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The Evidence Base Post

Beyond external control arms: Leveraging real-world data to support regulatory applications in oncology

  • The Evidence Base

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Randomized controlled trials are the gold standard for clinical research; however, in many areas, like oncology, they are infeasible because new therapies often target rare tumors and, therefore, the indicated population is small. Single-arm trials are frequently used as a feasible alternative to evaluate a treatment's effectiveness without a traditional control group. The interpretation of the results from a single-arm trial can be supported by a real-world external control arm (ECA)—where real-world data (RWD) are used to estimate the trial outcome rate in a group of patients who have not received the investigational treatment for comparison with the outcome rate observed in the single-arm trial. ECAs have been used successfully to support FDA approval of rare disease therapies and are increasingly being explored in clinical development. In oncology applications, however, FDA reviewers have found their utility to be limited. Given this, can RWD be useful in supporting oncology regulatory applications, beyond the use in ECAs?

During the 2024 Drug Information Association Real-World Evidence (DIA RWE) Conference (October 24–25, 2024, Philadelphia, PA, USA), Ulka Campbell (Head of Scientific Strategy, Aetion, Inc., USA) explored this topic in the presentation, “Beyond External Control Arms: Utility of Real-World Data in Oncology Regulatory Applications.”

Here, we take a Deep Dive into the session, summarizing the key discussions and main takeaways from the oncology case studies illustrating how RWD can support regulatory decision-making, such as rationalizing trial design and assuring the representativeness of trial populations.

Case studies discussed:

  1. Previously published RWD analysis demonstrates lack of spontaneous remission of disease
  2. Previously published RWD analysis supports endpoint threshold and size of pivotal trial
  3. New RWD analysis demonstrates unmet need
  4. New RWD analysis provides benchmarks for assessing representativeness of trial population
  5. New RWD analysis informs pivotal trial sample size
  6. New RWD analysis supports the rationale for a non-randomized pivotal trial
READ THE FULL ARTICLE HERE

Sponsorship for this Deep Dive was provided by Aetion