Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands.

ABSTRACT:

Background and hypothesis

In 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.

Methods

Specialists 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 respons

SUBMITTER: Deesker LJ 

PROVIDER: S-EPMC12477465 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature