Slowdown of Gene Therapy Research Spurs Call to Action

Global organizations unite to urge collaborative approaches to reducing barriers to advancement of care.

The National Bleeding Disorders Foundation (NBDF) has joined with three international bleeding disorders organizations in their call to action to continue research and development into gene therapy as a treatment option for hemophilia.

The joint statement from the International Society on Thrombosis and Haemostasis (ISTH), the European Association for Haemophilia and Allied Disorders (EAHAD), and the World Federation of Hemophilia (WFH) addresses the elimination or scaling back of gene therapy development programs from pharmaceutical companies, citing limited interest from the bleeding disorders community. 

“The recent curtailment of gene therapy programs sends a troubling message to the global bleeding disorders community and risks undermining decades of progress,” said Phil Gattone, M.Ed., president and CEO of NBDF.

The joint statement pushes back against the idea that the bleeding disorders community, including affected individuals and physicians, has “low interest” in gene therapy as a treatment option. Rather, it is the “delayed development of appropriate reimbursement models,” as well as disparate global regulatory systems that create barriers to innovation.

The past decade of research into bleeding disorders has led to novel therapies that have changed the landscape of bleeding disorders treatment. However, people with bleeding disorders globally still experience breakthrough bleeds, joint damage, and barriers to products and care. 

“It is imperative that we sustain momentum in research and development—particularly in gene therapies and other novel treatments—while addressing the systemic and commercial barriers that limit access and adoption,” Gattone continued. 

The joint statement urges all stakeholders to “intensify collaborative approaches to reduce burdens and barriers” so that safe, transformative therapies can be accessible to all people with bleeding disorders around the world.

NBDF has been joined in their support of the joint statement with other prominent global medical and scientific organizations and patient advocacy groups. 

Read the complete statement from ISTH, EAHAD, and WFH.

Source: National Bleeding Disorders Foundation