Author's Reply

Jul 1, 2022, 00:00 AM
Section Order : 1256
First Page : 1256
We wish to thank the commentators for their time and effort in providing valuable feedback to our study.
The purpose of our study was intended to describe the landscape and outcomes of health technology assessment (HTA) submissions of products launched based on single-arm trials. In this context, we present the general trends among the use of external comparators (ECs), type of EC, and HTA outcomes. Due to concerns over potential bias resulting from observed and unobserved confounding factors, we did not present any significance tests for the use of real-world data (RWD) ECs and positive HTA outcomes. For example, we mention that HTA submissions involving drugs with orphan designation have different threshold for evidence requirements to non-orphan drugs. Consequently, we do see lower difference in positive outcomes for orphan drugs with and without RWD ECs than non-orphan drugs. We intended to be clear in writing this article that we do not to provide a causal link between the use of RWD or EC data and the probability of a successful submission, and we would like to reiterate this point.
HEOR Topics :
  • Clinical Trials
  • Confounding, Selection Bias Correction, Causal Inference
  • Methodological & Statistical Research
  • Study Approaches
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Regions :
  • Global