FAST-TRACKING RARE DISEASES: USE OF FDA EXPEDITED PATHWAYS FOR ORPHAN DRUGS
Author(s)
Sam Dean, MBA, Kristen Cribbs, MPH, PhD;
Alkemi LLC, Manchester Center, VT, USA
Alkemi LLC, Manchester Center, VT, USA
OBJECTIVES: Regulatory designations for orphan conditions and expedited review pathways are designed to accelerate access for innovative therapies. This study examined how frequently expedited review pathways are used for orphan-designated drugs over the past decade.
METHODS: We searched FDA databases to identify orphan drugs with FDA indication approval via an expedited pathway (i.e., Priority Review, Fast Track, Breakthrough Therapy, Accelerated Approval) between January 1, 2015, and December 31, 2024. For identified drug indications, we reviewed FDA letters to abstract data on approval status, drug type, therapeutic area, and expedited pathway. Descriptive analyses were conducted.
RESULTS: Among the 338 orphan indications approved during the study period, 184 were approved via an expedited pathway (54.5%), encompassing 148 unique drugs. Expedited pathway approvals were most frequent in 2016 (14.7%; 27/184) and 2017 (14.1%; 26/184). Most approved indications were small molecules (125/184; 67.9%), followed by biologics (40/184; 21.7%) and nucleic acid/RNA therapies or monoclonal antibodies (19/184; 10.3%). The most common disease areas were blood and circulatory disorders (59/184; 32.1%), brain and nervous system disorders (19/184; 10.3%), and immune system disorders (18/184; 9.8%). Among expedited indications, the majority (82.6%; 152/184) were reviewed under >1 pathway (average 2.36 pathways per indication). Of indications with a single pathway (17.4%, 32/184), Priority Review was the most common (59.4%, 19/32), followed by Breakthrough Therapy (6/32; 18.8%), Accelerated Approval (5/32; 15.6%), and Fast Track (2/32; 6.3%).
CONCLUSIONS: Over the past decade, more than half of orphan drug indications were approved through an FDA expedited pathway, with Priority Review used most frequently. Future analysis should assess the impact of these regulatory incentives on drug development timelines and access.
METHODS: We searched FDA databases to identify orphan drugs with FDA indication approval via an expedited pathway (i.e., Priority Review, Fast Track, Breakthrough Therapy, Accelerated Approval) between January 1, 2015, and December 31, 2024. For identified drug indications, we reviewed FDA letters to abstract data on approval status, drug type, therapeutic area, and expedited pathway. Descriptive analyses were conducted.
RESULTS: Among the 338 orphan indications approved during the study period, 184 were approved via an expedited pathway (54.5%), encompassing 148 unique drugs. Expedited pathway approvals were most frequent in 2016 (14.7%; 27/184) and 2017 (14.1%; 26/184). Most approved indications were small molecules (125/184; 67.9%), followed by biologics (40/184; 21.7%) and nucleic acid/RNA therapies or monoclonal antibodies (19/184; 10.3%). The most common disease areas were blood and circulatory disorders (59/184; 32.1%), brain and nervous system disorders (19/184; 10.3%), and immune system disorders (18/184; 9.8%). Among expedited indications, the majority (82.6%; 152/184) were reviewed under >1 pathway (average 2.36 pathways per indication). Of indications with a single pathway (17.4%, 32/184), Priority Review was the most common (59.4%, 19/32), followed by Breakthrough Therapy (6/32; 18.8%), Accelerated Approval (5/32; 15.6%), and Fast Track (2/32; 6.3%).
CONCLUSIONS: Over the past decade, more than half of orphan drug indications were approved through an FDA expedited pathway, with Priority Review used most frequently. Future analysis should assess the impact of these regulatory incentives on drug development timelines and access.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR145
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
SDC: Rare & Orphan Diseases