Disease burden and unmet medical need in severe hemophilia in Greece: insights from clinicians and patients.

Objectives: Hemophilia is the most common severe hereditary bleeding disorder, with significant unmet needs in its management. This study aimed to assess the burden and unmet needs of severe hemophilia in Greece from a clinician and patient perspective. Methods: Two independent Delphi panels were conducted: one comprising six clinical experts from all specialized hemophilia centers across Greece, and another including 15 patients-members of the Greek Hemophilia Society with severe hemophilia. Following a targeted literature review, two structured Delphi questionnaires were developed with evidence-based statements addressing the clinical, humanistic, and economic burden of severe hemophilia, as well as unmet medical needs, treatment preferences and expectations. Delphi panelists rated the statements over up to three rounds using a five-point Likert scale (from "strongly disagree" to "strongly agree") to capture consensus and real-world perspectives. Consensus and its strength were established based on predefined criteria. Two focus group meetings, one with clinicians and one with patients, followed to discuss the Delphi findings in greater depth, validate areas of agreement or divergence, and collect qualitative insights. Results: Consensus was achieved on 97.3 and 97.2% of statements among clinicians and patients, respectively. The two groups showed largely concordant views, with limited areas of divergence. Both highlighted that, despite existing prophylactic treatments, spontaneous bleeds persist, significantly affecting the patients' and caregivers' quality of life and resulting in considerable non-medical and surgery costs. Suboptimal adherence remains a challenge, with main barriers including the frequency/time commitment of factor replacement therapy, venous access challenges, and needle phobia. Both groups demonstrated the importance of safety in emerging treatments, highlighting their preference for treatments that support a more active lifestyle, while offering subcutaneous administration, superior efficacy, and greater convenience compared to the current standard of care. Conclusion: Clinical and patient insights highlight the need for improved treatments for severe hemophilia in Greece. Safer, more effective therapies are needed to address residual burden and unmet needs. These should offer simpler administration, less frequent dosing, and more convenient delivery.