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The proportion of North American cancer trials that evaluate novel targets
Eli Gumnit1, Aden C Feustel1, Sandy Wong1
1Biomedical Ethics Unit/Social Studies of Medicine, McGill University, 3647 Peel Street, Montreal, QC, H3A 1X1, Canada.
Abstract:
Major advances in cancer care often emerge from the development of novel targets. We randomly sampled 10% of cancer trials on clinicaltrials.gov with start dates 2013-2016 to determine the proportion of trials and research subjects directed at evaluating novel targets. We found that 87 of 378 trials (23.0%) enrolling 9225 of 44,525 patients (20.7%) tested interventions that are directed towards novel targets. 146 of 378 trials (38.6%) enrolling 19,132 of 44,525 patients (43.0%) investigated treatments that were not FDA approved but utilized a previously studied target for treating cancer. Combined, 233 of 378 trials (61.6%) enrolling 28,357 of 44,525 patients (63.9%) investigated treatments that were not FDA approved. Furthermore, 36 of 378 trials (9.5%) enrolling 6592 of 44,525 patients (14.8%) investigated FDA approved anticancer drugs in their approved indication and combination while 109 of 378 trials (28.8%) enrolling 9576 of 44,525 patients (21.5%) investigated FDA approved anticancer drugs outside of their approved indication or combination. Logistic regression found that phase 1 trials were significantly more likely to test novel target interventions than phase 2 and 3 trials (p value = 0.00197 and 0.00130 respectively). Industry sponsored trials were also significantly more likely to involve novel target interventions than non-industry trials (p value <0.001). In conclusion, most cancer trials involve unapproved treatments, but a majority of these treatments are well-characterized or involve a previously studied target to treat cancer.
Insights
Most cancer trials evaluate unapproved treatments, with over 20% targeting novel cancer pathways. Early-phase and industry-sponsored trials are more likely to explore these new therapeutic targets.
Area of Science:
- Oncology
- Clinical Trial Research
- Drug Development
Background:
- Novel therapeutic targets are crucial for advancing cancer care.
- Understanding the landscape of cancer trial interventions is essential for strategic research and development.
- The evaluation of novel targets versus established targets in clinical trials requires detailed analysis.
Purpose of the Study:
- To determine the proportion of cancer clinical trials and patient enrollment focused on novel therapeutic targets.
- To analyze the characteristics of trials investigating novel targets, including phase and sponsorship.
- To differentiate between novel target interventions and those using previously studied targets or approved drugs.
Main Methods:
- A random sample of 10% of cancer trials from clinicaltrials.gov (2013-2016) was analyzed.
- Trials were categorized based on whether they tested novel targets, previously studied targets, or FDA-approved drugs.
- Logistic regression analysis was used to identify factors associated with novel target interventions.
Main Results:
- 23.0% of trials and 20.7% of patients were involved in evaluating novel cancer targets.
- 61.6% of trials and 63.9% of patients investigated non-FDA-approved treatments.
- Phase 1 trials and industry-sponsored trials showed a significantly higher likelihood of testing novel targets.
Conclusions:
- The majority of cancer clinical trials involve unapproved treatments.
- A significant portion of these trials focus on novel targets, particularly in early phases and industry-funded studies.
- While many trials use unapproved treatments, a substantial number involve well-characterized or previously studied targets.
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