Steven W. Pipe, MD: Supporting Gene Therapy Implementation for Hemophilia

As gene therapies grow in prominence in the hemophilia field, discussions are needed around the practical implementation of gene therapies, whether concerning the differences in gene therapies for hemophilia A and hemophilia B, accurately identifying eligible candidates for gene therapy, monitoring patients with hemophilia who receive gene therapies, and counseling patients and colleagues about gene therapies.

One such discussion was held at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024, in San Diego, California. Participants included Steven W. Pipe, MD, Professor and Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology, University of Michigan and CS Mott Children’s Hospital, Ann Arbor, Michigan; Doris V. Quon, MD, PhD, Associate Medical Director at Sarcoma Oncology Center; and Amy L. Dunn, MD, Director of Pediatric Hematology, Hemophilia Treatment Center, Nationwide Children's Hospital.

“I was surprised, even in my own clinical setting, that some of the institutional hurdles we had to go through are different than what we had to set up the clinical trial program. And so, I believe a lot of centers are now just sort of working through those processes,” Pipe told HCPLive® during the meeting.

HCPLive spoke with Pipe to learn more about the discussion and its main takeaways. He emphasized the importance of helping to guide patients and colleagues through determining eligibility for gene therapy and achieving clinical readiness for administering gene therapy. He noted that payers are beginning to understand the cost-effectiveness of gene therapy and thus, he thinks that education and hesitancy for clinicians and patients have overcome cost as the primary deterrent for gene therapy.

REFERENCE

Pipe SW, Quon DV, Dunn A. Clinical assessment of gene therapies for hemophilia A and hemophilia B: Key takeaways for your practice. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Satellite symposium.