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Welcome to another installment of the 2023 PAYERS’ GUIDE TO FDA UPDATES: Year-End Approvals, Upcoming Decisions

December 2023 Vol 16, Payers' Guide to FDA Updates - Foreword
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American Health & Drug Benefits (AHDB) is pleased to bring you the latest US Food and Drug Administration (FDA) actions in the December issue of the 2023 PAYERS’ GUIDE TO FDA UPDATES: Latest Approvals, Upcoming Decisions.

Developed to offer payers, providers, and other healthcare stakeholders an expert perspective on the challenges that the US healthcare system will face in 2024 and beyond, this edition of the Guide features an interview with AHDB Guest Editor Matt Mitchell, PharmD, MBA, MHP, FAMCP, Director, Board of Directors at the Academy of Managed Care Pharmacy and Associate Vice President, Pharmacy Services at SelectHealth, who provided his thoughts on the potential impact of the Inflation Reduction Act (IRA) on patients, payers, and manufacturers. In this interview, Dr Mitchell discussed several benefits for Medicare beneficiaries, including stabilization of premiums, the out-of-pocket cap, and copay smoothing. Regarding the effect the IRA may have on payer organizations, he pointed to the $2000 out-of-pocket maximum, which may increase the risk for adverse selection, and the introduction of the top 10 negotiated drugs feature, which may limit the ability of payer organizations to negotiate costs of those top 10 drugs. One unintended potential outcome of the IRA, according to Dr Mitchell, is that some manufacturers may limit their clinical development programs for rare or orphan drugs.

The Guide includes 2 in-depth profiles on important new FDA approvals in 2023. In November 2023, the FDA approved nirogacestat (Ogsiveo, SpringWorks Therapeutics Inc.) for adults with progressing desmoid tumors (DTs) who require systemic treatment. This is the first FDA-approved treatment for patients with DTs beyond surgery and radiation options.

A second profile highlights exagamglogene autotemcel (exa-cel; Casgevy, Vertex Pharmaceuticals Inc.) and lovotibeglogene autotemcel (lovo-cel; Lyfgenia, Bluebird Bio Inc.), approved by the FDA in December 2023 for the treatment of sickle cell disease (SCD) in patients aged ≥12 years with a history of recurrent vaso-occlusive crises. Exa-cel and lovo-cel are also being evaluated for use in the treatment of transfusion-dependent beta-thalassemia (TDT).

For decades, the management of patients with SCD and TDT was primarily supportive, but these agents strike at the genetic underpinnings of SCD and TDT, suggesting a new treatment paradigm for these chronic, debilitating diseases.

This edition of the Guide also offers a useful overview of new drugs approved by the FDA from August 14, 2023, through December 21, 2023. This entry represents a listing of all new molecular entities and new biologic license applications (although it does not include vaccines, allergenic products, blood and blood products, plasma derivatives, and cellular and gene therapy products), and it provides the indications, class/route, approval considerations, and approval dates for all agents approved in the United States during that period. In all, the FDA approved 57 new drugs in 2023, compared with 37 in 2022, and oncology continued in its position atop the list of most approvals this year, followed by neurology and infectious diseases.

The Guide also provides a table presenting the FDA pipeline from January 2024 through March 2024, a useful forward-looking listing that allows payers, providers, and other healthcare stakeholders to anticipate what is likely coming in terms of approvals.

The trend of accelerating the drug approval process continues, as evidenced by the many drugs approved under an expedited review pathway. Of the 25 approvals during the latter part of 2023, approximately half received fast track/accelerated/priority designation.

Additional updates of new drugs and new indications will be posted online at, together with the full Guide.

We hope you find this publication a useful tool for applying up-to-date information on new pharmaceuticals to benefit design decisions and patient care.

EDITOR’S NOTE: The FDA continues to make changes to the prescribing information of many drugs on an ongoing basis. Every effort has been made to update the information in each drug update and any other sections of this publication based on each drug’s prescribing information up to December 21, 2023. The Publisher is not responsible for any inaccuracies stemming from changes, new approvals, or company updates that became available after that date. Readers are advised to review the prescribing information for any future updates and revisions.

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Last modified: January 29, 2024