Gene Therapy Preparedness – It’s Time to Get Educated

With the advent of gene therapy, patients will need to educate themselves more about eligibility, risks, compliance and costs. And they should educate themselves in conjunction with their hemophilia treatment center (HTC). HTC staff also need to be prepared to help patients make this important decision. A new National Bleeding Disorder Foundation (NBDF) MASAC document, #282, discusses what is needed for a team decision about gene therapy. The document stresses that multi-stakeholder preparation is required to develop clinical protocols for eligibility screening, administration, and follow-up of gene therapy, and to identify ways to remove barriers to access and achieve excellence in clinical delivery to maximize health outcomes. Read here

Source: LA Kelley Communications, Inc. Communique, December 2023