Conference Coverage

How Do Physicians Prioritize Treatment Attributes When Selecting siRNA Therapy for Primary Hyperoxaluria Type 1?

Anthony Calabro, MA

Key Highlights

  • Physicians prioritized patient-related factors and dosing simplicity when evaluating siRNA treatments for primary hyperoxaluria type 1.
  • Ease of use, self-administration, and minimal healthcare staff involvement were preferred treatment features.
  • Cost, insurance coverage, and affordability were cited as the primary treatment challenges.
  • At-home administration and rapid dosing were viewed favorably across both adolescent and adult case scenarios.

In a recent qualitative study presented at the American Society of Nephrology (ASN) Kidney Week 2025, investigators explored how physicians select small interfering RNA (siRNA) therapies for patients with primary hyperoxaluria type 1 (PH1). The findings highlight the growing importance of treatment convenience, simplicity, and patient autonomy in clinical decision-making for rare metabolic disorders.

Primary hyperoxaluria type 1 is a rare genetic liver disorder characterized by oxalate overproduction, leading to progressive kidney injury and systemic oxalosis. Two siRNA-based therapies—lumasiran and nedosiran—have emerged as effective treatment options targeting hepatic enzymes responsible for oxalate synthesis. Given their efficacy and safety profiles, understanding how physicians differentiate between these therapies based on administration characteristics offers valuable insight into real-world clinical preferences.

Researchers conducted structured interviews with 17 physicians who had treated at least one patient with PH1 in the preceding year. Participants provided information on practice characteristics, patient demographics, current management strategies, and perceived challenges in PH1 care. The interviews also explored preferences for treatment administration attributes under the assumption that efficacy, safety, and cost were equivalent across options.

At the time of data collection, nedosiran was not yet commercially available, while lumasiran was prescribed to 41 patients (48%) by 11 of the participating physicians (64%). The most cited challenges to PH1 treatment were high drug costs, limited insurance coverage, and patient affordability barriers. Physicians also reported that existing therapies were complex to prepare and administer, requiring specialized handling and coordination between providers and patients.

When ranking key attributes of siRNA therapy, physicians prioritized treatment features in the following order: (1) patient-related factors, (2) dosing and regimen complexity, (3) site of care, and (4) treatment administration logistics. Across two illustrative case scenarios—one involving an adolescent and the other an adult with PH1—respondents consistently favored therapies that were easy to prepare, required minimal clinic visits, and allowed self-administration. Treatments with rapid administration and minimal disruption to daily life were strongly preferred, particularly those that could be managed at home without direct healthcare professional involvement.

Although the study involved a small sample size, limiting generalizability, it provides important qualitative insight into evolving physician priorities for PH1 therapy. Broader quantitative research may further elucidate how these preferences translate into prescribing behavior as new siRNA agents become available.

“Physicians prioritized patient-related factors and regimen complexity when selecting a PH1 treatment, preferring an siRNA therapy that is simple, easy to use, and minimally disruptive to patients’ daily lives,” the authors concluded.

Reference
Gutierrez L, Chen JV, Ambegaonkar A, Mahadik B, Yadav N, Salem S. Evaluating physician preferences for siRNA therapy in patients with primary hyperoxaluria type 1. Presented at: American Society of Nephrology Kidney Week; November 5–9, 2025; Houston, Texas.