Probability of Regulatory Approval Over Time: A Cohort Study of Cancer Therapies
Nora Hutchinson1, Selin Bicer2, Emma Feldhake2
1Division of Hospital Medicine, University of California, San Francisco, CA.
Purpose:
New cancer therapies are frequently evaluated in multiple disease indications. We evaluated whether the probability of achieving US Food and Drug Administration (FDA) approval for a new cancer therapy changes with time.
Methods:
We identified a cohort of anticancer drugs with a first registered efficacy trial from 2007 to 2011 on ClinicalTrials.gov. We downloaded all clinical trials for each included drug from the initiation of efficacy testing to January 11, 2021. Each trial was categorized by cancer indication and assigned to investigational trajectories on the basis of unique drug-indication pairings. We performed a univariate Cox's proportional hazards regression to assess the probability of a trajectory leading to regulatory approval over time since initiation of the first efficacy trial for a given drug.
Results:
We included 213 drugs in our cohort, of which 37 (17.4%) received FDA approval in at least one oncology indication. In our primary analysis, we found a 15% decrease in the probability of approval for every year since initiation of the first efficacy trial (hazard ratio [HR], 0.85 [95% CI, 0.73 to 0.99]; P = .032). We found a 45% increase in the probability of approval for the first trajectory launched for a given drug in comparison with all others (HR, 0.55 [95% CI, 0.33 to 0.91]; P = .021).
Conclusion:
Drug-indication pairings pursued years after initial testing for efficacy have lowered probability of affecting care. Clinical trial investigators, sponsors, and regulatory bodies may benefit from awareness of this trend when considering both early and late trajectory trials in a drug's development.
Insights
The likelihood of new cancer drugs gaining US Food and Drug Administration (FDA) approval decreases annually. Early drug development trajectories have a higher chance of success compared to later ones.
Area of Science:
- Oncology
- Drug Development
- Regulatory Science
Background:
- New cancer therapies undergo evaluation across multiple disease indications.
- Understanding factors influencing regulatory approval is crucial for drug development strategy.
Purpose of the Study:
- To determine if the probability of US Food and Drug Administration (FDA) approval for novel cancer therapies changes over time.
- To analyze the impact of development timelines on the success rate of oncology drug approvals.
Main Methods:
- A cohort of 213 anticancer drugs with initial efficacy trials between 2007-2011 was identified.
- Clinical trial data up to January 2021 was analyzed, categorizing trials by cancer indication and drug-indication pairings.
- Univariate Cox's proportional hazards regression assessed the probability of regulatory approval over time since the first efficacy trial.
Main Results:
- 17.4% of the evaluated drugs received FDA approval in at least one oncology indication.
- A 15% decrease in the probability of approval was observed for each year since the initiation of the first efficacy trial (HR, 0.85; P = .032).
- The first drug-indication trajectory showed a 45% increased probability of approval compared to subsequent trajectories (HR, 0.55; P = .021).
Conclusions:
- Drug-indication pairings explored later in the development process have a reduced likelihood of impacting patient care.
- Awareness of these trends can inform clinical trial investigators, sponsors, and regulatory bodies when planning early and late-stage drug development.
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