OBJECTIVES: Outcomes-based payment arrangements (OBA) for cancer drugs are used internationally with more limited use in the U.S. These arrangements seek to increase the realized value of pharmaceuticals and decrease value uncertainty for payers. Our objective was to identify and characterize OBAs involving oncology products.

METHODS: OBAs were defined as arrangements between a payer and a pharmaceutical developer where the price or reimbursement level is related to its performance in clinical practice. We used a systematic search, leveraging the University of Washington’s Performance Based Risk Sharing Database that contains information obtained by searching Google, PubMed, and government websites from 1993 to 2018. Included arrangements had a financial or health outcomes related component linked to reimbursement. We collected data on product name, year, country, tumor type, payer, arrangement structure, and outcome measure. We summarized the findings using descriptive statistics.

RESULTS: The search yielded 117 oncology-related arrangements for 55 drugs in 12 countries. The majority were from Italy (56%) followed by the United Kingdom and Australia. The U.S. had 3 cases. The most common tumor types were lung (13%), breast (11%), and kidney (9%). The distribution of outcome measures used was clinical (44%), financial/utilization (41%), cost-effectiveness (3%), hybrid (5%) and unknown (16%). 60% were first-in-therapeutic-class molecules in the US and 27% had an orphan designation. The primary clinical outcome measures included tumor response, disease progression, and toxicity. The primary financial/utilization measures included the number of treatment cycles and treatment duration. Arrangement structures primarily used rebates (41%), manufacturer funded treatment initiation (40%), and refunds (24%). 17 drugs in 19 tumor types had OBAs in multiple countries.

CONCLUSIONS: Outcomes-based oncology arrangements are used in many countries. Arrangements have employed a variety of endpoints, outcomes measures and contract structures. U.S. stakeholders can learn from global experiences to inform further implementation and research moving forward.