PNS245 SNAPSHOT OF EARLY ACCESS PROGRAMMES IN PORTUGAL: A NEW ACCESS HURDLE?

Abstract

The current regulatory framework of the National Health Technology System (SiNATS) determines that access to new hospital drugs (marketing authorization after September 2017) might be granted before concluding the Health Technology Assessment (HTA) exclusively through early access programmes (EAP), conditional on the lack of therapeutic alternatives and the risk of immediate death or severe complications to patients. EAP conditions imply free-of-charge supply during the expected legal duration of the HTA process (210 days). The aim of this research was to perform a quantitative and qualitative point analysis of EAP appraisals in Portugal. The list of published EAP on INFARMED’s online database was reviewed on 1st June 2019. Variables collected: target population, therapeutic area, orphan drug status, number of patients enrolled, appraisal outcome and date. Logistic regression models were implemented to determine the influence of therapeutic area and orphan drug status on appraisal outcome. A total of 37 EAP submissions were recorded, 28 are active EAP while 9 (24%) were rejected. The odds of rejection were estimated to be statistically significant higher for oncology drugs, compared to other therapeutic areas (OR=14.4, p-value=0.018). Furthermore, a non-significant trend towards lower rejection odds was observed for orphan drugs (OR=0.33; p-value=0.21). EAP rejections have been especially noticeable since November 2018, with 45% of submissions being rejected in this period (n=9/20). A considerably higher rejection rate was also observed for oncology drugs (n=8/9, 89%). At the time of the analysis, there were 630 patients under EAP in Portugal. The mean duration for active EAP is 253 days, with some being active for up to 543 days. A substantial proportion of submitted EAP have been rejected, with significant differences between therapeutic areas. Delays in the HTA process might be extending the duration of EAP, and ultimately generating inadvertent expenditure and compromising drug access.