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Silverman  E. Tennessee to become the first state to run a closed Medicaid drug formulary. STAT. January 8, 2021. Accessed September 6, 2021. https://www.statnews.com/pharmalot/2021/01/08/medicaid-trump-tennessee-massachusetts/
Bagley  N, Sachs  RE.  Limiting state flexibility in drug pricing.   N Engl J Med. 2018;379(11):1002-1004. doi:10.1056/NEJMp1809358PubMedGoogle ScholarCrossref
Payment for Covered Outpatient Drugs. 42 USC §1396r-8 (2009). Accessed September 9, 2021. https://www.govinfo.gov/app/details/USCODE-2008-title42/USCODE-2008-title42-chap7-subchapXIX-sec1396r-8/summary
Expedited Approval of Drugs for Serious or Life-threatening Diseases or Conditions. 21 USC §356 (2014). Accessed September 7, 2021. https://www.govinfo.gov/app/details/USCODE-2013-title21/USCODE-2013-title21-chap9-subchapV-partA-sec356/summary
High-cost specialty drugs: moving towards recommendations. Medicaid and CHIP Payment and Access Commission. March 2021. Accessed September 6, 2021. https://www.macpac.gov/publication/high-cost-specialty-drugs-moving-towards-recommendations/
US Food and Drug Administration.  New drug, antibiotic, and biological drug product regulations; accelerated approval—FDA. Final rule.   Fed Regist. 1992;57(239):58942-58960.PubMedGoogle Scholar
State Medicaid coverage of drugs approved by the FDA under accelerated approval pathway. News release. Centers for Medicare & Medicaid Services. June 27, 2018. Accessed September 6, 2021. https://www.medicaid.gov/medicaid-chip-program-information/by-topics/prescription-drugs/downloads/rx-releases/state-releases/state-rel-185.pdf
Gyawali  B, Hey  SP, Kesselheim  AS.  Assessment of the clinical benefit of cancer drugs receiving accelerated approval.   JAMA Intern Med. 2019;179(7):906-913. doi:10.1001/jamainternmed.2019.0462PubMedGoogle ScholarCrossref
Chang  CY, Nguyen  CP, Wesley  B, Guo  J, Johnson  LL, Joffe  HV.  Withdrawing approval of Makena—a proposal from the FDA Center for Drug Evaluation and Research.   N Engl J Med. 2020;383(24):e131. doi:10.1056/NEJMp2031055PubMedGoogle Scholar
Feuerstein  A. Sarepta gene therapy for Duchenne muscular dystrophy stumbles with failed study outcome. STAT. January 7, 2021. Accessed September 6, 2021. https://www.statnews.com/2021/01/07/sarepta-gene-therapy-for-duchenne-muscular-dystrophy-stumbles-with-failed-study-outcome/
Medicaid and CHIP Payment and Access Commission. April 2021 MACPAC Public Meeting; April 8-9, 2021. Accessed September 7, 2021. https://www.macpac.gov/public_meeting/april-2021-macpac-public-meeting/
Kaltenboeck  A, Mehlman  A, Pearson  SD. Strengthening the accelerated approval pathway: an analysis of potential policy reforms and their impact on uncertainty, access, innovation, and costs. Institute for Clinical and Economic Review. April 26, 2021. Accessed September 7, 2021. https://icer.org/wp-content/uploads/2021/04/Strengthening-the-Accelerated-Approval-Pathway-_-ICER-White-Paper-_-April-2021.pdf
Hwang  TJ, Kesselheim  AS.  Public referendum on drug prices in the US: will it bring relief?   BMJ. 2016;355:i5657. doi:10.1136/bmj.i5657PubMedGoogle Scholar
Rome  BN, Kesselheim  AS.  Will ending the Medicaid drug rebate cap lower drug prices?   JAMA Intern Med. 2021;181(8):1034-1035. doi:10.1001/jamainternmed.2021.2696PubMedGoogle ScholarCrossref
Medicaid drug spending trends. Medicaid and CHIP Payment and Access Commission. February 2019. Accessed September 6, 2021. https://www.macpac.gov/wp-content/uploads/2019/02/Medicaid-Drug-Spending-Trends.pdf
Beitz  JG. Accelerated approval of Makena. US Food and Drug Administration. February 3, 2011. Accessed September 6, 2021. https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/021945s000ltr.pdf
AMAG Pharmaceuticals files submission in response to the Food and Drug Administration’s notice of opportunity for a hearing and proposal to withdraw approval of Makena (hydroxyprogesterone caproate injection). GlobeNewswire. December 14, 2020. Accessed September 6, 2021. https://www.globenewswire.com/news-release/2020/12/14/2144543/0/en/AMAG-Pharmaceuticals-Files-Submission-in-Response-To-the-Food-And-Drug-Administration-s-Notice-of-Opportunity-for-a-Hearing-and-Proposal-To-Withdraw-Approval-of-Makena-hydroxyproge.html
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    Views 5,288
    Original Investigation
    October 8, 2021

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

    Author Affiliations
    • 1Washington University in St Louis School of Law, St Louis, Missouri
    • 2Yale School of Medicine, New Haven, Connecticut
    • 3Editorial Intern, JAMA Health Forum
    • 4Department of Health Policy and Management, University of Pittsburgh Graduate School of Public Health, Pittsburgh, Pennsylvania
    • 5Associate Editor, JAMA Health Forum
    • 6Vanderbilt-Ingram Cancer Center, Nashville, Tennessee
    • 7Department of Health Policy, Vanderbilt University School of Medicine, Nashville, Tennessee
    JAMA Health Forum. 2021;2(10):e213177. doi:10.1001/jamahealthforum.2021.3177
    Key Points

    Question  How much do prescription drugs approved through the US Food and Drug Administration’s accelerated approval program contribute to state Medicaid program spending?

    Findings  In this cross-sectional study of 216 drugs granted accelerated approval from 1992 through 2020, relative to all drugs paid for by Medicaid, products with accelerated approval comprised less than 1% of use. Despite their infrequent use, annual net spending on drugs with accelerated approval represented 6.4% to 9.1% of net spending on all drugs covered by Medicaid (in 2015 and 2018, respectively).

    Meaning  Medicaid spending on drugs with accelerated approval represents an outsized amount of spending relative to their use.


    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.