NEWARK, N.J. (PRWEB)
July 25, 2022

The latest results from APLUSA’s annual syndicated market surveillance tracker in hemophilia A and B, HEMOPHILIAsyndiTrack™, published in June, show that while unaided awareness of gene therapy for the treatment of both hemophilia A and B is high among physicians – 55% vs. 32% for antithrombin RNAi (RNAi) therapy and 30% for anti-tissue factor pathway inhibitor (TFPI) therapy – the unaided awareness of the specific gene therapies is very low (<5%), while at the same time, 29% of physicians mention Fitusiran (RNAi therapy) and 21% mention Concizumab (TFPI). Despite the generally high awareness of gene therapies, physicians are nearly equally likely to consider prescribing gene therapy within the first year after launch (56%) as they are for RNAi (51%) and TFPI (49%) therapies. Further, the prescribing decision is highly likely to be patient-directed, with physicians basing treatment on the patient’s satisfaction with current therapy.

According to HEMOPHILIAsyndiTrack™ study findings, which captures real-world patient chart data as provided by treating physicians, Hemlibra has been growing share steadily since 2019. At that time, Hemlibra was prescribed in 2% of the study’s patients with Hemophilia A – the most prevalent form of Hemophilia – and share has since grown to 22% in the most recent wave of the study. When asked about future intent, 19% of Hemophilia A treaters anticipate that they will increase their use of Hemlibra significantly, and 41% said they would increase use slightly.

The future success of gene therapies among Hemophilia A patients is further clouded by the heavy overlap in target patients, as Hemlibra and gene therapies both target younger, more physically active and severe patients. Additionally, Hemlibra patients have reported a generally positive experience on the product. APLUSA’s research shows that patients taking Hemlibra experience the lowest number annually of bleeds that require additional on-demand injections – even though Hemlibra patients are the most physically active and severe cases of Hemophilia A. This protection, combined with the reduced burden of treatment in terms of frequency and mode of injections, results in overall improvements in patient quality of life. In this context the anticipated benefits of gene therapy are not that compelling, especially given the questions related to long term side effects and efficacy of gene therapy.

“Something about gene therapy just makes me uneasy,” says one Hemophilia A patient diagnosed with severe disease, “even if I have to go through a year of immune suppressants and side effects and it does work, I’d still rather keep doing what I’m doing. There are too many unknowns for me to feel comfortable with it.”

Additional ‘voice of the patient’ social media studies conducted by APLUSA uncovered additional hesitancy and doubt among both Hemophilia A and B patients specifically regarding gene therapies. Patients with severe disease expressed concerns about the number of unknowns with gene therapies; belief that gene therapy would not ultimately result in much improvement in their quality of life; and problems with the adeno-associated virus (AAV) vector-based gene therapies – where people previously exposed to adenoviruses often had an immune mediated response resulting in anti-adenovirus antibodies, and therefore may not be good candidates for gene therapy.

According to Dr. Karen Higginbottom, APLUSA’s resident hematology expert, “These market factors – particularly the continued growth and potential entrenchment of Hemlibra – will create an uphill battle for gene therapies in the Hemophilia market.”

About APLUSA

APLUSA (with US affiliate APLUSA Bell Falla) is one of the world’s leading independent market research companies dedicated to healthcare. Thanks to the excellence and commitment of our teams, our challenge for globalization has been well and truly met. More than half of our turnover is generated overseas, two-thirds of our services are international (conducted in 75 countries), and our teams are a true reflection of our business: multi-nationals. We are now, globally, among the Top 10 Global Healthcare Market Research Companies, and the Top 3 Independent Players. The acquisition of IFOP and Sociovision and their inclusion into the APLUSA-IFOP group have increased our capabilities to develop innovative solutions based on advanced techniques used outside the health industry.

For more information on APLUSA, please visit us at https://www.aplusaresearch.com.

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