Current landscape of US Food and Drug Administration use of expedited regulatory approvals for colorectal cancer care.

IF 2.9 4区医学 Q2 HEALTH CARE SCIENCES & SERVICES

Journal of managed care & specialty pharmacy Pub Date : 2025-09-01 DOI:10.18553/jmcp.2025.31.9.929

Danea M Horn, Mireille Jacobson, Kathryn A Phillips

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引用次数: 0

Abstract

Background: US Food and Drug Administration (FDA) expedited regulatory pathways (ERPs) accelerate the availability of drugs and diagnostic tests for severe conditions and fill unmet medical needs. ERPs accelerate access to new therapeutics but also increase the uncertainty of the benefits of available treatments.

Objective: To examine how ERPs have influenced the landscape of pharmaceutical treatments for colorectal cancer (CRC), one of the most common forms of cancer in the United States.

Methods: This cross-sectional study used data from public FDA records on all CRC drug approvals before and after the passage of the FDA Safety and Innovation Act in 2012. Descriptive analyses were performed to characterize FDA approval trends by ERP. Primary outcomes included the number and timing of FDA-approved CRC drugs, the use of ERPs, outstanding postmarketing requirements, and the availability of molecular diagnostic tests associated with these treatments.

Results: Of the 24 FDA-approved CRC drugs on the market, 75% were approved through at least 1 ERP. The use of ERPs for FDA approvals increased by 18 percentage points (from 63% to 81%) pre-to-post 2012 or 30% relative to baseline. The most common pathway was Accelerated Approval, which accounted for 72% of ERP-approved drugs. CRC treatments have become increasingly targeted using molecular diagnostics, with 25% of CRC drugs approved before 2012 having associated molecular diagnostics, increasing to 75% after 2012 and 100% after 2018.

Conclusions: ERPs have expedited approvals for new and increasingly targeted CRC treatments. All CRC treatments approved through Accelerated Approval or Breakthrough Therapy Designation after 2018 still await confirmatory trial results. These findings highlight the complexity of available drugs and diagnostic tests and the challenges facing managed care pharmacists in formulary management, diagnostic test coordination, and the development of utilization criteria given limited long-term clinical evidence.

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美国食品和药物管理局使用加速监管批准结肠直肠癌治疗的现状。

背景：美国食品和药物管理局（FDA）加快了监管途径（erp），加快了重症药物和诊断测试的可得性，并填补了未满足的医疗需求。erp加速了对新疗法的获取，但也增加了现有疗法效益的不确定性。目的：研究erp如何影响结直肠癌（CRC）的药物治疗景观，结直肠癌是美国最常见的癌症之一。方法：本横断面研究使用了2012年FDA安全与创新法案通过前后所有结直肠癌药物批准的FDA公开记录数据。描述性分析通过ERP描述FDA批准趋势。主要结局包括fda批准的结直肠癌药物的数量和时间，erp的使用，上市后的突出要求，以及与这些治疗相关的分子诊断测试的可用性。结果：在fda批准上市的24种结直肠癌药物中，75%通过至少1个ERP获得批准。2012年前后，erp在FDA审批中的使用增加了18个百分点（从63%增加到81%），或相对于基线增加了30%。最常见的途径是加速审批，占erp批准药物的72%。分子诊断对结直肠癌治疗的针对性越来越强，2012年之前批准的结直肠癌药物中有25%具有相关的分子诊断，2012年之后增加到75%，2018年之后增加到100%。结论：erp加速了新的和越来越有针对性的结直肠癌治疗的批准。2018年之后通过加速审批或突破性疗法认定获得批准的所有结直肠癌治疗仍在等待验证性试验结果。这些发现突出了现有药物和诊断测试的复杂性，以及管理式护理药剂师在处方管理、诊断测试协调和制定使用标准方面面临的挑战，因为长期临床证据有限。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of managed care & specialty pharmacy Health Professions-Pharmacy

CiteScore

3.50

自引率

4.80%

发文量

131

期刊介绍： JMCP welcomes research studies conducted outside of the United States that are relevant to our readership. Our audience is primarily concerned with designing policies of formulary coverage, health benefit design, and pharmaceutical programs that are based on evidence from large populations of people. Studies of pharmacist interventions conducted outside the United States that have already been extensively studied within the United States and studies of small sample sizes in non-managed care environments outside of the United States (e.g., hospitals or community pharmacies) are generally of low interest to our readership. However, studies of health outcomes and costs assessed in large populations that provide evidence for formulary coverage, health benefit design, and pharmaceutical programs are of high interest to JMCP’s readership.