Proprietary Survey Shows Physicians Growing Interest in Hemophilia Gene Therapy
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Proprietary Survey Shows Physicians Growing Interest in Hemophilia Gene Therapy

Hemophilia is a rare, X-linked recessive genetic disorder characterized by insufficient production of a protein necessary to clot. There are approximately ~20,000 hemophilia patients in the US and ~200,000 patients worldwide.

Historically treatments have focused on directly providing patients with the missing protein via regular infusions – FVIII protein for Hemophilia A patients and FIX protein for Hemophilia B patients – or by administering FVIIa protein in the event of breakthrough bleeds.

In both the US and EU, most physicians are not only aware of hemophilia gene therapies but familiar with the class and associated risks to the point of being able to make a treatment decision on potential approval. Clinicians appear increasingly receptive to novel therapies and report growing familiarity with using them. Notably, most clinicians (53% now vs. 30% in ‘20) anticipate prescribing gene therapy within the first 12 months of approval, with a median expectation of 15% of patients to be on treatment in the first year (vs. 3% in ‘20), and median 28% within the first three years (vs. 13% in ‘20).

Overall, patients generally appear to be satisfied with current therapies. However, dosing frequency and bleed control leave room for improvement and are the two most cited reasons for wanting to switch. Consistent with our 2020 survey, patients report that the decision on what product to use tends to be their own instead of relying on their doctor’s recommendation. Furthermore, patients indicate that they are generally comfortable switching products; 71% of patients reported they are somewhat comfortable to very comfortable switching.

Consistent with 2020 responses, patients remain interested in gene therapy but voiced some hesitancy on early uptake. Common concerns focus on long-term safety and potential exclusion from future gene therapies. We expect these concerns to wane with more comprehensive gene therapy experience/data.

@Tiago Fauth