Anticancer drug prices and clinical outcomes: a cross-sectional study in Italy

Abstract

ObjectiveTo investigate whether the prices of new anticancer drugs correlated with their relative benefit despite negotiation.DesignRetrospective cross-sectional study correlating new anticancer drugs prices with clinical outcomes.SettingWe did a retrospective cross-sectional study including all new anticancer drugs approved by the European Medicines Agency (EMA) (2010–2016) and reimbursed in Italy.Main outcome(s) and measure(s)Information on clinical outcomes—in terms of median overall survival (OS), median progression-free survival (PFS) and objective response rate (ORR)—was extracted from pivotal trials as reported in the European Public Assessment Reports available on the EMA website. Cost of a full course treatment was estimated on negotiated official and discounted prices. Regression coefficients β, their levels of significance p and the coefficients of determination R2 were estimated adjusting by tumour type.ResultsOverall, 30 new anticancer drugs (with 35 indications) were available for analysis. Where data on OS were available, we observed no correlation between the improvement in median OS (in weeks) and negotiated price (R2=0.067, n=16 drugs for 17 indications). When the clinical outcomes were expressed as improvements in the median PFS or ORR, 25 drugs (29 indications) were available for the analysis, and again, there was no correlation with prices (R2=0.004 and 0.006, respectively).Conclusions and relevanceOur results suggest that the prices of anticancer drugs in Italy do not reflect their therapeutic benefit. Drug price negotiations, which is mandatory by law in Italy, do not seem to ensure that prices correlate with clinical benefits provided by the cancer drugs. These results call for further efforts to establish the standard determinants of drug prices available at the time of negotiation. These findings need to be confirmed in other countries where price negotiations are in place. Moreover, further investigations may verify whether outcome data obtained after drug marketing would improve the correlation between prices and therapeutic benefit.

Time to reimbursement and negotiation condition in Italy for drugs approved by the European Medicines Agency during the period 2014-2019

“…Likewise, oncology drugs do not show a quicker TTR compared to non-oncology drugs, but actually the TTR for oncology drugs is 2 months longer. It would be interesting to analyse their clinical data in order to assess their therapeutic added value compared to the standard of care at the time of their launch in terms of survival (improved rate or prolongation of survival) to determine whether this had an impact on the TTR (15).…”

A Proposal for Value-Based Managed Entry Agreements in an Environment of Technological Change and Economic Challenge for Publicly Funded Healthcare Systems

“…Italy negotiates drug prices only for binary decisions of approval or non-approval, not considering, during negotiations, whether there is a clear correlation between prices and benefits. Trotta et al ( 39 ) evaluated whether there were better correlations between cancer drug prices and clinical outcomes in a setting where central price negotiations are mandatory for every new medicine. The study demonstrated that:…”

Economic challenges of oncological diseases’ pharmacotherapy

“…Итальянские эксперты, проанализировавшие 30 новых противоопухолевых ЛС (зарегистрированы по 35 показаниям), одобренных в Евросоюзе в 2010-2016 гг., и стоимость которых возмещается в Италии, также пришли к заключению, что цены на эти препараты не отражают их терапевтическую пользу [33]. Аналогичное заключение было сделано и в недавно опубликованном анализе ЛС, зарегистрированных в США для лечения злокачественных новообразований ЖКТ [34].…”