10217046 |
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