PubMed · 16609277
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Mary E Duffy. The randomized controlled trial: basic considerations.. https://doi.org/10.1097/00002800-200603000-00004
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Much has been written recently about low productivity in the pharmaceutical industry and the high cost of drug development. Over a 10-year period ending in 2000, only approximately 11% of compounds tested in humans across 10 large pharmaceutical companies were eventually approved for marketing in the United States and/or Europe. Attrition was highest during phase II (62%) but still significant in phase III (45%) and at the time of registration (23%). Clearly, given the high cost and time required for clinical development, these late-stage failures are unsustainable.
Functional human proteins are constitutively produced in genetically modified bacteria that survive on human mucosal surfaces, to the benefit of the host. The successful Phase I clinical trial with IL-10-producing Lactococcus lactis for Crohn's disease has opened new avenues for the use of transgenic bacteria as delivery vehicles. The major advantage of this novel strategy is the avoidance of systemic side effects associated with conventional therapies. This methodology opens up an alternative method for local delivery of therapeutic proteins to various mucosal tissues.
In this paper, rules are given on how to construct escalation, A + B, and group designs for dose-finding trials. Operational characteristics of these designs are discussed and compared via simulations. Dose-finding designs for trials with ordinal toxicity outcome are considered.