Single-arm studies with external comparators for cancer drug development: a statistical methodology study to evaluate External Comparator Arm (ECA) study results versus Randomised Controlled Trial (RCT) results

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/50418
• EU PAS number:
  EUPAS43709
• Study ID:
  50418
• Official title and acronym:
  Single-arm studies with external comparators for cancer drug development: a statistical methodology study to evaluate External Comparator Arm (ECA) study results versus Randomised Controlled Trial (RCT) results
• DARWIN EU® study:
  No
• Study countries:
  Netherlands
• Study description:
  Randomised controlled trials (RCTs) are generally considered by regulators to be the gold standard for establishing a causal relationship between medications and patient outcomes. Several new clinical trial designs have been introduced in oncology drug development based on clinical scientific insights. This is related to the fact that for many cancers, or their (biomarker-selected) subtypes, the target populations are relatively small and/or there is a high unmet medical need. An example of this are trial designs without a parallel randomised control group, such as single-arm trials (SATs), which can be contextualised by providing an external comparator group. The purpose of this statistical methodology study is to advance the knowledge around using SATs with external comparators for cancer drug development, and to develop recommendations on the best practice to incorporate external comparators in the analysis. The results of this study, using data from RCTs and real-world data (RWD), are intended to contribute to the development of recommendations regarding methodologies to be applied and characteristics of situations where evidence generated from a SAT with an external comparator can form a basis for drug approval.
• Study status:
  Finalised

Research institutions and networks

Institutions

IQVIA

United Kingdom

Study timelines

Date when funding contract was signed

Planned: 23/11/2020

Actual: 23/11/2020

Study start date

Planned: 01/04/2021

Actual: 16/07/2021

Date of final study report

Planned: 01/09/2022

Actual: 14/11/2022

Sources of funding

EMA

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable