Alternative Pricing Arrangements as an Opportunity for Broader Access to Rare Disease Treatments: Case Study for an Outcomes-Based Agreement for Nusinersen in Norway

Problem Statement: Treatments for rare diseases can pose challenges for payers relying on cost-effectiveness evaluations. Small patient populations, short follow-up time for treatment effect, and uncertainty associated with new treatment mechanisms, are some of the challenges that complicates decision making. Alternative pricing strategies such as outcomes-base agreements (OBA), have the potential to reduce some of this uncertainty by making the financial decision partially reversible and by facilitating further generation of real-world evidence.

Description: A framework for advancing OBA proposals for the Norwegian Hospital Procurement Agency was established in 2020. Based on this, an OBA to expand access for adult SMA patients to nusinersen treatment was developed and implemented in May 2023, being the first OBA of such complexity in Norway. It is a five-year agreement where motor- and respiratory functions and disease progression are measured systematically in all treated patients during this period. Outcomes are registered by treating physicians in a national registry. A pre-defined scoring system for different treatment outcomes determines on a group level whether price will change at the end of the agreement period in the following way: 1) Stable disease = No change in price, 2) Significant disease improvement = Increased price, 3) Significant deterioration = Decreased price. All outcomes are measured as group-level averages.

Lessons Learned: OBA has proved to be a viable approach to provide access to treatments associated with high decision uncertainty. Early dialogue with payers, clinicians and registry holders are crucial to secure effective execution of such agreements. A predefined submission framework for OBA and a centralized decision-making body and registry, are also central for agreements of this complexity to be successful.

Stakeholder perspective: Industry perspective