A map of molecular drug targets and therapeutics for the US FDA-approved drugs: The impact of expedited regulatory pathways and first-in-class drug approvals on drug innovation.
Level 4 - case-series / case-control
Cross-sectional descriptive analysis of regulatory approvals (by design analogy, non-clinical CEBM)
PubMed 41187816 · doi:10.1016/j.pharmthera.2025.108945
What was done
The authors mapped molecular targets, therapeutic categories, and regulatory approval pathways for 465 drugs approved by the US FDA between 2015 and 2024. They quantified the proportions of new molecular entities (NMEs), biologics, orphan designations, first-in-class mechanisms, target protein classes, and regular versus expedited review pathway utilization.
What was found
Among 465 approved drugs: - 71% were NMEs and 29% were biologics. - 50% targeted orphan diseases and 41% were first-in-class agents. - Primary target classes were enzymes (17%), kinases (16%), GPCRs (12%), transporter proteins (4%), and nuclear receptors (3.7%). - 67% utilized at least one expedited review program, with >70% of those using multiple pathways; priority review was most frequent and accelerated approval was least frequent. - Oncology drugs had the highest expedited pathway utilization (80%–100%). - By disease category, approvals included cancer (29%), cardiovascular diseases (8.6%), respiratory illnesses (4.3%), and diabetes (3%).
Why it matters
This review provides an empirical 10-year summary of US regulatory trends, demonstrating that expedited mechanisms and orphan indications have become the standard path for most newly approved therapeutics.
Limits
The study is restricted to US FDA approvals from 2015–2024 without comparison to international agencies (e.g., EMA). The abstract provides descriptive registry-level proportions but does not assess post-market clinical efficacy, safety endpoints, or the completion rates of confirmatory trials for drugs granted accelerated approval.
Cited by
- context There are approximately 4,000 FDA-approved drugs approved for about 4,000 human diseases.