Journal Publications

Cheung MC, Chan KK, Golden S, Hay A, Pater J, Prica A, Chen BE, Leighl N, Mittmann N. Minimization of resource utilization data collected within cost-effectiveness analyses conducted alongside Canadian Cancer Trials

Cost-effectiveness analyses embedded within randomized trials allow for evaluation of value alongside conventional efficacy outcomes; however, collection of resource utilization data can require considerable trial resources.

These results suggest that the collection of resource utilization data within cancer trials could be narrowed. Omission of certain cost categories that have minimal impact on incremental cost-effectiveness ratio, such as routine laboratory investigations, could reduce the costs and undue burden associated with the collection of data required for cancer trial cost-effectiveness analyse

Reassessing the Net Benefit of Cancer Drugs With Evolution of Evidence Using the ASCO Value Framework

Regulatory approval of cancer drugs is often based on evidence from surrogate endpoints. Recent work suggests that most drugs have been approved by the FDA or the European Medicines Agency based on improvements in surrogate endpoints alone without evidence of extending or improving life.^1–3 These surrogate outcomes, such as response rates or progression-free survival (PFS), are also reported in primary publications, at which time overall survival (OS) data may be immature.¹ Other clinically relevant data, such as quality of life (QoL) and long-term survival outcomes, are often reported in subsequent publications after initial drug approval.² However, the submission of such subsequent evidence is generally not required for the reassessment of ongoing approval, despite its importance in clinical decision-making. This highly relevant information is thus often unavailable at the time of approval of novel oncology drugs and may not be considered by regulatory agencies for ongoing approval.

On average, among FDA-approved drugs, no overall improvement in the ASCO-VF NHB occurred with longer follow-up and evolution of evidence. As further evidence was published, more than 25% of indications changed in classification, which could be either an increase or a decrease in NHB. QoL information was updated postapproval in >40% of indications. Thus, reassessing the ASCO-VF NHB when more evidence becomes available may be beneficial to inform clinical shared decision-making and evidence reappraisal for regulatory and reimbursement decisions.