[Considerations for anti-cancer drug application by single arm trials]

Area of Science:

• Oncology
• Drug Development
• Clinical Trials

Background:

• Single arm trials (SAT) are increasingly utilized for novel anti-cancer drug New Drug Applications (NDAs).
• SAT offers a significantly shorter timeline for drug approval compared to traditional randomized controlled trials (RCTs).
• Pharmaceutical companies are motivated to pursue SAT for expedited market entry of anti-cancer agents.

Purpose of the Study:

• To review the background and critical considerations of using SAT for anti-cancer drug NDAs.
• To discuss the criteria for accepting SAT in the NDA process.
• To systematically explain the design and underlying principles of SAT.

Main Methods:

• Literature review of SAT in anti-cancer drug development.
• Analysis of regulatory trends and acceptance criteria for SAT in NDAs.
• Systematic elaboration on SAT design and principles.

Main Results:

• SAT has demonstrably shortened the time to market for new anti-cancer drugs.
• Key issues and conditions for SAT acceptance in NDAs are identified.
• Principles for effective SAT design are outlined.

Conclusions:

• SAT is a viable strategy to accelerate the development and approval of innovative anti-cancer therapies.
• Understanding the specific conditions and design principles of SAT is crucial for successful regulatory submissions.
• This review provides a comprehensive guide for leveraging SAT in oncology drug development.