MASAC Document 288 - MASAC Resolution on Proposed Hemophilia Treatment Guidelines from the International Society on Thrombosis and Haemostasis (ISTH)

Treatment guidelines play an important role in managing patients with chronic diseases, including hemophilia. Treatment guidelines offer guidance on treatment selection and patient choices, taking into account outcomes, and balance both efficacy and risk in achieving optimal outcomes.

• Whereas recombinant DNA produced products are well established as the most viral safe treatment for hemophilia, with essentially zero risk of infection transmission;
• Whereas treatment products developed over the past 10 years have enabled protective trough levels of clotting factors to aim for a goal of zero bleeds for all, and risk for inhibitor development with these products were not available for inclusion in the SIPPET trial;
• Whereas the treatments used prior to 10 years ago have resulted in worse bleeding sequelae and disability that are not comparable to health outcomes compared using products available today;
• Whereas newer products have enabled improved patient-centered outcomes, including increased daily living participation, economic activity, and quality of life;
• Whereas treatment of hemophilia A inhibitor patients has undergone revolutionary change in the last 6 years;
• Whereas health inequity remains a challenge and guidelines that limit access to products can exacerbate the problem in many countries of all socioeconomic statuses;
• Whereas draft guidelines produced by the International Society on Thrombosis and Haemostasis (ISTH) conclude many treatments are supported by weak to very weak evidence and have low to very low certainty of efficacy. ISTH did not allow for revision or substantive changes to the guideline recommendations during the open comment period despite overwhelming concern from the international hemophilia community;
• Whereas the methodology used by the ISTH, or the interpretation of said methodology may be inappropriate when used for rare disease treatments that often produce limited randomized control trial evidence for new treatments;

The draft ISTH guidelines for hemophilia treatment do not support the changed treatment paradigm in hemophilia (Skinner et al). They are counterproductive toward reaching the goal of zero bleeds, and work against the goals of shared decision making and improved patient-centered outcomes. If Ministries of Health follow the draft ISTH guidelines, newer products that achieve better outcomes may be restricted and less available to patients. Consequences for patients include worse outcomes, including more morbidity and mortality without these newer products.

Since the draft guidelines state the evidence of efficacy for many products is insufficient, the ISTH should address the consequences of their planned publication of the guidelines. They should provide context and develop mitigation strategies to prevent harm to patients worldwide by cost-conscious governments, payors, and insurance companies that may use these guidelines to block improved treatments for patients with hemophilia.

References

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