Development of a Unique Tool for Assessing the Feasibility of an External Control Arm Study
Author(s)
Jason Simeone, MS, PhD1, Thomas Wilke, PhD2.
1GIPAM GmbH, Winchester, MA, USA, 2GIPAM GmbH, Wismar, Germany.
1GIPAM GmbH, Winchester, MA, USA, 2GIPAM GmbH, Wismar, Germany.
OBJECTIVES: 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 performed to identify ECA guidelines from relevant agencies and other literature describing best practices for the conduct of an ECA. A structured questionnaire was first developed to assess general topics, data feasibility and design feasibility. Questions were coded with skip logic when appropriate. A checklist indicating positive (i.e., supportive of the ECA feasibility) and negative (i.e., challenges to the ECA feasibility) responses with a report summarizing the responses and recommended advice per question based on expertise from relevant literature was generated.
RESULTS: A total of 24 questions (2 general questions, 13 data feasibility questions, and 9 design feasibility questions) were developed. Sample questions included “General: Have you reviewed relevant previous ECA submissions to the regulatory or Health Technology Assessment agency that you are preparing to submit to?”, “Data feasibility: Are key patient attributes that may be associated with study outcomes such as relevant clinical characteristics (e.g., disease severity) captured in your selected data source?”, and “Design feasibility: Have you identified validated algorithms (e.g., using diagnosis/procedure codes) for identifying the study sample in a comparable way to the trial?”. The structured questionnaire, checklist, and summary report were developed in an interactive platform designed for all key stakeholders involved in the management of evidence generation for drug development.
CONCLUSIONS: A user-friendly electronic tool to assess the feasibility of an ECA can de-risk the evidence generation process, saving resources. This process can quickly identify gaps for focus during a full feasibility assessment.
METHODS: A comprehensive review was performed to identify ECA guidelines from relevant agencies and other literature describing best practices for the conduct of an ECA. A structured questionnaire was first developed to assess general topics, data feasibility and design feasibility. Questions were coded with skip logic when appropriate. A checklist indicating positive (i.e., supportive of the ECA feasibility) and negative (i.e., challenges to the ECA feasibility) responses with a report summarizing the responses and recommended advice per question based on expertise from relevant literature was generated.
RESULTS: A total of 24 questions (2 general questions, 13 data feasibility questions, and 9 design feasibility questions) were developed. Sample questions included “General: Have you reviewed relevant previous ECA submissions to the regulatory or Health Technology Assessment agency that you are preparing to submit to?”, “Data feasibility: Are key patient attributes that may be associated with study outcomes such as relevant clinical characteristics (e.g., disease severity) captured in your selected data source?”, and “Design feasibility: Have you identified validated algorithms (e.g., using diagnosis/procedure codes) for identifying the study sample in a comparable way to the trial?”. The structured questionnaire, checklist, and summary report were developed in an interactive platform designed for all key stakeholders involved in the management of evidence generation for drug development.
CONCLUSIONS: A user-friendly electronic tool to assess the feasibility of an ECA can de-risk the evidence generation process, saving resources. This process can quickly identify gaps for focus during a full feasibility assessment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA109
Topic
Health Technology Assessment, Organizational Practices, Study Approaches
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas