Parametric Survival Extrapolation of Early Survival Data in Economic Analyses: A Comparison of Projected Versus Observed Updated Survival.

OBJECTIVES: To establish the value of cancer drugs by cost-effectiveness analysis, lifetime parametric survival extrapolations are often fitted to early data. Recent literature suggests that the benefit of cancer agents in primary publications is often different compared with updated data. This study aimed to examine the projected survival based on parametric extrapolations compared with observed survival based on updated data. METHODS: US Food and Drug Administration oncology approvals from January 2006 to December 2015 were reviewed to identify randomized controlled trials, with updated overall survival (OS) or progression-free survival (PFS) data within 5 years. Individual patient data were reconstructed using established methods on initial and updated publications. Projected survival was calculated as the best-fit parametric restricted mean survival time (RMST) based on extrapolated initial Kaplan-Meier curves whereas observed survival was calculated as observed RMST based on updated Kaplan-Meier curves. Mean deviations, mean absolute error (MAE), mean absolute percentage error, and linear regressions were conducted to examine the relationship between projected and observed survival. RESULTS: In total, 32 randomized controlled trials were included. The MAE between the projected RMST and observed RMST was 3.18 months (OS) and 2.84 months (PFS) and absolute percentage error of 100% (OS) and 23% (PFS), suggesting substantial imprecision of the projected RMST in predicting the updated RMST. The linear regression indicated MAE increased as time extrapolated and as the percentage of censored patients increased. CONCLUSIONS: This study demonstrated substantial difference in projected survival between initial and updated publications. Health technology assessment committees need to be aware of the potential uncertainty of incremental effectiveness and resultant value-for-money assessment when making reimbursement decisions based on initial publications with immature survival data.

A cost analysis of a community-based support centre for cancer patients and their families in Ireland: the EVeCANs study.

Cancer patients and their families experience a range of physical, psychological and financial adverse effects. Community-based cancer centres offer a range of services and interventions, free of charge, to support those affected by cancer. While shown to be effective, there is a lack of information on the costs of these services. Our aim was to estimate the resource impact of a community-based cancer support centre. Over a 7-month period, there were 2032 contacts with 238 clients whose average age was 60 years. The most frequently used services were transport to treatment (20%), complementary therapies (48%), exercise classes (10%) and counselling (9%). This cost analysis estimated total annual cost to provide all services was €313,744. Average annual cost per person was €1138. Current uptake at the centre represents 8% of all cancer incidences in seven counties surrounding the centre. If uptake increases by 10%, scenario analyses predict an increase in total costs increase to €429,043 and a decrease in costs per patient to €915. As cancer incidences increase, the need for supportive care is growing. Community-based services have been established to meet these needs and fill this gap in national health services. Long-term sustainability of these centres is uncertain as they are entirely reliant on donations and volunteers. This analysis estimates the costs of one such community-based cancer support centre, for the first time in Ireland. Findings can be used to inform future planning of cancer supportive care services, including establishing links between tertiary and community-based centres, and cost effectiveness analyses, nationally and internationally.

Cost and public reimbursement of cancer medicines in the UK and the Republic of Ireland.

INTRODUCTION/AIMS: There are disparities in the availability of systemic anticancer therapies (SACTs) globally. We set out to investigate the cost and reimbursement of SACTs in the United Kingdom (UK) and the Republic of Ireland (ROI) in conjunction with efficacy and licensing authority decisions in the United States (US) and the European Union (EU). METHODS: We sought data pertaining to licensing in the EU, reimbursement in ROI/UK and cost/efficacy of SACTs licensed by the Food and Drug Administration (FDA) between January 2015 and May 2021. Independent samples t tests, chi-square test and Pearson's correlation were used for statistical analysis. RESULTS: We identified that the majority of FDA-approved regimens are licensed by the European Medicines Agency (EMA) (n = 91, 67.9%). However, only a minority of these are currently reimbursed in the UK (n = 60, 45%) or the ROI (n = 28, 21%) as of the 1st of May 2021. In addition, only a minority of regimens have demonstrated a statistically significant OS benefit (n = 54, 40%). There was no association between cost of regimens and either the presence (t = 0.846, p = 0.40) or duration of OS benefit (t = - 0.84, p = 0.64). CONCLUSIONS: Our study highlights that many licensed systemic anticancer treatments are not currently reimbursed in ROI/UK. The high cost of these medicines is independent of the presence of an OS benefit. Collaboration between regulatory agencies, governments and industry partners is needed to ensure health expenditure is directed towards the most effective treatments.