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Can the US Dodge a Recession? This Economist Thinks So - Bloomberg
“In science we use models all the time, and they’re simplifications of reality,” he said. “And part of the skill of the scientist is to know when to deploy the model and when not to or, in other words, to know the limitations of the model. And maybe I’m in a good position of knowing the limitations, given that it’s my model.”
Statistical considerations for rare diseases drug development. - PubMed - NCBI
One of the most challenges for rare disease clinical trials is probably the availability of a small patient population. It is then a great concern on how to conduct clinical trials with a small number of subjects available for obtaining substantial evidence regarding safety and effectiveness for approval of the rare disease drug product under investigation. FDA, however, does not have the intention to create a statutory standard for approval of orphan drugs that are different from the standard for approval of drugs in common conditions. Thus, it is suggested that innovative trial designs such as a complete n-of-1 trial design or an adaptive design should be used for an accurate and reliable assessment of rare disease drug products under investigation. In this article, basic considerations, innovative trial designs, and statistical methods for data analysis are discussed. In addition, some innovative thinking for the evaluation of rare disease drug products is proposed.