منابع مشابه
Clinical trials in rare diseases: a review of practice
Background The evaluation of treatments for rare diseases presents a number of challenges for trial practitioners, regulators and policy makers. Small sample sizes mean that ‘standard’ approaches to trial design and analysis may not be appropriate and alternatives such as Bayesian trial designs have been recommended [Lilford et al 1995]. However, little is known about the design and analysis ap...
متن کاملA Bayesian adaptive design for clinical trials in rare diseases
Development of treatments for rare diseases is challenging due to the limited number of patients available for participation. Learning about treatment effectiveness with a view to treat patients in the larger outside population, as in the traditional fixed randomised design, may not be a plausible goal. An alternative goal is to treat the patients within the trial as effectively as possible. Us...
متن کاملRare disease clinical trials
The limb-girdle muscular dystrophies (LGMDs) encompass a collection of genetic muscle diseases with proximal-predominant weakness of the limbs. Thirty-two of these disorders are named via the common nomenclature, including 8 autosomal-dominant (LGMD1A-H) and 24 autosomal-recessive (LGMD2A-X) disorders.(1) In addition, numerous other genetic muscle diseases, including Bethlem myopathy, dystrophi...
متن کاملA comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov
OBJECTIVES To provide a comprehensive characterisation of rare disease clinical trials registered in ClinicalTrials.gov, and compare against characteristics of trials in non-rare diseases. DESIGN Registry based study of ClinicalTrials.gov registration entries. METHODS The ClinicalTrials.gov registry comprised 133,128 studies registered to September 27, 2012. By annotating medical subject he...
متن کاملClinical Applicability of Stem Cell Therapy in Dental Diseases: A Systematic Review of Available Clinical Trials
Background: Developing cell-based therapeutic methods for the differentiation of various stem cells to the dental tissues could be a revolutionary step towards the use of cell therapy for dental disease. In the present study, we aimed to systematically review the literature to establish the efficiency of stem cells in treatment of various dental diseases. Methods: A literature search was perfo...
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ژورنال
عنوان ژورنال: BMJ
سال: 1996
ISSN: 0959-8138,1468-5833
DOI: 10.1136/bmj.312.7035.910a