Abstract: FR-PO0279
Evaluating Physician Preferences for siRNA Therapy in Patients with Primary Hyperoxaluria Type 1
Session Information
- Bone and Mineral Metabolism: Clinical Epidemiology and Outcomes
November 07, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Bone and Mineral Metabolism
- 502 Bone and Mineral Metabolism: Clinical
Authors
- Gutierrez, Lissette, Novo Nordisk, Plainsboro, New Jersey, United States
- Chen, Jing Voon, Novo Nordisk, Plainsboro, New Jersey, United States
- Ambegaonkar, Ambarish, APPERTURE LLC, Marlboro, New York, United States
- Mahadik, Bhargavi, APPERTURE LLC, Marlboro, New York, United States
- Yadav, Nishi, APPERTURE LLC, Marlboro, New York, United States
- Salem, Sandra, Novo Nordisk, Plainsboro, New Jersey, United States
Background
Primary hyperoxaluria type 1 (PH1) is a rare genetic metabolic liver disorder causing excess oxalate. The small interfering RNA (siRNA) therapies lumasiran and nedosiran are effective treatment options. This study evaluates physicians’ preferences for siRNA treatment administration attributes in managing patients with PH1.
Methods
Interviews conducted with 17 physicians, who treated at least one patient with PH1 in the past year, collected data on physician and patient characteristics, current PH1 treatment approaches, and siRNA treatment attribute preferences.
Results
With nedosiran not commercially available at the time of the study, lumasiran was prescribed to 41 (48%) patients by 11 (64%) physicians. In general, physicians considered high treatment costs, insurance coverage, and patient affordability as primary challenges in treating PH1. They also reported that current treatments were complex to prepare and administer. When considering treatment administration attributes, the order of physician preference for siRNA treatment was: patient-related factors, dosing/regimen complexity, site of care, and treatment administration, assuming efficacy, safety, and cost were constant (Figure 1). In two case studies involving an adolescent and an adult patient with PH1, physicians showed preference for an siRNA treatment with a simple regimen, easy preparation, and rapid administration. At-home self-administration, and minimal involvement of HCP/staff were also preferred.
Conclusion
Physicians prioritized patient-related factors and regimen complexity when selecting a PH1 treatment. They preferred an siRNA treatment that was easy to use, required minimal HCP involvement/allowed for self-administration and did not negatively impact the patient's daily activities, including school or work.
Funding
- Commercial Support – Novo Nordisk A/S