Abstract
Randomized controlled trials (RCTs) are considered gold standard to determine the efficacy of a new therapy, however, Health Technology Assessment (HTA) bodies recognize situations when this is not possible. For drugs targeting a small patient population and/or in therapeutic areas of high unmet need, the use of single-arm trials, accompanied by indirect treatment comparisons, as the main evidence for HTA submissions is being used as an alternative. This study aims to explore these trends in Portugal and compare them to other European countries.
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Schedule a callMore From: Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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