Objectives: The Early Access Program (EAP), created in September 2017 as part of the regulatory framework of the National Health Technology System (SiNATS), is the Portuguese strategy to allow patients to have access to new hospital drugs before the Health Technology Assessment (HTA) process is concluded. With this research, we intend to analyze the EAP appraisals in Portugal in a quantitatively and qualitatively perspective.

Methods: For this research, we analyzed the EAP published in the INFARMED's database until 31st of May 2021 and took under consideration the variables: Therapeutic Area, Number of patients, Date, Target Population and Appraisal Outcome.

Results: In the period of 2017 to 31st of May 2021, there were 120 EAPs submitted, aiming for 1250 patients, 20% of EAPs included pediatric. The number of EAPs have had a huge increase of 75% in 2019 (20 and 35 EAPs in 2018 and 2019, respectively) and stabilized in 2020 (36 EAPs, an increase of 3%). In 2021 we see already 27 submissions (average approval rate of 74%) and so an increase trend is back. The approval rate of overall EAPs is 63% (76 EAP) while 37% (44 EAP) were denied. While oncology represents more than 50% (64 EAPS; enrolling 539 patients) of the total number of EAPs, the odds of rejection are 3 times higher (OR= 3,29, p-value= 0,002) than other therapeutic areas.

Conclusions: The number of EAPs have increased over the years, despite the stabilization of growth in 2020, possibly caused by the pandemic situation. More and more patients are having beforehand access to innovative drugs through a healthcare solution as a EAP. The most representative area is Oncology most likely because EAPs are for life threating situations, where the denial rate is higher when compared to other medicines.