Retrospective pharmaceutical financial benefits and cost avoidance analysis of clinical trial participation in the Australian haematology setting.

Abstract

Clinical trial participants receiving investigational new drugs, which subsequently become approved by a medicines regulatory authority for its trialled indication, effectively gain free early access to efficacious treatment. Participants may also benefit from receiving approved, but unsubsidised medicines. These financial benefits of clinical trial participation have not previously been defined or quantified. Additionally, there are limited Australian pharmaceutical cost avoidance studies quantifying government savings through sponsored clinical trials. To calculate pharmaceutical financial benefits and cost avoidance of clinical trial participation at a single Clinical Research Unit. Recruiting clinical trials between 1 January 2006 and 31 December 2017 conducted at the Haematology Clinical Research Unit, Concord Repatriation General Hospital, Sydney were reviewed. Dispensing records were used to quantitate the pharmaceuticals dispensed to every participant. Financial calculations were based on Pharmaceutical Benefits Scheme (PBS) pricing, or from UpToDate for non-PBS listed agents. Thirty-six eligible clinical trials involving 245 participants accrued AU$3 971 357 in financial benefit from early access to subsequently approved investigational new drugs, AU$12 209 538 in financial benefit from accessing approved medications not PBS listed, and AU$6 728 576 in government cost avoidance. These findings totalled AU$22 909 471, 89% of which was derived in the past 5 years. Pharmaceutical financial benefit is a previously unquantified aspect of clinical trial participation, its assigned value reflecting a measure of the quality and quantity of life delivered to patients. These data, albeit from a single discipline and institution, suggest that financial benefit represents a greater value than cost avoidance, and that its inclusion in cost-analyses may better reflect the monetary benefits of accessing efficacious pharmaceutical agents through clinical trials.