A review to determine regulatorily and reimbursement successes of studies conducted using data from Canadian patient support programs based on the real-world evidence guidelines published by Canadian drug agency and health Canada.

Abstract

Introduction: Patient Support Programs (PSPs) are growing globally to support early reimbursement, disease and medication dosing management. In Canada, the lack of public health support has promoted the rapid expansion of company-supported disease-specific or drug-product-specific PSPs. Data collected from these programs generate unique Canadian data serving as a valuable source of real-world data (RWD), generally adopted in EU and US as a source of evidence generation. This review evaluates the suitability of PSP data for regulatory or reimbursement submissions, based on recently published Real World Evidence guidelines by the Canadian Drug Agency (CDA-AMC).

Methods: Peer-reviewed publications evaluating patients with chronic diseases enrolled in a PSP from 1 January 2020, to 31 March 2025, were selected for review. The checklist in the CDA-AMC RWE Guideline was used to measure the quality and suitability of the PSP data.

Results: Nine studies were reviewed against the checklist. Based on the criteria required to inform decision-making, most studies failed to meet key criteria for regulatory submissions. One recently published study, "Therapeutic Drug Monitoring of Infliximab" met most regulatory and reimbursement submission requirements.

Conclusion: Data quality validation, data source transparency, validated methodology to manage study bias, measured or unmeasured confounders, and robust outcome analysis, including sensitivity and quantitative bias analysis, are essential to ensure PSP data analysis results in successful decision-making.