Use of "Efficacy" and "Effectiveness" Often Misleading and May Skew Reimbursement Decisions - Presented at ISPOR Europe 2012

A recent global analysis of product evaluations for Health Technology Assessments – which are often influential in decision making for approving and/or providing reimbursement for pharmaceuticals and other medical technology – found that the use of the word "efficacy" and effectiveness" proved misleading in the context of reimbursement decisions, which may negatively affect their outcomes. The study, presented at the International Society of Pharmacoeconomics and Outcomes Research conference in Berlin, suggests that while agencies state they are evaluating efficacy and effectiveness a similar number of times, their evaluations are generally for efficacy. This is important based on the actual meanings of the terms. Efficacy entails how a drug performs in an ideal or controlled circumstance, as in the context of a clinical trial. However, effectiveness describes a drug's success in real-world circumstances where the patient population and other variables cannot be controlled. Although reimbursement decisions are based on HTAs, the study demonstrated that the term effectiveness is often used when the evidence presented for consideration is often based on clinical trial scenarios rather than real world circumstances.

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