Danea M Horn, Mireille Jacobson, Kathryn A Phillips
{"title":"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。","authors":"Danea M Horn, Mireille Jacobson, Kathryn A Phillips","doi":"10.18553/jmcp.2025.31.9.929","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":16170,"journal":{"name":"Journal of managed care & specialty pharmacy","volume":"31 9","pages":"929-936"},"PeriodicalIF":2.9000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12397974/pdf/","citationCount":"0","resultStr":"{\"title\":\"Current landscape of US Food and Drug Administration use of expedited regulatory approvals for colorectal cancer care.\",\"authors\":\"Danea M Horn, Mireille Jacobson, Kathryn A Phillips\",\"doi\":\"10.18553/jmcp.2025.31.9.929\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>\",\"PeriodicalId\":16170,\"journal\":{\"name\":\"Journal of managed care & specialty pharmacy\",\"volume\":\"31 9\",\"pages\":\"929-936\"},\"PeriodicalIF\":2.9000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12397974/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of managed care & specialty pharmacy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.18553/jmcp.2025.31.9.929\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of managed care & specialty pharmacy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.18553/jmcp.2025.31.9.929","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
Current landscape of US Food and Drug Administration use of expedited regulatory approvals for colorectal cancer care.
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.
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.
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