viernes, 26 de mayo de 2017

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 | Orphanet Journal of Rare Diseases | Full Text

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 | Orphanet Journal of Rare Diseases | Full Text

Biomed Central



Orphanet Journal of Rare Diseases

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

  • Florian LaschEmail author,
  • Kristina Weber,
  • Mwe Mwe Chao and
  • Armin Koch
Contributed equally
Orphanet Journal of Rare Diseases201712:102
DOI: 10.1186/s13023-017-0655-8
Received: 6 March 2017
Accepted: 16 May 2017
Published: 25 May 2017

Abstract

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. We highlight some major problems when research is based on single-arm trials compared to randomized controlled trials. After debunking common arguments for the conduct of single-arm trials in rare disease we conclude that particularly in rare disease research should be based on randomized building blocks simply because more robust evidence is generated. 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.

Keywords

Randomization Single-arm trial RCT Rare disease Historical control Fanconi anemia Study planning Evidentiary standards

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