ArticleNephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association2025
Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands.
Article in Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed.
- Medicine Development and Access for Rare Diseases: Can We Do Better?Journal of inherited metabolic disease · 2026Review
- Targeting Oxalate Production by Combining Enzyme Inhibition and Proteolysis Activation: A Novel Therapeutic Approach for Primary Hyperoxaluria Type 1.Journal of medicinal chemistry · 2026Article
- The Dawn of Precision Medicine in Pediatric Nephrology: Lumasiran and the Era of siRNA Therapies for Primary Hyperoxaluria Type 1.Journal of personalized medicine · 2026Review
- Cystine-Binding Medication Is Poorly Accessible for the Treatment of Cystinuria.Kidney & blood pressure research · 2026Article
- Coverage with evidence development for medicines with insufficient evidence of clinical benefit: experience from the Netherlands.International journal of technology assessment in health care · 2025Article
- Two decades of nephrology research: progress and future challenges.Nature reviews. Nephrology · 2025Article
- Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes.Healthcare (Basel, Switzerland) · 2025Article
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10 authors.
Funding
Abstract
background and hypothesisIn search for controlled access to expensive innovative orphan drugs, a national access route called 'Orphan Drug Access Protocol' (ODAP) was developed and piloted with lumasiran, a new drug for patients with primary hyperoxaluria type 1 (PH1). Here, we present a 2-year evaluation of this pilot study.
methodsSpecialists from the Dutch PH1 Expert Centre and the national ODAP steering group developed a protocol for controlled and conditional treatment of children and adults with PH1 with lumasiran. Indication for treatment is based on specific clinical characteristics. Cessation or continuation of therapy is evaluated every 6 months for 5 years by a national indication committee consisting of PH1 specialists, based on biochemical and clinical response. Drug wastage is minimized by centralizing and pooling patients for administration.
resultsBetween September 2022 and September 2024, 21 PH1 patients were reviewed and 76% were deemed eligible for lumasiran treatment. Ten patients were already receiving lumasiran through clinical trials or early access programs at time of assessment. The follow-up time with lumasiran was 0.1-6.6 years, including trial years. All patients with >1 year lumasiran treatment responded significantly biochemically and clinically. Details on outcomes are presented. Denials for lumasiran therapy were nearly all based on full pyridoxine responsiveness. All denied patients, except one, had good clinical outcomes. This patient received lumasiran after initial denial based on clinical and biochemical course. Patient selection and minimizing wastage saved approximately €3 227 065 per year based on the official list price.
conclusionsThis national ODAP protocol enabled access to lumasiran therapy for severely ill patients, prevented unnecessary treatment in others, and provided new insights into the real-world effectiveness of lumasiran in PH1 patients through systematic monitoring. It may serve as a template for future access routes to new expensive therapeutics in orphan diseases.
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