Search

Unlike other high-income countries, Canada has no national policy for drugs treating rare diseases (orphan drugs). Nevertheless, in 2022, the Canadian government committed to creating a national strategy to make access to these drugs more consistent. Our aim was to study whether recommendations made by the Canadian Agency for Drugs and Technology in Health (CADTH) translated into coverage decisions for orphan drugs in Ontario, the largest Canadian province. This study is the first to look at this question for orphan drugs, which are at the center of policy attention. We included 155 orphan drug-indication pairs approved and marketed in Canada between October 2002 and April 2022. Cohen's kappa was used to test the agreement across health technology assessment (HTA) recommendations and coverage decisions in Ontario. Logistic regression was used to test which factors, relevant to decision-makers, might be associated with funding in Ontario. We found only fair agreement between CADTH's recommendations and coverage decisions in Ontario. Although a positive and statistically significant association between favorable HTA recommendations and coverage was found, more than half of the drugs with a negative HTA recommendation were available in Ontario, predominately through specialized funds. Successful pan-Canadian pricing negotiations were a strong predictor of coverage in Ontario. Despite efforts to harmonize access to drugs across Canada, considerable room for improvement remains. Introducing a national strategy for orphan drugs could help increase transparency, consistency, promote collaborations, and make access to orphan drugs a national priority. • Recommendations by the Canadian Agency for Drugs and Technology in Health do not always translate to provincial coverage decisions for new drugs. • We explored whether Canadian Agency for Drugs and Technology in Health's recommendations are aligned with coverage decisions for orphan drugs in Ontario. • Negative health technology assessment recommendations did not necessarily translate to no pan-Canadian pricing negotiations. • More than half the drugs with negative health technology assessment recommendations were available in Ontario through specialized funds. • A national strategy for orphan drugs could prioritize access to these treatments at a national level and harmonize access, at least to some extent, across Canada. [ABSTRACT FROM AUTHOR]

• All-cancer radiotherapy utilization ratios (RTUR) varied across Canada by 9%, and up to 28% by cancer type. • RTURs were below Ontario benchmarks for many provinces and cancer subsites. • RTURs can be a reasonable surrogate for linked (patient-specific) utilization. Access to radiotherapy (RT) is a key component of a cancer control strategy. However, radiotherapy utilization (RTU) rates fall short of desired benchmarks in certain Canadian provinces. We aimed to describe provincial variations in RTU across Canada. We calculated radiotherapy utilization ratios (RTUR) for each Canadian province from 2016 (RT case counts divided by incidence counts), by cancer type (all cancers, lung, breast, rectal, prostate) and treatment intent (curative, palliative) where data were available. Data were extracted from each provincial RT data repository, cancer registry and/or RT department. We compared RTURs descriptively across provinces and to Ontario benchmarks, and calculated an estimated national RTUR. In provinces with capacity for data linkage, RTURs were compared to a linked (patient-specific) method of calculating utilization, by linking each incident case to whether RT was received within 1 year of diagnosis (RTU-1 yr). Excluding three provinces that included re-treatments, all-cancer RTURs ranged from 0.31 in Manitoba to 0.40 in Nova Scotia. The national all-cancer RTUR was 0.35, which was comparable to Ontario benchmarks (0.34). Larger variations were seen by cancer type, with an absolute difference in RTURs of 28% for lung cancers, 27% for breast cancers, 21% for rectal cancers, and 18% for prostate cancers. RTURs for nearly all provinces were below established Ontario benchmarks for each cancer type, except prostate cancer. RTURs over-estimated RTU-1 yr by at most 5%, except for prostate cancers where they over-estimated RTU-1 yr by up to 15%. RTU varies by province in Canada, and most notably by cancer subsite. More granular data at the regional level and by healthcare facility is required to further tailor strategies aimed at improving RT access. RTURs also serve as a reasonable surrogate for linked RTU, and both methods can contribute meaningfully to measure RTU depending on the context and data availability. [ABSTRACT FROM AUTHOR]