Rare diseases and the assessment of intervention: What sorts of clinical trials can we use?
There is increasing emphasis on the importance of practising evidence-based medicine. Randomized controlled trials are the standard way to assess the bene¢ts of an intervention, and observational studies are not usually accorded much weight; the results are likely to be considered misleading. For...
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| Formato: | Artículo |
| Lenguaje: | Inglés |
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Universidad de Belgrano - Documentos CEEGMD - Centro para el estudio de enfermedades genéticas, metabólicas y discapacidades. Facultad de Ciencias Exactas
2014
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| Acceso en línea: | http://repositorio.ub.edu.ar/handle/123456789/2886 |
| Aporte de: |
| Sumario: | There is increasing emphasis on the importance of practising
evidence-based medicine. Randomized controlled trials are the standard way
to assess the bene¢ts of an intervention, and observational studies are not usually
accorded much weight; the results are likely to be considered misleading. For rare
diseases, there are great dif¢culties in obtaining adequate evidence for
interventions or for the bene¢ts of early diagnosis. This is because the disorders
are not only very rare but also have variable expression, may have very long
courses, and have incompletely known late effects; and surrogate end-points
often have to be used. Randomized controlled trials are usually impossible
because of inadequate power, and because there are preconceived notions of
the effects of treatments already in use. The adoption of the best possible design
for observational trials, formation of a central registry of such trials, and a
greater general appreciation of the problems that rare diseases pose will help
in obtaining the best possible evidence for the effects of interventions. |
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