加速获取与早期阶段证据：15年PBAC提交的经验教训 Jiasu huoqu yu zaokai jiduan zhengji: 15nian PBAC提交 de jingyan xunliu

Accelerated Access and Early-PhaseEvidence: Lessons from 15 Years of JACK SHEPPARD, Associate Principal, Management Consulting, IQVIA Australia & New Zealand DR BRANDON WRIGHT, Consultant, Management Consulting, IQVIA Australia & New Zealand Table of contents Executive summaryMethodologyOutcomes by phase of dataEvolution over timeSubmission methodology and therapy areas Executive summary This report analyses the use of early-phase clinical trial data in submissionsto the Pharmaceutical Benefits Advisory Committee (PBAC) for the funding of Between 2010 and 2025, only 3.8% of PBAC submissionsrelied on early-phase clinical trial data, yet theseachieved notable success when supported by robustevidence and high unmet needs. Of 57 submissions Methodology We extracted data from IQVIA Health TechnologyAssessment data assets to find all submissions made toPBAC from 2010 onwards, filtered by the latest phase(e.g. Phase III is higher than Phase II) of clinical trial dataused. Our report examines medicines recommendedby PBAC for funding that had been submitted using Activity increased from 2016 onward, peaking during2020–2023 amid global shifts toward accelerated access.Oncology dominated (76% of submissions), reflectingchallenges in conducting large-scale trials for rarecancers. Economic evaluations primarily used cost- Between 2010 and 2025, only 3.8% of PBAC submissions relied on early-phase clinical trial data, yet these achieved notable success when supported a 75% recommendation rate. Submissions supportedby Phase II/III data were evenly split, with half Outcomes by phase of data In total, 33 unique molecules were submitted toPBAC with early-phase data during the study period,accounting for 57 individual submissions. Thisrepresents 3.8% of the ~2,000 total PBAC submissions These findings suggest that while early-phase dataare not commonly used in PBAC submissions, theycan be effective when the evidence is robust, andthe clinical context justifies earlier access. The high Most early-phase submissions were supportedby Phase II data, which accounted for 47 of the57 submissions. These submissions had a notably PBAC demonstrates flexibility when early-phase data is supported by strong evidence rejected — providing a clear view of both volume and Evolution over time An analysis of the time distribution of PBACsubmissions supported by early-phase clinical trialdata reveals several important trends. Figure 2 The data show that early-phase submissions occurredsporadically in the first half of the decade, withisolated accepted submissions in 2010 and 2011, Despite the relatively stable number of early-phasesubmissions in recent years, this trend must beviewed in the context of the overall growth in PBACsubmissions. While the absolute number of early- lodged — three accepted and one rejected. This marksthe beginning of a more consistent pattern of early- The years 2020 through 2023 show the highest levelsof activity, with each recording between four andsix submissions. Notably, the majority of these wereaccepted, while a smaller proportion were deferred. Overall, the evolution of early-phasesubmissions over time reflectsa dynamic interplay between — accounting for 76% of all cases. This dominancelikely reflects the urgent need for new treatments in Submission methodology This section explores two key dimensions of PBACsubmissions supported by early-phase clinical trialdata: thehealth economic methodologyused in thesubmission and thetherapeutic areaof the medicine 44% of submissions used cost-effectiveness analysis, while 41%used cost-utility analysis. Figure 3A highlights that most early-phasesubmissions employed either a cost-effectiveness orcost-utility approach. Specifically, 44% of submissionsused cost-effectiveness analysis, while 41% used cost-utility analysis. These methodologies are typically EXAMPLE CASE 1 A notable example is the submission of Rozlytrekfor ROS1-positive Non-Small Cell Lung Cancer(NSCLC), a variant affecting only 1–2% of NSCLCpatients. This cost-minimisation submission In contrast, only 13% of submissions used a cost-minimisation approach. This methodology is generallyreserved for situations where the new treatment isdemonstrably equivalent in clinical outcomes to an Together, the findings indicate that early-phasesubmissions are most viable in areas where there is astrong unmet need, a clear economic rationale, and Figure 3B provides insight into the therapeutic areasmost associated with early-phase submissions. The Flow of submissions The Sankey diagram (Figure 4) maps the journey ofearly-phase submissions through the PBAC process,from initial submission to eventual recommendation. Of the 33 early-phase submissions identified in thisanalysis, 25 were ultimately accepted, representinga 76% overall success rate. Notably, a substantialproportion of these were successful on their firstsubmission, indicating that early-phase data can be EXAMPLE CASE 2 An example