Conditions and Restrictions on Early Access to Innovative Medicines in Portugal

OBJECTIVES: Patients living with critical diseases may require access to medicines that are unavailable to them for various reasons. In 2014, the National Health Technology Assessment System (SiNATS) facilitated access to innovative medicines through special early access programs (EAPs). Those are meant for accelerated pathways making new medicines available to patients before formal HTA assessment in Portugal. The aim of this study was to identify the type of restrictions on the target population of innovative medicines under EAPs, and quantify those restrictions according to the therapeutic indication resulting from public financing after formal HTA.

METHODS: All the EAPs approved and published on the INFARMED - National Authority of Medicines and Health Products website from January 2020 until December 2023 were reviewed. The database of EAPs was matched with the database of public reimbursement decisions. Restriction within the same target population in the EAPs was considered as an individual observation. Restrictions to drug utilization under EAP relative to therapeutic indication resulting from public financing after formal HTA were classified according to: patient population demographics; disease characteristics, treatment history; efficacy and safety variables.

RESULTS: A set of 7 restriction criteria were agreed upon: age, previous treatments, lack of efficacy or safety, resistance/refractory disease, contraindication/intolerance/ineligibility, disease-associated scores (e.g., ECOG), and disease characteristics/genetic profile/mutations. Of 55 EAPs reviewed, 45,5% restricted their target population due to contraindication/intolerance/ineligibility criteria, and 25,5% required EAP utilization after prior treatment. Overall, 29,0% of the EAPs were for orphan drugs, for which the most prevalent restrictions on early access were related to the disease's physiopathology and contraindications.

CONCLUSIONS: The most prevalent restrictions on early access to innovative medicines in Portugal are aligned with the legal requirement regarding the absence of therapeutic alternative for a specific target population in which patients are at immediate risk of life or of suffering serious complications.