Value for money in genitourinary oncology.

Abstract

1 Background: Innovation in the treatment of genitourinary (GU) malignancies is accelerating, however, many of these new treatments are often not available for patients. We sought to investigate patient access, cost and value of new systemic anti-cancer compounds (SACTs) in GU oncology. We compared the US and Europe with a focus on the United Kingdom (UK) and the Republic of Ireland (IRL). Methods: Data on licensing and reimbursement decisions was collected on SACTs approved for GU oncology between 01/01/2010 to 09/01/2020. Overall survival (OS) benefit, progression free survival (PFS) benefit and cost of therapy were included in our analysis. Results: There were 29 regimens/indications approved by the Federal Drug Agency (FDA) for GU malignancies between 2010 and 2020. The majority of these (21/29, 72%) were also approved by the European Medicines Agency (EMA). Nearly all regimens/indications were approved by the FDA prior to the EMA (27/29, 93%), with a median time to EMA approval of 4 months (SD = 6, range -6 to 17). Only a minority of these regimens/indications are reimbursed by the public health systems in the UK (12/29, 41%) and IRL (6/29, 21%). Following EMA approval, public reimbursement of these regimens in the UK and IRL is often delayed with a median time of 8 months (+/- 6.3, range 4 - 21) and 11.5 months (+/- 12.5, range 0 - 33) respectively. Approximately half (15/29, 52%) of all reimbursed regimens/indications have demonstrated an overall survival benefit for patients. Androgen signalling inhibitors (ASIs) are more likely to have a proven OS benefit and had a lower mean cost than non-ASI regimens (See Table). A significant minority of regimens/indications (6/29, 21%) have demonstrated neither an OS or PFS benefit. The median price per month (USA list price) was $15,086 +/- $7,019. Regimens with a proven OS benefit were not more expensive than those without ($14,229 +/- $6,466 versus $16,004 +/- $7,704). There was no significant correlation between the length of OS benefit and the cost of regimens (Pearson’s Correlation Coefficient = -2.72, p = 0.445). Conclusions: Patients in the UK and IRL experience clinically significant delays in accessing FDA approved regimens in GU oncology. Only 52% of these regimens have a proven OS benefit. In our study, ASI regimens are cheaper and more effective than non-ASI regimens. There is no association between overall survival benefit and list prices in the USA. We need to radically overhaul licensing and funding of SACTs in GU oncology to ensure continued access for patients to effective medicines. [Table: see text]