OBJECTIVES: The healthcare industry is increasingly negotiating outcome-based agreements (OBAs) to provide real-world evidence and accelerate access to innovative therapies with high clinical uncertainty. This study aimed to compare the OBAs implementation between the US and Spain, a selected example of an European country with a recently established database to collect OBAs.

METHODS: Different sources including PubMed, Spanish databases (VALTERMED and BIFIMED), ISPOR database, US commercial insurer websites, and US and Spanish medical press websites were searched to identify information on OBAs implemented in the last five years. Data describing the main features and technical aspects of the OBAs were extracted.

RESULTS: A total of 51 (US) and 25 (Spain) OBAs were identified. In the US, 39 OBAs were negotiated with commercial payers and 10 with public payers. In Spain, 15, 8, and 2 OBAs were negotiated with national, regional, and local payers, respectively. Pay-for-performance constituted most contracts in both the US (82%) and Spain (96%). Conditional coverage requiring evidence development was rarely implemented in Spain (4%) and in the US (8%). Only 12% OBAs in the US involved oncology indications versus 72% in Spain. Drivers for OBA implementation for oncology therapies involved their associated high budget impact in Spain. Most OBAs in the US involved chronic non-oncology diseases. In both countries, OBA required outcome measures corresponded to primary or secondary endpoints in the pivotal clinical trial aiming to substantiate clinical value (e.g., clinical response, avoiding disease exacerbations).

CONCLUSIONS: While Spain developed a national database to track OBAs, the main source of information in US remains press release. Increased transparency in the US could improve knowledge sharing and OBAs facilitation. Understanding the outcome measures, administrative considerations and facilitating data collection would help manufacturers to better prepare for and benefit from OBAs implementation.