Value-Based Healthcare in Practice: IDEATE, a Collaboration to Design and Test an Outcomes-Based Agreement for a Medicine in Wales.

Objective: To develop a sustainable, scalable methodology for the design of outcome-based agreements (OBAs) that works on the ground and dynamically overcomes historical challenges.

Methods: Project IDEATE co-created solutions to known (and emergent) challenges via iterative workshops and real-world data analysis to develop and refine a hypothetical model for an OBA in a trusted research environment. A cross-disciplinary collaboration between National Health Service (NHS) Wales, industry and academia was developed. Data were collected from Welsh national datasets and used to construct a novel linked dataset. OBA scenarios, with different contract parameters, were analysed to assess impact on the proportion of contract payment due and the volatility of payments.

Results: An approved, in market, locally advanced and metastatic breast cancer treatment was selected as the test case. The total number of patients in the treatment cohort (2017-2020) was n = 99, and 286 in the control cohort (2014-2016). The final outcome variables selected were: (1) 1-year survival,( 2) intolerance to treatment (deferral), and (3) the total days disrupted by care. The primary scenario included all three outcomes measured at the population level and used a linear payment model. Volatility analyses demonstrated contract parameters can dramatically alter the contract output with greatest risk from a single, binary outcome contract design.

Conclusions: The design of an OBA is a complex process that requires a multi-disciplinary approach. By assessing solutions to data, outcomes and contracting challenges, IDEATE provides a strong foundation for future success of OBAs in the UK. Outcome-based agreements (OBAs) are a way to pay for medicines if they help patient health in a specific way over time. These agreements can make it faster for people to get new medicines, but they also have challenges, like needing a lot of time and effort to manage them. A team from the NHS Wales, life sciences, and Swansea University created Project IDEATE to find a better way to design OBAs and solve some of these problems. Welsh datasets were used to create a new breast cancer dataset to test different OBAs and see how payments would change. A breast cancer treatment was used for the project. The project had 99 patients who got the medicine (2017-2020) and 286 patients who had breast cancer but did not get the medicine (2014-2016). Three health outcomes were measured: (1) living for one year after treatment, (2) patients needing to stop the medicine, and (3) days spent in care. The main OBA option we tested used all three health outcomes; the more the outcomes improved, the more the payments could go up until they hit the highest amount agreed. The analysis showed that the way an OBA is designed can make a big difference in how stable or risky it is, especially if one of the health outcomes has only two options. Project IDEATE showed that making an OBA can be hard, but when people from different fields work together, they can overcome many challenges and succeed.