Clinical trials—we get what we pay for
Ces deux études présentent, pour l'une, une estimation des coûts associés aux essais cliniques pivots évaluant l'efficacité de médicaments, dont des anticancéreux, ayant reçu une autorisation de mise sur le marché par la FDA entre 2015 et 2016 (138 essais pivots; 59 nouveaux agents thérapeutiques), pour l'autre, une estimation des coûts associés aux essais cliniques évaluant l'efficacité de médicaments pédiatriques (141 essais, 54 agents ; 20 240 enfants inclus sur la période 2007-2012)
It is universally agreed that a clinical trial provides invaluable insights and knowledge, particularly when done well, through the use of randomization, blinded allocation, by including a control arm, and by focusing on a clinical outcome as opposed to a surrogate marker of disease. Clinical trials form the basis of many of the most important determinations in medicine and health care broadly, including the US Food and Drug Administration’s (FDA’s) determination of medical product safety and efficacy, the determination made by the Centers for Medicare & Medicaid Services and other commercial payers to provide coverage for a medical product, and clinical recommendations made by professional societies and government agencies to guide practice. But a common objection to performing a clinical trial is that trials are expensive, requiring extensive time and resources (although such costs pale in comparison to the costs of the health care interventions spent after approval, with or without coverage).
JAMA Internal Medicine , éditorial, 2017