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Updated: Dec 22, 2025

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Investigator clinical trials: How can we ask the question?

Jean-François Schved1

  • 1Hemophilia Treatment Center, Montpellier University Hospital, Montpellier, France.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|May 2, 2020
PubMed

Insights

Investigator-initiated trials (IITs) are crucial for drug development but require precise research questions. This study highlights challenges and misinterpretation risks in IITs for hemorrhagic diseases.

Area of Science:

  • Clinical research methodology
  • Pharmacology and therapeutics
  • Hematology

Background:

  • Clinical trials are essential for advancing medical treatments.
  • Investigator-initiated trials (IITs) offer an alternative to industry-sponsored studies.
  • Defining clear research questions is critical for successful IITs.

Purpose of the Study:

  • To identify challenges in developing investigator-initiated trials.
  • To explore the risks of misinterpreting results from IITs.
  • To examine these issues through examples in hemorrhagic disease drug development.

Main Methods:

  • Retrospective analysis of three case studies in hemorrhagic disease drug development.
  • Qualitative assessment of trial design and execution in IITs.
  • Review of published results for potential misinterpretations.

Main Results:

  • IITs face unique design and execution hurdles.
  • Ambiguous research questions can lead to ambiguous or misleading results.
  • Specific examples from hemorrhagic disease research illustrate these difficulties.

Conclusions:

  • Careful formulation of research questions is paramount for IITs.
  • Awareness of potential misinterpretations is vital for accurate clinical decision-making.
  • Further research into optimizing IIT design and analysis is warranted.
Keywords:
clinical trialhemorrhagic disease

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