OBJECTIVES :

Growing pressure on the health budget and medicines expenditure led to risk-sharing agreements (RSA) between pharmaceutical industry and payer. A RSA for pertuzumab and trastuzumab in metastatic breast cancer (mBC) treatment was agreed within the scope of the reimbursement assessment in the Portuguese NHS hospitals in 2015. This agreement implies full support of pertuzumab and trastuzumab treatment by Roche after a certain treatment duration which requires real world data (RWD) collection by NHS hospitals.

Our aim is to analyze RWD reported in the scope of pertuzumab RSA implementation and put it in perspective with already available evidence.

METHODS :

Quantitative analysis of RSA reporting by hospitals based on anonymous and aggregated data collected from January 2016 to April 2019. Analysis of treatment duration, number of pertuzumab and trastuzumab cycles performed, supported by hospitals or Roche, and reporting time.

RESULTS : A total of 522 breast cancer patients were included in pertuzumab RSA. Median treatment duration with pertuzumab was 30 cycles (95% CI: 26–34) slightly longer (~21 months) than in pivotal Cleopatra trial (16,1 months, ~23 cycles).

Overall, 71% of the cycles estimated for payback by Roche were reported, suggesting a significant time lag between treatment administration and reporting from specific centers, emphasizing the benefit and need for monitoring patient access schemes.

This data refers to 33 NHS hospitals corresponding to 97% of hospitals that purchase pertuzumab.

CONCLUSIONS :

Data collected under pertuzumab RSA presents a valuable source of RWD describing patients’ treatment outside the scope of a clinical trial, in Portuguese NHS hospitals. RWD demonstrates pertuzumab RSA has the potential to provide better financial outcomes in the NHS perspective than initially estimated based on clinical trial evidence and presents a good option to deal with uncertainty of innovative therapies.