gms | German Medical Science

62. Jahrestagung der Deutschen Gesellschaft für Medizinische Informatik, Biometrie und Epidemiologie e. V. (GMDS)

Deutsche Gesellschaft für Medizinische Informatik, Biometrie und Epidemiologie

17.09. - 21.09.2017, Oldenburg

A plea to provide best evidence in trials under sample-size restrictions: the example of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients

Meeting Abstract

Suche in Medline nach

  • Florian Lasch - Medizinische Hochschule Hannover, Hannover, Deutschland
  • Kristina Weber - Medizinische Hochschule Hannover, Hannover, Deutschland
  • Mwe Mwe Chao - Medizinische Hochschule Hannover, Hannover, Deutschland
  • Armin Koch - Medizinische Hochschule Hannover, Hannover, Deutschland

Deutsche Gesellschaft für Medizinische Informatik, Biometrie und Epidemiologie. 62. Jahrestagung der Deutschen Gesellschaft für Medizinische Informatik, Biometrie und Epidemiologie e.V. (GMDS). Oldenburg, 17.-21.09.2017. Düsseldorf: German Medical Science GMS Publishing House; 2017. DocAbstr. 274

doi: 10.3205/17gmds053, urn:nbn:de:0183-17gmds0536

Veröffentlicht: 29. August 2017

© 2017 Lasch et al.
Dieser Artikel ist ein Open-Access-Artikel und steht unter den Lizenzbedingungen der Creative Commons Attribution 4.0 License (Namensnennung). Lizenz-Angaben siehe http://creativecommons.org/licenses/by/4.0/.


Gliederung

Text

Einleitung: In planning a clinical trial for demonstrating the efficacy of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients we had to discuss the need for a randomized controlled trial particularly under sample-size restrictions as very promising results were available from a single-arm clinical trial. Unfortunately, at a later stage, we had to suffer from the fact that single-arm clinical trials may sometimes mislead. When revisiting our planning at a later stage of a grant application, results of a randomized controlled trial had become available which were less impressive, but may still be of clinical interest. However, these results were perceived as disappointing in the light of previously raised hopes based on the results of the single-arm trial and research was stopped.

Methoden: Based on this example we highlight some major problems when research is based on single-arm trials compared to randomized controlled trials and debunk common arguments for the conduct of single-arm trials in rare disease. We contrast a single-arm based research strategy with a decision making strategy based on RCTs, which can be viewed (and used) as Lego®-type building blocks.

Ergebnisse: The assumption of knowing the counterfactual is easily violated in single-arm trials without possibility of testing this assumption. Undocumented and undetected patient selection is one of the main downfalls of single-arm trials. Contrary to the common opinion, addressing a new research question with single-arm trials requires more patients in the long run than starting with a (small) RCT from the beginning.

Diskussion: Particularly in rare disease research should be based on randomized building blocks because more robust evidence is generated: unbiased estimates of the treatment effect, avoidance of undocumented selection of patients and evidence-synthesis requiring fewer assumptions. The plea for single-arm trials should be substituted by a plea for cooperation of all stakeholders to provide best evidence for decision making under sample-size restrictions.



Die Autoren geben an, dass kein Interessenkonflikt besteht.

Die Autoren geben an, dass kein Ethikvotum erforderlich ist.