Commercial Health Plan and Enrollee Out-of-Pocket Spending on Accelerated Approval Products in 2019.

This cross-sectional study examines spending by health care plans and enrollees on products with accelerated approval.

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. Evidence: The FDA's current approaches to regulatory flexibility with respect to standards for drug approval; examples of shortcomings in the postmarket period; existing statutes and regulations governing the scope of the FDA's authority to impose and enforce postmarket study requirements; and recent legislative reform and agency action regarding the accelerated approval pathway. Findings: Drawing on the broad language of the federal Food, Drug, and Cosmetic Act, the FDA could independently extend its core accelerated approval authorities-required postmarket efficacy studies and expedited withdrawal procedures-to any drug approved with substantial residual uncertainty regarding benefit, such as those supported by a single pivotal trial. To avoid exacerbating existing problems that have become evident during the past 3 decades of experience using the accelerated approval pathway, however, the FDA must ensure that postmarket studies are well designed and completed quickly, while compelling expedited withdrawal when needed. Conclusions and Relevance: Under current FDA approaches to drug approval, patients, clinicians, and payers may be left with little confidence about a drug's benefit not only when it first enters the market but also for an extended period thereafter. If policy makers continue to favor earlier market access over evidentiary certainty, flexible approvals must be matched by more expansive use of postmarket safeguards, an approach possible within the FDA's existing legal authorities.

Time to Confirmatory Study Initiation After Accelerated Approval of Cancer and Noncancer Drugs in the US.

This cross-sectional study examines and compares the time taken from the accelerated approval of cancer and noncancer drugs to the initiation of confirmatory studies in the US.

Preapproval Promises to Voluntarily Withdraw FDA-Approved Drugs.

This Viewpoint provides recommendations for improvements to strengthen legal obligations and decrease ambiguity for the US Food and Drug Administration regarding their reliance on voluntary preapproval withdrawal pledges.

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. There is a need for policy reforms that balance the goal of speeding approval of important medicines with states' real concerns regarding spending on medications with little evidence of clinical benefits. Areas of potential reform include formulary exclusion, Medicaid rebates, value-based pricing, and consolidated purchasing or carve outs. Conclusions and Relevance: Policy makers may wish to consider options for reforming reimbursement for accelerated approval products in addition to reforms to the FDA's operation of the pathway. Policy reform proposals can provide a range of options to evaluate trade-offs of access and pricing.

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. But the findings also highlight one issue that policy stakeholders have not yet devoted substantial attention to: the use of surrogate endpoints involved in the postapproval confirmatory studies for most of the products in this study's sample. The granularity of the study's results enabled an analysis of the impact of different policy recommendations on both the accelerated approval pathway and Medicaid programs. These findings inform the current policy debate, suggesting that policy stakeholders might focus attention on products converting their approval on the basis of surrogate outcomes rather than on clinical outcomes.

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. From a policy perspective, however, the Trump administration failed to implement any of their more ambitious reform ideas. This article considers three of the Trump administration's signature policies-state-sponsored prescription drug importation, Medicare Part B international reference pricing, and reforms to the Medicare Part D rebate system-and explores how they represent both the political ambitions and policy failures of the Trump administration. The fate of the Trump administration's prescription drug proposals also reveals lessons about innovation and access, which will be important to ongoing drug pricing reform efforts.

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. Little is known about the set of products with accelerated approval over time, their use among Medicaid beneficiaries, or the magnitude of their financial influence on state Medicaid programs. Objective: To identify the number and class of drugs approved through the FDA's accelerated approval pathway and analyze state Medicaid programs' use and spending on these drugs from 2015 through 2019. Design Setting and Participants: In this cross-sectional study, biannual FDA reports were used to identify products granted accelerated approval and their associated indications approved between December 1992 and December 2020. State Medicaid Drug Utilization Data files available for 1992 through 2019 were used to estimate national totals for spending and use of outpatient drugs. Main Outcomes and Measures: National Medicaid use and gross and net spending on drugs with accelerated approval from 2015 through 2019. Results: Since the inception of the FDA's accelerated approval pathway in 1992 through 2020, 216 product-indication pairs granted accelerated approval were identified, comprising 149 unique products. The composition of drugs approved through the pathway has changed over time, with 28 of 30 (93.3%) product-indication pairs receiving accelerated approval in 2020 being indicated for cancer. Relative to all outpatient prescription drugs paid for by Medicaid, products with accelerated approval ranged from 0.2% to 0.4% of use (1.3-2.4 million prescriptions annually). Despite their infrequent use, drugs with accelerated approval represented a minimum annual net spending on all drugs covered by Medicaid of 6.4% ($2.2 billion of $34.6 billion) in 2015 and a maximum of 9.1% ($2.5 billion of $27.6 billion) in 2018. Estimated annual gross spending on drugs with accelerated approval ranged from $4.2 billion to $4.9 billion over 2015 through 2019, and estimated net spending from $2.2 billion to $2.6 billion. Conclusions and Relevance: In this cross-sectional study of 216 drugs granted accelerated approval, state spending on drugs approved through the FDA's growing accelerated approval program represented an outsized amount of spending relative to use. Because drugs with accelerated approval have come to market on the basis of trials using surrogate end points, considerable amounts of this spending may have been attributable to products with unproven clinical benefits.