Development of a Unique Tool for Assessing the Feasibility of an External Control Arm Study
Background: External control arms (ECAs) are used to generate comparative evidence (e.g., effectiveness) when only a single-arm trial has been conducted during the drug development process. However, key stakeholders often face substantial delays in determining the feasibility of an ECA; a responsive tool could streamline this process in a structured, rigorous manner
Methods: A comprehensive review was conducted to identify ECA guidelines and reflection papers from major agencies, including the FDA and EMA, as well as other literature outlining best practices for conducting ECAs. Based on this, a structured questionnaire was developed to evaluate general topics, data feasibility, and design feasibility, incorporating skip logic where appropriate. A checklist was then created to distinguish between supportive and challenging factors for ECA feasibility, helping users identify potential gaps during strategic planning. The collected responses were summarized in a report that included recommended advice derived from relevant literature and expert insights. To support this process, the ECA Questionnaire Tool was implemented as an interactive web application built with a modern JavaScript framework and a C# backend connected to a SQL database. This setup enables dynamic question logic, progress tracking, automated report generation, secure data storage, real-time feedback, and seamless integration of feasibility checklists and reporting features.
Results: ECA Feasibility Questionnaire: A total of 24 questions, including two general questions, 13 data feasibility questions, and nine design feasibility questions, were developed. Following the completion of the questionnaire, the status of all feasibility questions is summarized for user review, and a comprehensive explanation of all responses, along with associated guidance, is then generated. A user-friendly electronic tool to assess the feasibility of an ECA across multiple domains can de-risk the evidence generation process, saving time and financial resources; the tool is available in GIPAM’s EVIGATOR platform or independently.