Abstract
There is a potential tension between the desirable goals of assuring early availability of promising agents for treatment of serious illnesses without adequate therapy and developing such agents in a rigorous way that will yield scientifically valid answers about their safety and effectiveness. Because the two goals are linked to fundamental principles and values, it is possible for people who feel strongly about one of them to ignore the importance of the other. But it is neither necessary nor desirable to choose one goal exclusively; Early access is reasonable on both ethical and medical grounds and can be provided, with care and planning, without degrading the quality of evidence we develop to support new agents. A clear picture of the effectiveness and safety of a drug, and information on how best to use it is most critical, not to academics and regulators, but to the patients whose lives and health may depend on the drug, and obtaining this information does not require that access be denied until it is all in hand.
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