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.