Where HTA Meets Physics: Motion and Acceleration in Access to Innovative Medicines

OBJECTIVES: Accelerated assessment (AA) at the European Medicines Agency (EMA) is a regulatory procedure that reduces the marketing authorization (MA) review period from 210 to 150 days. However, patients access to medicine dependent on pharmaceutical and economic policy in each EU Member State. In Portugal, two major mechanisms are set to grant access to innovative medicines: Early Access Programs (EAP) and Public Reimbursement (PR) after rigorous HTA. EAP is to speed up access and PR is to maintain access sustainable. We borrowed the concepts of motion and acceleration from Physics to determine if market access acceleration at EMA is mirrored by access to innovative medicines in the Portuguese market.

METHODS: Medicines approved by EMA since 2010 (n=1,029) were matched with EAR (n=183) and PR (n=775) decisions in Portugal. Variables analysed included medicine name, status and date of MA, AA approvals by EMA, national access data regarding registration in the country, marketing status and dates of EAP and PR initiation if positive. Negative public financing decisions were treated as censored observations. Standard time-to-event data analysis was used to assess time-to early access, time-to-public reimbursement, and time-to-first access whether AE or PR. Incidence rates (number of new accessible medicines by month) provide evidence on the speed of access across the European and country context.

RESULTS: New medicines approved by AA at EMA faced incidence rates of time-to-first access 3.4 times higher than non-accelerated EMA approvals (p-value<0.001). Time-to-public reimbursement was more accelerated for AA medicines when PR occurred without EAP, incidence rates of 0.015 vs 0.004 (p-value<0.001). Rate of new accessible medicines was lower when PR and preceded by early access programs, incidence rates of 0.0047 vs 0.0016 (p-value<0.001).

CONCLUSIONS: Mechanism of granting access to innovative medicines in Portugal are associated with faster access in EMA accelerated procedures.