FDA uses expedited regulatory pathways (ERPs) in order to accelerate the availability of drugs and diagnostic tests for severe conditions that have unmet medical needs. How often is this process used and has this changed over time? A recent paper by Horn et al. (2025) provides the answer as applied to colorectal cancer (CRC) drugs. First, they provide a summary of the legislative changes that allowed for FDA’s use of ERPs.
Under ERPs, the FDA reduces review time and allows for more frequent developer interactions. The first ERP, Priority Review, started in 1992 under the Prescription Drug User Fee Act (PDUFA). PDUFA allowed the FDA to collect fees from drug sponsors to reduce regulatory review time, more than tripling the rate of drugs approved in the United States from the initial year of the PDUFA. Since then, ERPs have evolved and expanded. The most significant change came from the 2012 FDA Safety and Innovation Act, which created the Breakthrough Therapy Designation and allowed for the use of surrogate endpoints, or outcomes that can “reasonably” predict clinical benefit (eg, progression-free survival). Development and approval time of therapeutics approved through ERPs can be 30% faster than normal approval pathways.
Then, the authors examine to what extent the use of ERPs has changed over time as applied to CRC drugs:
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.
You can read the full paper here.
