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
| Autor: | Kristina Weber, Florian Lasch, Armin Koch, Mwe Mwe Chao |
|---|---|
| Rok vydání: | 2017 |
| Předmět: |
medicine.medical_specialty
Randomization Clinical Decision-Making lcsh:Medicine law.invention 03 medical and health sciences Single-arm trial 0302 clinical medicine Plea Randomized controlled trial law Fanconi anemia medicine Humans Hypoglycemic Agents Pharmacology (medical) Intensive care medicine Position Statement Genetics (clinical) Randomized Controlled Trials as Topic Erythroplakia Evidence-Based Medicine Pioglitazone business.industry lcsh:R General Medicine medicine.disease Study planning 3. Good health Clinical trial Evidentiary standards Fanconi Anemia Historical control ddc: 610 Sample size determination 030220 oncology & carcinogenesis Sample Size Thiazolidinediones Biometrie business Rare disease 030217 neurology & neurosurgery Leukoplakia RCT |
| Zdroj: | Orphanet Journal of Rare Diseases Orphanet Journal of Rare Diseases, Vol 12, Iss 1, Pp 1-6 (2017) 62. Jahrestagung der Deutschen Gesellschaft für Medizinische Informatik, Biometrie und Epidemiologie e.V. (GMDS); 20170917-20170921; Oldenburg; DOCAbstr. 274 /20170829/ |
| ISSN: | 1750-1172 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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