Novel Treatment Modalities


NBDF’s Collaborative Learning Exchange series on “Emerging Clinical Issues in Novel Treatment Modalities” was designed to educate hematologists, physician assistants, and nurse practitioners who regularly prescribe therapies for individuals living with hemophilia A and B.



With several new treatments on the horizon, patients and healthcare providers need to prepare for how each of these novel treatments has the potential to impact current therapeutic management of hemophilia. Along with gene therapy products, several additional types of emerging “non-factor” therapeutics (such as, RNA interference (RNAi) and anti-tissue factor pathway inhibitor (anti-TFPI) hold promise. While these new treatments are still in clinical development, clinician prescribers may not fully understand the science and clinical evidence underpinning these novel therapies. 

NHF, along with a few key experts in the field, developed this training series to foster opportunities for prescribers to discuss how novel non-factor therapies might influence treatment decision-making and impact future management challenges for hemophilia A or B (or in specific patient scenarios).   

The following activities were held as live virtual interactive events in 2022.


"Understanding Mechanisms of Action and Utility of Novel Treatments for Hemophilia A and B" was held live on 4/6/22.

Current and emerging novel non-factor therapies, such as FVIIIa mimetic bispecific antibodies and anti-Tissue factor pathway inhibitor (anti-TFPI) were presented. A summary of the latest clinical trial data (available at the time) was shared, as well as a discussion of the potential advantages and challenges in non-factor therapies for hemophilia A and B patients.

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“Shared Decision Making in the Age of Novel Technologies for Hemophilia” was held live on 5/5/22.

The model of “shared decision-making” was presented, along with how this approach benefits clinicians and patients in determining treatment plans and therapeutic options. A few ways clinicians could apply “SDM” into specific discussions about approved novel therapies with their patients was also identified and discussed further.

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“Managing Complex Cases with Novel Non-Factor Therapies" was held live on 6/9/22.

A brief treatment and management review for acute bleeding in patients with hemophilia A was presented by an experienced pediatric hematologist. By utilizing a challenging patient case, specific clinical concerns and considerations for novel non-factor therapy were highlighted and shared. A final discussion on the indications and recommendations (available at the time) for clinical use of an approved novel non-factor therapy (emicizumab) in hemophilia A ensued.  

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"Navigating Reimbursement for Patients on Non-Factor Therapy” was held live on 7/12/22.

Tighter treatment management for hemophilia and cost considerations specific to the coverage of novel non-factor therapies were presented. Also discussed were the importance to having appropriate documentation for reimbursement of non-factor therapies and the additional resources that could support an appeal in instances when coverage is denied.

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Please note:  Each of these events in their recorded formats are no longer available

for CME/CE credit.