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UBC Theses and Dissertations

Describing sources of uncertainty in cancer drug formulary priority setting across Canadian provinces Jenei, Kristina


Introduction: Over the years, there have been significant advances in oncology. However, the rate that therapeutics come to market have increased while the strength of evidence has decreased - leaving decision makers with more uncertainty than ever before. Currently, there is limited understanding about how this uncertainty is understood and managed in provincial funding decisions for cancer therapeutics. Methods: A qualitative, two-phase study approach was employed. Phase I comprised of semi-structured key-informant interviews (n=6) with senior officials from four Canadian provinces (BC, AB, QC and ON). In part II, a document review of the uncertainties found in clinical evidence in the pan-Canadian Oncology Drug Review (pCODR) assessments was conducted. Interviews in part I were audio-recorded and transcribed verbatim. Results: Participants included stakeholders from British Columbia (BC) (n=1), Alberta (AB) (n=1), Quebec (QC) (n=3) and Ontario (ON) (n=1) whom held a variety of roles in ministries of health, cancer agencies and national health technology assessment (HTA) organizations that evaluate evidence and make funding recommendations. Participants reported considerable uncertainty related to a lack of solid clinical evidence (early-phase clinical trials: generalizability, immature data and the use of unvalidated surrogate outcomes). Clinical uncertainty was exacerbated with high costs and accelerated approvals. Other sources of uncertainty were related to external influences. Proposed strategies to deal with the uncertainty included risk-sharing agreements, collection of real-world evidence (RWE) and ongoing collaboration between federal groups and provinces. The document review added to the reported uncertainties by classifying them into five main categories: trial validity, population, comparators, outcomes and intervention. Conclusion: This study highlights that decision makers have to deal with more uncertainty in funding decisions for cancer drugs than ever before and that this uncertainty generally stems from clinical trials. Since only one decision maker could identify a deliberative priority setting process and cancer drugs are rarely reassessed, this situation might leave ineffective drugs in the health system. These drugs can incur opportunity costs. There is a critical need for transparent priority setting processes and mechanisms to reevaluate drugs to ensure benefit given the high level of uncertainty of novel therapeutics.

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