Financial Characteristics of Outcomes-Based Agreements: What Do Canadian Public Payers and Pharmaceutical Manufacturers Prefer?

OBJECTIVES: This study sought to gain insight into the financial characteristics of outcomes-based agreements (OBAs) considered most suitable to Canadian public payers and pharmaceutical manufacturers, and the rationale for their preferences. METHODS: A total of 17 public payers and pharmaceutical manufacturers participated in semistructured qualitative interviews, which assessed their knowledge of OBAs and their preferred financial characteristics. RESULTS: Payers identified 5 OBA financial models that they considered both acceptable and feasible, in no preferential order: (1) discontinuation of therapy, (2) rebates for nonresponders, (3) free trial period, (4) adjustable pricing, and (5) blended rebate. Payers had a clear preference for short-term OBAs (<1 year), whereas both payers and manufacturers agreed OBAs with longer durations (up to 5 years) would be manageable if appropriately designed. Six key success factors to design suitable and acceptable OBA financial models were identified, including the areas of interim reporting, easily measurable health outcomes, trusted data sources, engaging unbiased third-party data experts, harmonizing OBA billing methods, and the inclusion of budget caps. CONCLUSIONS: Manufacturers and payers showed high level of interest in OBAs and a robust understanding of their potential role in supporting timely market access for patients in need, with the caveat that they need to be carefully designed to provide value. Further opportunities for discussion and engagement between public payers and manufacturers are needed to establish how to implement OBAs at a pan-Canadian level and how individual provinces and territories can incorporate them within their existing governance infrastructures.

Data collection within patient support programs in Canada and implications for real-world evidence generation: the authors' perspective.

Patient support programs (PSPs) offer a unique opportunity to collect real-world data that can contribute to improving patient care and informing healthcare decision making. In this perspective article, we explore the collection of data through PSPs in Canada, current advances in data collection methods, and the potential for generating acceptable real-world evidence (RWE). With PSP infrastructure already in place for most specialized drugs in Canada, adding and strengthening data collection capacities has been a focus in recent years. However, limitations in PSP data, including challenges related to quality, bias, and trust, need to be acknowledged and addressed. Forward-thinking PSP developers have been taking steps to strengthen the PSP datasphere, such as engaging third parties for data analysis, publishing peer-reviewed studies that utilize PSPs as a data source and incorporating quality controls into data collection processes. This article illustrates the current state of PSP data collection by examining six PSP RWE studies and outlining their data characteristics and the health outcomes collected from the PSP. A framework for collecting real-world data within a PSP and a checklist to address issues of trust and bias in PSP data collection is also provided. Collaboration between drug manufacturers, PSP vendors, and data specialists will be crucial in elevating PSP data to a level acceptable to healthcare decision makers, including health technology assessors and payers, with the ultimate beneficiary being patients.

Building infrastructure for outcomes-based agreements in Canada: can administrative health data be used to support an outcomes-based agreement in oncology?

BACKGROUND: Outcomes-based agreements (OBAs) have the potential to provide more timely patient access to novel therapies, although they are not suitable for every new medication or reimbursement scenario. The authors of this paper studied how to operationalize an OBA in oncology by leveraging existing real-world data (RWD) infrastructure in the province of Alberta. OBJECTIVE: The main objectives were to (1) evaluate which health outcomes in oncology are suitable for OBAs and whether they can be tracked with existing infrastructure, and (2) determine how RWD in oncology can be used to implement an OBA and the expected timing for delivery. METHODS: Using the Oncology Outcomes (O2) Group infrastructure and Alberta administrative data, a review of five key oncology outcomes was performed to determine suitability to support an OBA. RESULTS: Overall survival and time-to-next-treatment were determined as potentially suitable oncology outcomes for OBAs; progression-free survival, patient-reported outcomes, and return to work were deemed inadequate for OBAs at the current time due to data limitations. CONCLUSIONS: Results indicate that it is feasible to leverage RWD to support OBAs in oncology in Alberta, with minimal additional data, resources, and infrastructure. The operational processes and steps to collect and analyze RWD for OBAs were identified, starting with performing an RWD feasibility study. The expected timeframe to fulfill the real-world evidence (RWE) requirements for an OBA is approximately 3 years for cancers with short trajectories.