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Inside the FDA Through the Eyes of a Clinical Investigator

August 21, 2023

September 2023

Naseema Gangat, MBBS

Naseema Gangat, MBBS, is a professor of medicine in the Division of Hematology at Mayo Clinic in Rochester, Minnesota.

Title: Drugs and the FDA: Safety, Efficacy, and the Public’s Trust Author: Mikkael A. Sekeres, MD, MS Length: 320 pages Published: September 2022, MIT Press

Title: Drugs and the FDA: Safety, Efficacy, and the Public’s Trust

Author: Mikkael A. Sekeres, MD, MS

Length: 320 pages

Published: September 2022, MIT Press

About the Author

Mikkael A. Sekeres, MD, is a professor of medicine and chief of the Division of Hematology at Sylvester Comprehensive Cancer Center of the University of Miami in Florida, a preeminent physician, clinical investigator, and former chair of the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA). A regular essayist for the New York Times, he is the author of the top-rated When Blood Breaks Down: Life Lessons from Leukemia (MIT Press; 2020) and a former editor-in-chief of ASH Clinical News. His latest release, Drugs and the FDA, has not strayed from this trend.

About the Book

Drugs and the FDA is an insider’s view into the FDA hearing on bevacizumab, sold under the brand name Avastin, for metastatic breast cancer. The hearing was conducted in 2011 and led to withdrawal of bevacizumab’s breast cancer indication. Dr. Sekeres details the beginnings of the regulation of food and medicine for quality and safety in the U.S., starting with the Biologics Control Act enacted in the early 20th century. However, it was not until 1930 that the FDA formally came into being in the wake of a series of medical tragedies that included childhood mortality from contaminated diphtheria and smallpox vaccines, deaths from the antifreeze-containing antibiotic elixir sulfanilamide, and thalidomide-induced limb deformities after its use for morning sickness in pregnancy. In 1992, an accelerated approval program for drugs was developed to improve access for patients with life-threatening diseases, with an initial focus on HIV drugs. In recent years, emergency authorization of COVID-19 vaccines brought the FDA’s role in drug and vaccine development to the public forefront. Accordingly, Drugs and the FDA speaks to a wide audience and aims to demystify the workings of the FDA in the drug approval and withdrawal processes.

Exclusive Elements

The story is spun in an authentic manner through Dr. Sekeres’ distinct experience serving as an ODAC member during the FDA hearing on bevacizumab, as a physician directly involved in patient care, and as lead investigator of pivotal clinical trials. He squarely conveys the palpable emotions and inner conflicts of patient, pharmaceutical, ODAC, and FDA representatives at the hearing. Moreover, his mother’s brush with recurrent lung cancer lends a personal touch to the narrative, bringing in the family perspective.

Breast cancer survivor Heraleen Broome’s moving statement at the hearing, “I don’t think it is reasonable for you to set a number of people that need to be alive as a result of this drug in order to allow it to be sold. … My life should be enough, and it’s not just my life but the lives of my family, friends, coworkers, and everyone I meet,” resonates strongly as a stark reminder of the delicate equipoise between individual patient outcomes and statistical findings.

Personally, the chapter titled “The Moxie To Do Battle” stood out; the story of James (Jimmy) Petrarcha, a gentleman with relapsed and refractory acute myeloid leukemia (AML), hit home, as my own patients with leukemia face similar situations, despite the FDA’s approval of eight drugs for AML over a span of two years (2017-2018). Jimmy’s valor and metaphorical reference to “Olympic athlete” and “boxer,” coupled with Dr. Sekeres’ substantive and candid dialogue with Jimmy, impart inspiration and ward off conflicts that may arise during the conduct of clinical trials in routine practice.


In the past decade, not surprisingly, approximately 85% of accelerated drug approvals by the FDA have been granted in oncology, of which 37% were for hematologic malignancies.1,2 As the breadth of cancer therapeutics continues to evolve, it is critical to (re)discover the salient principles of drug development and clinical trials, along with an understanding of the workings of the FDA. Accordingly, Dr. Sekeres underlines a patient-centered approach to conducting clinical trials and, in a conversational style, masterfully delivers the trials and tribulations of clinical trials: What are clinical trials? What are the phases of clinical trials? Why are they important? Why do patients participate in trials? Is progression-free survival a meaningful endpoint?


There is no better time than now, on the heels of the COVID-19 pandemic and proliferation of cancer drug approvals, to plunge into Drugs and the FDA. What drew me to it? Foremost, I immediately connected with the authentic storyline, and second, the narrative voice. The enduring value of this book lies in how it articulates the FDA drug approval and withdrawal processes; the timeless take-home messages will still be relatable decades later, which makes it a must-read for all physicians who are engaged in clinical research and, especially, for our patients.


  1. Beaver JA, Pazdur R. “Dangling” accelerated approvals in oncology. N Engl J Med. 2021;384(18):e68.
  2. Demoss P, Kanapuru B, Gormley N. Hematologic malignancies and the Food and Drug Administration’s accelerated approval program. Hematologist. 2023;20:3.


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