Alnylam announces new framework for VBAs to help PH1 patients get Oxlumo

Alnylam announced today a new framework for value-based agreements, or VBAs, designed to help people with primary hyperoxaluria type 1, or PH1, gain access to Oxlumo. Now approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients, Oxlumo is the first-ever approved targeted therapeutic that substantially curbs oxalate production in patients living with PH1, an ultra-rare genetic disease characterized by oxalate overproduction. Alnylam is in active discussions with payers and has reached an agreement in principle with Express Scripts, Harvard Pilgrim, and Highmark to pursue VBAs for Oxlumo. The company has worked with payers on a new and enhanced VBA framework. Since Oxlumo is indicated for both pediatric and adult patients, and is dosed by weight, related costs can vary relative to each patient and use over time. As such, Alnylam has structured a new VBA component that specifically addresses many payers' concerns for budget predictability and value, particularly for ultra-rare orphan disease therapies that are administered across a wide spectrum of ages from infants to adults. The new VBA component, called a patient need adjustment, or PNA, is now being added to Alnylam's overall VBA offering for Oxlumo. Participating payers will qualify for the PNA rebate if the average number of vials utilized by a plan member exceeds an established threshold, providing payers with greater short-term and long-term predictability. The PNA was designed to mitigate the risk of escalating or varying costs associated with dosing requirements, thereby accelerating access for people diagnosed with PH1. To further address budget predictability, Alnylam is also making available its prevalence based adjustment, or PBA, component, which was first introduced last year for Alnylam's second-approved therapy, Givlaari. There are often uncertainties in diagnosis rates and disease prevalence estimates in ultra-rare, poorly diagnosed orphan diseases, making it challenging for payers to predict the number of patients who will be covered within their plans. This feature triggers a rebate to participating payers if the number of diagnosed patients they cover exceeds current epidemiologic estimates for PH1.