The value of the accelerated approval pathway: real-world outcomes associated with reducing the time between innovations

<b>Aim:</b> We investigated the effect of shortening time between innovations with the accelerated approval (AA) pathway on patient outcomes for three solid tumors. <b>Methods:</b> This real-world analysis evaluated patients receiving sequential AA pathway-approved innovations after initial treatment with existing therapies in three solid tumor case studies. Outcomes attributable to AA were estimated and assumed approval occurred at the time of conversion to approval and extrapolated to the US population. <b>Results:</b> Survival gains from accessing innovative therapies were 2.3–3.8-times higher when using the AA pathway. At the US population level, AA was associated with ∼8000 life-years gained across all three tumor case studies. <b>Conclusion:</b> In areas of rapid clinical development, the value of existing therapies can be enhanced by earlier access to AA pathway innovations and should be considered when evaluating the AA program. The US Food and Drug Administration's accelerated approval pathway provides patients with access to innovative drugs sooner than standard regulatory pathways. Using three case studies in solid tumors, this study measured how many patients on current cancer drugs received future cancer drugs because of the accelerated approval pathway and asked whether quicker access to new drugs resulted in them living longer. In three cancer case studies, the accelerated approval pathway led to more patients receiving future cancer drugs. Patients who received future drugs through the AA pathway lived longer than patients who did not have access to them. The accelerated approval pathway is important because it can improve outcomes of current cancer drugs by giving patients additional treatments to choose from in the future and therefore a chance to live longer. Policymakers should consider this when thinking about making changes to the accelerated approval pathway. The accelerated approval (AA) pathway increased the number of patients already being treated with existing therapies who were able to access future subsequent treatments in three solid tumor case studies. The AA drugs added additional survival to that of the initial drugs, with a majority of this additional survival being attributable to the AA pathway. The additional survival provided by the AA pathway led to meaningful life-year gains at the US population level. The results were driven by both the effectiveness of the AA drug as well as tumor severity and suggest that tumors with rapid disease progression may derive greater benefits from shortening the time between innovations via the AA pathway. Policymakers should address the improved outcomes as a result of the earlier access due to the AA pathway when considering future modifications to the US FDA's AA program.

研究目的：本研究旨在探讨加速批准（Accelerated Approval, AA）通路下缩短创新疗法间隔时间对三种实体瘤患者预后的影响。
研究方法：本项真实世界分析针对三项实体瘤案例研究，评估了在接受现有疗法初始治疗后，序贯接受加速批准通路获批创新疗法的患者。研究估算了与加速批准通路相关的临床结局，并假设批准于获批转换时生效，同时将研究结果外推至美国人群。
研究结果：通过加速批准通路获取创新疗法时，患者的生存获益较对照提升2.3~3.8倍。在美国人群层面，三项实体瘤案例研究中，加速批准通路累计带来约8000个生命年的增益。
研究结论：在临床快速发展领域，提前通过加速批准通路获取创新疗法可强化现有疗法的临床价值，该结论应在评估加速批准计划时予以考量。美国食品药品监督管理局（U.S. Food and Drug Administration, FDA）的加速批准通路较标准监管通路更早为患者提供创新药物可及性。本研究通过三项实体瘤案例研究，量化了当前接受癌症治疗的患者因加速批准通路得以使用后续抗癌药物的比例，并探究了更快获取新药是否能延长患者生存期。三项癌症案例研究显示，加速批准通路使更多患者能够接受后续抗癌疗法。通过加速批准通路获取后续药物的患者，其生存期较未获得该通路可及性的患者更长。加速批准通路的重要性在于，它可为患者提供未来更多可选治疗方案，从而提升现有疗法的临床获益并延长患者生存期，政策制定者在调整加速批准通路相关政策时应考虑这一点。在三项实体瘤案例研究中，加速批准通路提升了已接受现有疗法的患者能够获取后续治疗的比例。加速批准药物为初始疗法增添了额外生存获益，其中大部分额外生存获益可归因于加速批准通路。加速批准通路带来的额外生存获益，在美国人群层面实现了具有临床意义的生命年增益。研究结果同时受加速批准药物的疗效与肿瘤严重程度影响，提示疾病进展迅速的肿瘤或可通过加速批准通路缩短创新疗法间隔，从而获得更大获益。政策制定者在考虑修订美国FDA加速批准计划时，应将加速批准通路通过更早给药带来的预后改善纳入考量范畴。