Changes in the Use of Hydroxyprogesterone Caproate Injection After Confirmatory Trial Failure
The US Food and Drug Administration (FDA) accelerated approval pathway allows new drugs with uncertain clinical benefits to be approved on the basis of clinical trials involving surrogate end points. For state Medicaid programs, which must cover nearly all drugs, including those receiving accelerated approval, stakeholders are concerned about spending on accelerated approval products that remain on the market despite failing to complete FDA-required follow-on clinical trials or where those trials fail to confirm clinical benefits. Hydroxyprogesterone caproate injection (Makena) illustrates the challenges that accelerated approval may pose for Medicaid. Makena was approved by the FDA in 2011 based on its ability to reduce recurrent preterm births and required postapproval trials confirming clinical benefits for neonatal outcomes. Yet a decade later, the use of and appropriate patient selection for progesterone supplementation, including Makena, remains controversial. How Makena’s use in Medicaid—the largest payer nationwide for preterm births —has changed with the evolving evidence base is unknown.
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bioethics health law policy pharmaceuticals public health rachel sachs regulation