Cost and value of cancer medicines in a single-payer public health system in Ontario, Canada: a cross-sectional study.

Background: The financial impact of cancer medicines on health systems is not well known. We describe temporal trends in expenditure on cancer medicines within the single-payer health system of Ontario, Canada, and the extent of clinical benefit these treatments offer.

Methods: In this cross-sectional study, we identified cancer medicines and expenditures from formularies and costing databases (the New Drug Funding Program, Ontario Drug Benefit Program, and The High-Cost Therapy Funding Program) during 10 consecutive years (April 1, 2012, to March 31, 2022) in Ontario, Canada.

Probability of Regulatory Approval Over Time: A Cohort Study of Cancer Therapies.

Purpose: New cancer therapies are frequently evaluated in multiple disease indications. We evaluated whether the probability of achieving US Food and Drug Administration (FDA) approval for a new cancer therapy changes with time.

Methods: We identified a cohort of anticancer drugs with a first registered efficacy trial from 2007 to 2011 on ClinicalTrials.

Large numbers of patients are needed to obtain additional approvals for new cancer drugs: A retrospective cohort study.

Patients endure risk and uncertainty when they participate in clinical trials. We previously estimated that 12,217 patient-participants are required to bring a new cancer drug to market. However, many development efforts are aimed at extending the label of already approved drugs.

Bypassing phase 2 in cancer drug development erodes the risk/benefit balance in phase 3 trials.

Objectives: Drug developers sometimes launch phase 3 (P3) trials without supporting evidence from phase 2 (P2) trials. We call this practice "P2 bypass." The aims of this study were to estimate the prevalence of P2 bypass and to compare the safety and efficacy results for P3 trials that bypassed with those that did not.

Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers: An Overview of Global Research Activity.

Background: In this study, we compared and contrasted design characteristics, results, and publications of randomized controlled trials (RCTs) in gastrointestinal (GI), lung, and breast cancer. Methods: A PUBMED search identified phase III RCTs of anticancer therapy in GI, lung, and breast cancer published globally during the period 2014−2017. Descriptive statistics, chi-square tests, and the Kruskal−Wallis test were used to compare RCT design, results, and output across the cancer sites.