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

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

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

Searching for Scandinavians in pre-Viking Scotland : Molecular fingerprinting of Early Medieval combs

The character and chronology of Norse colonisation in Early Medieval northern Scotland (8th–10th centuries AD) is hotly debated. The presence of reindeer antler raw material in ‘native’ or ‘Pictish’ type combs from the Orkney Isles, northern Scotland has been put forward as evidence for a long and largely peaceful initial period of cultural contact, as opposed to a shorter, more polarised period probably in the late ninth century. Here this hypothesis is tested using a minimally-destructive collagen peptide mass fingerprinting method (ZooMS) to speciate the raw material of 20 combs. Eleven were identified as red deer, four as reindeer and one as whale. The accuracy and gentleness of this method was tested by the subsequent application of ancient DNA (aDNA) methods to fourteen of the same samples: in ten, amplification was successful and all supported the preliminary ZooMS identification. All ‘native’-type combs in the sample are identified as red deer, and all Norse types as reindeer. These results challenge previous species identifications for these combs' raw materials. The balance of evidence no longer supports the existence of a long period of cultural contact between Atlantic Scotland and Scandinavian settlers before the late 9th century. ZooMS is shown to have considerable potential for identification of worked bone and antler artefacts, with applications in archaeology and wildlife/art-history forensics

Accelerated Approval – Taking the FDA’s Concerns Seriously

As the U.S. Congress debates a must-pass bill reauthorizing Food and Drug Administration (FDA) user fees for drug and device companies, the future of the FDA’s accelerated-approval program has become a focus of policy discussion. This debate was amplified in part by the agency’s controversial approval in June 2021 of the Alzheimer’s disease drug aducanumab (Aduhelm), whose high price and unclear benefits highlighted key challenges associated with the accelerated-approval process and reignited criticisms of the program. In both formal legislative-proposal requests and appearances before Congress, FDA officials have asked for greater authority to require companies to conduct more timely confirmatory trials of drugs that are granted accelerated approval and to expedite the agency’s process for withdrawing drugs when such trials don’t show evidence of clinical benefit. Although earlier congressional proposals would have addressed both these issues, legislators settled on compromise proposals that do not

Confronting State Medicaid Drug Spending Pressures

Across the country, state Medicaid budgets are under increasing strain, driven by factors including the high and increasing prices of prescription drugs. Nearly 30% of state spending supports Medicaid programs, and Medicaid programs spent more than $29 billion on prescription drugs in 2017 after accounting for manufacturer discounts. Perhaps most concerning is the increasing share of spending devoted to a few high-cost drugs. In 2017, drugs costing more than$1000 per claim represented just 1.2% of claims but accounted for 43.7% of all Medicaid drug spending, which is up from 31.1% in 2014

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

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 post-approval 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 post-approval 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 curriculum for the hospitalized aging medical patient program: A collaborative faculty development program for hospitalists, general internists, and geriatricians

10.1002/jhm.348Journal of Hospital Medicine35384-39