Presentation (CTI)

OBJECTIVES: In Canada, patient support programs (PSPs) help patients access specialty medications prior to reimbursement. PSPs are also a rich source of real-world evidence (RWE) as data can be collected across the patient journey. Here, we discuss opportunities and pitfalls associated with using Canadian PSP data for RWE, illustrated in a case study assessing trastuzumab deruxtecan (T-DXd) in breast cancer.
METHODS: A hybrid longitudinal cohort study was conducted among patients with HER2-positive and HER2-low metastatic breast cancer receiving treatment with T-DXd. Mainly, this study leveraged secondary data from the PSP, which included clinical/demographic characteristics and treatment information (e.g., duration, modification). These data were supplemented with primary data collected via an optional patient questionnaire (e.g., follow-up on therapies). Key outcomes included early discontinuation rates, dose modifications, time to treatment discontinuation, and reasons for treatment discontinuation.
RESULTS: This study showed that it was feasible to generate robust RWE using PSP data. We employed several strategies to ensure the quality and rigor of RWE, including study registration (NCT06386263), strict data governance/quality processes, third-party verification of analyses, a robust study design to meet RWE reporting guidelines, and an optional patient questionnaire for additional baseline data collection. Additionally, this study leveraged unique strengths of PSP RWE, including national representation of patients across Canada and the earliest opportunity to assess T‑DXd in the real-world. A few challenges, common to PSP RWE, were identified. First, due to reliance on secondary data from the PSP, all potential prognostic/confounding variables were not captured. Second, there were methodological/data rigor challenges (e.g., limited follow-up time, perceived bias with industry-funded PSP). These challenges were mitigated using strategies highlighted above.
CONCLUSIONS: RWE using PSP data offers unique opportunities to enhance decision making and improve patient outcomes. However, RWE leveraging the PSP should be planned early, ensure transparency/methodological rigor, and outline all known limitations.