PHP224 - TEMPORARY ACCESS STRUCTURES FOR INNOVATIVE, HIGH-VALUE THERAPIES: COMPARISON OF MODELS AND CONSEQUENT REIMBURSEMENT TIMELINES

Abstract

The overall research aim was to compare temporary access structures for innovative, high-value therapies to evaluate the hypothesis that these can expedite patient access. In-depth secondary research identified and archetyped temporary access structures across major EU markets. A framework was developed to analyse 10 case studies, considering value statements, stakeholders, and timelines associated with an innovative, high-value therapy from early human studies to conclusion of a temporary access mechanism(s). The framework was designed to assess effectiveness of the temporary access mechanisms and suitability for the local environment and infrastructure / political climate. Temporary access mechanisms manifest differently, depending on ability to collect and analyse data. Legal infrastructure and political will to expedite patient access are not equivalent. FRA’s Temporary Authorisation for Use (ATU) has been one of the more successful examples, providing a national-level temporary access mechanism to identify innovative, high-value therapies addressing public health priorities where immature efficacy data may otherwise impede timely access. Political and social public health pressures, as well as perceptions of specific shortcomings of traditional value assessment procedures and methods, have jointly given rise to therapy area-specific temporary access mechanisms, including the CDF in ENG, which now provides a mechanism and budget for managed access to oncology drugs, pending evidence planning and generation. Further, therapy-specific budget impact barriers have led to ad-hoc temporary access mechanisms, such as earmarked funds for SOVALDITM (sofosbuvir, $GILD) in ITA, ESP, and ENG. Based on benchmarking, temporary access mechanisms are associated with expedited time to patient access. The analysis highlights opportunities and challenges to optimising broad, timely, and sustainable patient access through temporary access structures, and considerations for the equitable and sustainable operation of these schemes as clinical and economic features of the healthcare landscape evolve.