The Center for Drug Evaluation (CDE) has released the “Technical Guidelines for the Applicability of Single-arm Clinical Trials to Support the Market Filing for Antitumor Drugs.” The document provides clarity on the limitations of single-arm clinical trials, their applicability, and the requirements for confirmatory clinical trials in support of both conditional and regular market filings. The aim is to guide companies in assessing whether to conduct a single-arm clinical study as a pivotal study supporting market filing after early clinical trials.
Changing Landscape and Risk Assessment
The guidelines note that as more innovative drugs enter the market, some tumors are increasingly being treated as chronic diseases. Consequently, the acceptable level of uncertain risks in single-arm clinical trials is evolving. When using single-arm research to support drug market filing, its application conditions must be strictly controlled to ensure safety and efficacy.
Applicability Criteria
The document specifies that a single-arm clinical trial can serve as the pivotal regulatory study under the following conditions:
- The study population has no effective treatment option.
- The mechanism of action of the drug is clear.
- External control efficacy data for the indication is clear.
- The drug’s efficacy is outstanding.
- The security risk is controllable.
- The trial involves rare tumors.
These guidelines are designed to help companies navigate the complexities of clinical trial design and ensure that single-arm trials are used appropriately in the development of antitumor drugs.-Fineline Info & Tech
