OBJECTIVES: Patient access to cancer therapies can be limited due to restrictions set by national or regional health technology assessment (HTA) and/or pricing and reimbursement (P&R decision makers). This research explores variation from regulatory to P&R decisions impacting clinically eligible patients’ ability to receive appropriate pharmacotherapies.

METHODS: HTA and P&R assessments from Australia, Belgium, Canada, Denmark, France, Germany, Italy, the Netherlands, Poland, Portugal, Spain, Sweden, and the UK were obtained for oncology drugs approved in six cancers (breast, kidney, lung, multiple myeloma, melanoma, and prostate) by the European Medicines Association, Therapeutic Goods Association and Health Canada during 2006–2016. From published HTA documents, indications were classified according to the level of restriction between the regulatory label population, and those eligible for reimbursed access. The number of impacted patients was estimated from published epidemiology and budget impact data; potential survival gains from the pharmacotherapy were applied to estimate the impact of (disability) life years lost.

RESULTS: Of the 65 drug/indication combinations identified, in 50% no market access restrictions were imposed beyond the regulatory label; 22% of HTA decisions were not yet published, and 13% resulted in restriction to all clinically eligible patients across all countries; Poland, Scotland and Australia had the highest percentage of restrictions to licensed indications. A further 15% of HTA assessments limited access to a subgroup of the licensed population. Reasons for these limitations were infrequently reported across countries but when cited focused on price /budget impact and challenges around efficacy.

CONCLUSIONS: Discrepancies between regulatory and reimbursement decisions in Australia, Canada and Europe can impact health outcomes of cancer patients who are clinically eligible for treatment. There is variability in the factors that drive HTA/P&R decision making at the national level, and often the reasons for discrepancies between regulatory and HTA decisions are not transparent.