Reasons for Drug Market Access Failure in Portugal: A Retrospective Analysis of Public Reimbursement Reports

OBJECTIVES: This study investigates the reasons for failures in drug market access in Portugal, particularly due to negative reimbursement recommendations within the National Health Technology Assessment (HTA) System. The main objective is to determine the reasons of these failures.

METHODS: The INFARMED website was searched for Reimbursement Evaluation Reports published between Jul-2020 and Feb-2024. Data was extracted from these reports to record whether each HTA process was approved, rejected or archived regarding reimbursement decisions, and the reasons for these verdicts. Sources of evidence and results of studies considered in each process were analyzed versus the assessment matrix defined by the HTA Committee (CATS) (i.e., PICO criteria). For each drug with a positive pharmacotherapeutic evaluation (e.g., added therapeutic value [ATV] versus comparators), the results of the pharmacoeconomic evaluation (e.g., Incremental Cost-Effectiveness Ratios [ICERs]) and the negotiation stage were examined to elucidate potential causes of reimbursement rejection. Descriptive statistics were used (Microsoft Excel®).

RESULTS: Out of 223 processes, 192 (86%) were recommended for reimbursement, and 21 (9%) received a negative recommendation. Ten (5%) processes were archived. Most negative reimbursement recommendations (n=16; 76%) was supported by failures in pharmacotherapeutic evaluation, namely inability to show ATV versus comparators. The reasons were lack of adequate evidence for comparisons (n=11; 69%), negative/ non-robust results to demonstrate clinical value in the evidence supporting comparisons (n=3; 19%) or both reasons (n=2; 12%). The remaining negative recommendations (n=5; 24%) were supported by high acquisition costs of the drugs, considered unsustainable for the National Health System.

CONCLUSIONS: The present findings suggest that pharmacotherapeutic evaluation is the stage of the HTA process that most impacts the outcome of a reimbursement recommendation. The mismatch between the requirements of the evaluation matrices (PICO) and the clinical evidence available on the medicines under evaluation and their comparators limits market access of therapeutic innovation.