作者: Martin Kolb , Gisli Jenkins , Luca Richeldi
DOI: 10.1136/THORAXJNL-2016-208670
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摘要: Predicting the future is one of greatest challenges and for many people hopes humanity. This applies to any aspect human life medicine included. In respiratory medicine, predicting particularly difficult chronic remodelling disorders, such as pulmonary hypertension or fibrosis.1 The course recovery from an acute illness are usually easier foresee than progression rate decline diseases. particular, major current actually effect available pharmacological treatments on idiopathic fibrosis (IPF), which paramount importance but still rarely possible. Nonetheless, more we enter era so-called personalised , anticipating response a specific drug becoming part realistic expectations.2 Safety efficacy drugs assessed in context placebo-controlled randomised clinical trials (RCTs). Although well-established worldwide accepted methodology, RCTs have limitations: these fact that necessarily last definite period time, IPF typically 12 months, during time all participants blinded active treatment placebo. limitation intrinsic unavoidable, given need balance between harm benefit when new with unknown effects tested patients. However, once approved, undergo mandatory postapproval surveillance several years. While this type postmarketing provides valid information about long-term safety drugs, there no formal way assessing efficacy, even if studies report they never controlled therefore evidence base less rigorous prospective trials. For reason, other forms research may be used inform …