Extending the US Food and Drug Administration's Postmarket Authorities.

Importance: The US Food and Drug Administration (FDA) has expansive regulatory flexibility regarding the quality and quantity of evidence it deems sufficient to approve new drugs, which has been increasingly used to grant approval based on less certain evidence of benefit. However, the FDA's regulatory flexibility with respect to standards for approval has not been matched by sufficient stringency in its exercise of postmarket safeguards, including the FDA's authority and willingness to require confirmation of benefit through postmarket efficacy studies or to withdraw approval when benefit is not confirmed.

Objective: To identify and evaluate opportunities for the FDA to extend its authority to require postmarket efficacy studies and use expedited withdrawal procedures for drugs approved despite substantial residual uncertainty outside the accelerated approval pathway.

Reforming Reimbursement for the US Food and Drug Administration's Accelerated Approval Program to Support State Medicaid Programs.

Importance: The US Food and Drug Administration (FDA) has an accelerated approval program that has become the subject of scholarly attention and criticism, not only for the FDA's oversight of the program but also for its implications for payers.

Observations: State Medicaid programs' legal obligations to provide reimbursement for accelerated approval products have created fiscal challenges for Medicaid that have been exacerbated by industry's changing use of the accelerated approval program over time. Although strategies for accelerated approval reforms have been proposed, most focus on reforming the FDA's accelerated approval pathway and product regulation without taking into account the implications of this pathway for state Medicaid programs.

Recent Trends in Medicaid Spending and Use of Drugs With US Food and Drug Administration Accelerated Approval.

Importance: State Medicaid programs have reported concerns about rising drug prices and spending, particularly regarding drugs entering the market through the accelerated approval program under the US Food and Drug Administration (FDA). The accelerated approval program enables the FDA to approve drugs on the basis of unverified surrogate end points, meaning that clinical benefits for these products are uncertain at the time of approval. However, state Medicaid programs are legally required to cover these drugs.

Medicaid and Accelerated Approval: Spending on Drugs with and without Proven Clinical Benefits.

Many state Medicaid officials are concerned about rising prescription drug spending, particularly drugs approved through the Food and Drug Administration's (FDA) accelerated approval pathway. The authors examined how much of Medicaid programs' accelerated approval spending is attributable to products that have demonstrated clinical benefits versus those that have not. Their findings provide support for states' concerns that pharmaceutical companies often fail to complete their required postapproval confirmatory studies within the FDA's requested timeline.

Changes in the Use of Hydroxyprogesterone Caproate Injection After Confirmatory Trial Failure.

This cross-sectional study examines changes in hydroxyprogesterone caproate fills from 2010 through 2020.

The Rhetorical Transformations and Policy Failures of Prescription Drug Pricing Reform under the Trump Administration.

Throughout his four years in office, President Trump made prescription drug pricing a focus of his policy agenda. President Trump not only used strong language to criticize the pharmaceutical industry and its practices but also introduced ambitious reform policies that had previously lacked acceptance among Republican policy makers. President Trump appears to have been successful in developing a new populist form of rhetoric that Republicans can use in support of novel drug pricing reforms such as the ones his administration considered.

Ensuring the safe and effective FDA regulation of fecal microbiota transplantation.

Scientists, policymakers, and medical professionals alike have become increasingly worried about the rise of antibiotic resistance, and the growing number of infections due to bacteria like , which cause a significant number of deaths and are imposing increasing costs on our health care system. However, in the last few years, fecal microbiota transplantation (FMT), the transplantation of stool from a healthy donor into the bowel of a patient, has emerged as a startlingly effective means to treat recurrent infections. At present, the FDA is proposing to regulate FMT as a biologic drug.