OBJECTIVES: Requesting access to new hospital medicines through Early Access Programs (EAP) before mandatory health technology assessment is concluded by the Ministry of Health (MoH) has become increasingly popular in Portugal. We aimed to assess the likelihood of rejection/deferral decisions and state-changes over time, of new EAP requests in the period between November 2019 and February 2023.

METHODS: An EAP state is a unique combination of the type of decision (rejected/deferred), exact therapeutic indication, number of patients allowed to enter, and cost implications due to the possibility of an initial free-of-charge period (EAP active with or without costs to the MoH). The odds of EAP rejection were analyzed through logistic regression, whereas the analysis of state-changes was based on descriptive statistics.

RESULTS: Data was identified on 296 states of 189 unique new EAP, covering 133 distinct medicines. The majority of medicines (n=105, 78.9%) had only one EAP, while the remainder presented two (n=17; 12.8%) and three or more (n=11, 8.3%) EAP. For the 189 EAP overall, deferral rates were high (n=133, 70.4%). The likelihood of EAP deferral has been increasing over time: for every additional year during the observation period, the odds of EAP deferral were estimated to be 1.96 times higher than the year before (95% CI: [1.38;2.86]). State-changes were observed in over half of the 133 deferred EAP (n=68, 51.1%), the majority of which included at least a change from an “active without costs” to an “active with costs” state (n=60, 45.1%).

CONCLUSIONS: The estimated increase in number of EAP deferred over time suggest that early access to innovative medicines in the Portuguese pharmaceutical market may be improving since 2019. Addressing equity concerns and anticipating the long-term policy implications stemming from the restrictive nature of EAP might become essential when meeting stakeholder expectations of broader access to innovation.