1370P - A New Proposal of Drug Pricing Based on Value for Effectiveness in Real Practice Oncology

Abstract

ABSTRACT In Europe pricing of new drugs is assessed directly by member countries contracting with pharmaceutical companies. Health technology evaluation is based on efficacy/safety results in approval RCTs and scarce resource allocation. Since clinical outcomes in wider population are more variable and conditions less selected, the clinical outcomes may result changed. The real cost benefit value should be revised once effectiveness data from clinical practice is available. The clinical data were collected from web-based national oncology registry Onco-AIFA, as part of mandatory surveillance. The observational study was performed on 724 advanced cancer patients treated with high novel oncology drugs. The median follow up was 67 months form May 2006 to December 2011. Approval RCTs of seven selected drugs were reviewed. The comparison was performed between survival outcomes achieved from RCT and those from our setting (see table). Progression free survival is a valuable indicator for efficacy/effectiveness assessment .Based on the difference in benefit in PFS, a new price adjusted for effectiveness was proposed. The post-marketing evaluation showed that in all analysed drugs the PFS from clinical practice was shorter in comparison to RCTs outcomes. As a result the real price value should be revised taking into account the difference of clinical response. The calculated effectiveness adjusted price is ex-factory price, expressed in euro per 1 mg, proportional to the difference of PFS form RCTs and that from clinical practice. The post marketing study allows for assessment of response outcomes inreal life practice in order to verify both effectiveness and safety in general population testing external validity of the randomized trials. This kind of assessment lacks in approval RCTs, further emphasizing the importance of observational investigations in clinical practice. The price should be based on net clinical benefits achieved in real life practice as more appropriate in evaluating cost-benefit balance. Disclosure All authors have declared no conflicts of interest. Table: 1370P Drug Type Cancer Type PFS in RCT (months) PFS in Clinical Practice Oncology (months) Ex-factory (euro /1mg) Effectiveness Ajusted Price (euro /1mg) Difference in priceDiscount proposal Sorafenib Kidney 5,5 3,2 0,16 0,09 44% Sorafenib Liver 5,5 3,0 0,16 0,09 44% Erlotinib NSCLC 2,2 2,0 0,48 0,44 8% Sunitinib Kidney 11,0 7,0 3,87 2,46 36% Cetuximab Colorectal 4,1 3,0 2,08 1,52 27% Pemetrexed Lung adenoca 2,9 1,8 3,02 1,87 38% Bevacizumab Colorectal 10,6 6,3 3,36 2,00 40% Bevacizumab Breast 11,8 7,9 3,36 2,25 33% Panitumumab Colorectal 2,0 1,9 4,22 4,01 5%