OBJECTIVES: Outcomes-based agreements (OBAs) have become a powerful strategy to mitigate payer concerns on the clinical value of innovative therapies. However, their use has been subject to feasibility and implementation challenges. This study aimed to identify drivers of OBAs success in Spain that can guide future OBA negotiations with payers.

METHODS: Different data sources (e.g., PubMed, BIFIMED, VALTERMED) were searched to identify publicly available information on OBAs in Spain during the last 5 years. Identified literature was screened to extract the main features that drove OBA implementation, including OBA technical characteristics.

RESULTS: Between 2017-2022, 25 OBAs and mixed agreements were identified at the national (n=15), regional (Catalonia and Galicia, n=8), and hospital (n=2) levels. Almost all (96%) OBAs involved patient-level payment-by-results. Some (n=7) OBAs included a mixture of payment-by-results plus discount and expenditure cap, or staged payments. Most OBAs were identified for oncology indications (n=18), and some for orphan indications (n=10). The principal outcome measures included achieving or sustaining CR, achieving a response based on set criteria, or no discontinuation due to adverse events or disease progression. These measures corresponded to primary or secondary clinical trial endpoints, including endpoints demonstrating direct improvement in disease exacerbations or patient functional status, or surrogate endpoints. For oncology, OS was not identified as an outcome measure and the RECIST criteria was frequently used. Timeframes to monitor outcomes ranged from 3 to 18 months, depending on the duration of treatment, time to response, or survival.

CONCLUSIONS: While Catalonia is the most active region implementing OBAs, national implementation is expected to increase following the establishment of VALTERMED. Payers in Spain favor outcome metrics based on clinical trial endpoints allowing for simple and unanimous assessment of responses widely used in clinical practice. Timeframes to measure outcomes should mirror follow-up periods, time to response, or time to discontinuation observed in trials.