Article
J. Mulder, S. Teerenstra, P. B. van Hennik, A. M. G. Pasmooij, V. Stoyanova-Beninska, E. E. Voest & A. de Boer
Early phase clinical trial played a critical role in the Food and Drug Administration-approved indications for targeted anticancer drugs: a cross-sectional study from 2012 to 2021
Summary
- Analysis of 188 FDA-approved indications for 95 molecular targeted anticancer drugs between 2012 and 2021.
- 59.6% of indications were approved based on Early Phase Clinical Trials (EPCTs).
- There was a notable annual increase of 22.2% in approvals based on EPCTs, compared to a 5.0% increase for phase three RCTs.
- EPCTs provide essential preliminary efficacy and safety evidence, contributing to the accelerated development of targeted therapies.
- 32 of 112 indications approved based on EPCTs were derived from Dose Expansion Cohort (DEC) trials, showing a significant annual increase of 29.7%.
- DEC trials are favored for their efficiency and simplified design, often leading to accelerated approvals and priority reviews.
- Phase 2 Single-Arm Trials (SATs) accounted for 67.0% of EPCT-based approvals, with a significant annual increase of 18.7% from 2017 to 2021.
- EPCTs, particularly DEC trials and SATs, are beneficial for rare cancer subtypes and drugs showing dramatic effects.
- Challenges remain with EPCTs, including potential biases and the need for further methodological improvements and real-world RCTs to confirm clinical benefits.
- The study emphasizes the growing importance and application of EPCTs in the context of precision oncology.
- Limitations include reliance on FDA data and the study's focus on approvals within the past decade.
With the development of precision oncology, molecular targeted anticancer drug has become the most attractive research field in new drug development A lot of time, money and resources have been invested in developing novel targeted anticancer drugs. More and more blockbuster drugs have been approved by the US Food and Drug Administration (FDA) in the past decades. However, historical data showed that only less than 10% of new drug applications (NDAs) could get approvals from the FDA.
Most of NDAs failed in providing sufficient efficacy and safety data in clinical trials and thus solid evidence from pivotal clinical trial is the key point in the application of drug approvals...
Early phase clinical trial, Dose-expansion cohort, Single-arm trial, Pivotal trial, FDA approved indications, Targeted anticancer drugs
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