By Fineline Cube 
On August 20, 2025, the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) published a draft of the “Technical Guideline for Single-Arm Clinical Trials to Support Conventional Marketing Applications for Oncology Drugs” for public comment. The commenting period will last one month from the date of publication.
This guideline aims to expedite the market launch of new oncology drugs by allowing single-arm trials (SATs) to support conditional approvals under specific conditions. Unlike traditional randomized controlled trials (RCTs), SATs do not include parallel control groups and are typically open-label without randomization or blinding.
When Are Single-Arm Trials Acceptable?
In certain scenarios, SATs can be used as confirmatory studies for oncology drug approvals:
- Rare or Difficult-to-Study Indications: When an RCT is not feasible, such as in pan-tumor indications or rare cancers.
- Clear External Control Data: When historical or real-world data provide a reliable benchmark for comparison.
- Well-Understood Mechanism of Action: When the drug’s mechanism is clearly defined and supported by robust scientific evidence.
- Exceptional Efficacy: When preliminary results demonstrate remarkable clinical benefits.
Key Requirements for Single-Arm Trials as Confirmatory Studies
For an SAT to serve as a confirmatory study, it must meet two critical objectives:
- Survival Benefit Evaluation: The trial must demonstrate that the investigational drug provides a significant survival benefit to the target population through extended follow-up.
- Robustness of Results: The study must enroll a larger sample size to ensure the reliability and consistency of the findings.
Only when these criteria are met can the efficacy of the drug be confirmed, ensuring that observed benefits are not incidental but rather a result of the treatment.-Fineline Info & Tech