NBDF Supports WFH Statement on ISTH Hemophilia Treatment Guidelines

The NBDF concurs with WFH’s assessment that the proposed guidelines 
risk setting hemophilia treatment back 30 years. 

 The National Bleeding Disorders Foundation (NBDF) echoes the concerns raised by the World Federation of Hemophilia (WFH) regarding the proposed International Society on Thrombosis and Haemostasis (ISTH) guidelines on the treatment for people with hemophilia. The NBDF shares WFH’s apprehensions regarding the potential adverse impact of these guidelines on individual access to essential treatments for all individuals with hemophilia. 

According to Michael Recht, MD, PhD, MBA, NBDF’s Chief Medical and Scientific Officer, “If published, these guidelines will have far-reaching implications, potentially limiting access, more effective treatments for individuals all over the world impacted by hemophilia. Furthermore, these recommendations contradict the previously stated goals of NBDF and many other professional medical societies and organizations advocating for those with hemophilia, including preventing all bleeding episodes, improving physical and emotional outcomes, and economic productivity for all.”

First issued in draft form in October 2023, the proposed guidelines consist of 13 recommendations concerning the treatment of individuals with hemophilia A or B. Despite receiving over 400 comments from the community, the ISTH indicated the wording of each recommendation would remain unchanged. The final guidelines are expected to be published in May 2024. 

We at the NBDF concur with WFH’s assessment that the proposed guidelines risk setting hemophilia treatment back decades. With most recommendations being deemed “conditional” due to “very low to low certainty evidence,” significant concerns arise regarding the effectiveness and safety of the proposed therapeutic recommendations. 

Of particular concern is the conclusion that various treatment options, including plasma-derived clotting factor concentrates (CFCs), recombinant CFCs, recombinant extended half-life factors (EHLs), and emicizumab, are presented as equivalent in safety and effectiveness for prophylaxis against bleeding. This overlooks scientific evidence highlighting the superiority of newer products in terms of bleeding protection and other outcomes important to those impacted by hemophilia.

The NBDF recognizes the efforts of international professional societies, advocacy organizations, and clinicians in challenging the proposed guidelines by standing in solidarity with our colleagues from across the globe to in recognizing the guidelines fail to consider crucial scientific and clinical evidence supporting product differentiation, which has been integral to recent evidence-based guidelines. 

If published, these guidelines could have far-reaching implications, potentially limiting access to newer, more effective treatments for individuals impacted by hemophilia. Furthermore, these recommendations contradict the goals of many professional medical societies for hemophilia treatment, including zero bleeds, improved physical and emotional outcomes, and economic productivity for people worldwide.

Recognizing the potential unintended consequences of the ISTH’s proposed guidelines, NBDF’s Medical and Scientific Advisory Council (MASAC) released MASAC recommendation #288, imploring ISTH to address the consequences of the guidelines as well as provide context and develop mitigation strategies to prevent harm to those impacted by hemophilia. 

According to Phil Gattone, NBDF’s President and CEO, “We stand in full support of WFH’s statement and urge ISTH to reconsider these guidelines to safeguard the well-being of the worldwide bleeding disorders community. At NBDF, we believe that only by working collaboratively and considering all perspectives can we truly ensure that all patients receive the care they deserve and need.”

The NBDF stands with WFH in calling upon the ISTH not to publish the guidelines in their current form. Instead, the NBDF urges the ISTH to collaborate with patient and physician associations to address concerns and ensure the guidelines align with the best interests of the individuals impacted by hemophilia and uphold health equity principles. 

References: 

Source: National Bleeding Disorders Foundation, May 2024

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