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Drugs and the FDA: Safety, Efficacy, and the Public's Trust
How the FDA was shaped by public health crises and patient advocacy, told against a background of the contentious hearings on the breast cancer drug Avastin.
Food and Drug Administration approval for COVID-19 vaccines and the controversial Alzheimer’s drug Aduhelm made headlines, but few of us know much about how the agency does its work. Why is the FDA the ultimate US authority on a drug’s safety and efficacy? In Drugs and the FDA , Mikkael Sekeres—a leading oncologist and former chair of the FDA’s cancer drug advisory committee—tells the story of how the FDA became the most trusted regulatory agency in the world. It took a series of tragedies and health crises, as well as patient advocacy, for the government to take responsibility for ensuring the efficacy and safety of drugs and medical devices.
Before the FDA existed, drug makers could hawk any potion, claim treatment of any ailment, and make any promise on a label. But then, throughout the twentieth century, the government was forced to take action when children were poisoned by contaminated diphtheria and smallpox vaccines, an early antibiotic contained antifreeze, a drug prescribed for morning sickness in pregnancy caused babies to be born disfigured, and access to AIDS drugs was limited to a few clinical trials while thousands died. Sekeres describes all these events against the backdrop of the contentious 2011 hearings on the breast cancer drug Avastin, in which he participated as a panel member. The Avastin hearings, he says, put to the test a century of the FDA’s evolution, demonstrating how its system of checks and balances works—or doesn’t work.
Food and Drug Administration approval for COVID-19 vaccines and the controversial Alzheimer’s drug Aduhelm made headlines, but few of us know much about how the agency does its work. Why is the FDA the ultimate US authority on a drug’s safety and efficacy? In Drugs and the FDA , Mikkael Sekeres—a leading oncologist and former chair of the FDA’s cancer drug advisory committee—tells the story of how the FDA became the most trusted regulatory agency in the world. It took a series of tragedies and health crises, as well as patient advocacy, for the government to take responsibility for ensuring the efficacy and safety of drugs and medical devices.
Before the FDA existed, drug makers could hawk any potion, claim treatment of any ailment, and make any promise on a label. But then, throughout the twentieth century, the government was forced to take action when children were poisoned by contaminated diphtheria and smallpox vaccines, an early antibiotic contained antifreeze, a drug prescribed for morning sickness in pregnancy caused babies to be born disfigured, and access to AIDS drugs was limited to a few clinical trials while thousands died. Sekeres describes all these events against the backdrop of the contentious 2011 hearings on the breast cancer drug Avastin, in which he participated as a panel member. The Avastin hearings, he says, put to the test a century of the FDA’s evolution, demonstrating how its system of checks and balances works—or doesn’t work.
320 pages, Hardcover
Published September 27, 2022
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Displaying 1 - 13 of 13 reviews
October 13, 2022
In the aftermath of the COVID-19 pandemic and other high profiles events concerning the Food and Drug Administration (FDA), such as the controversial approval of Alzheimer's drug aducanumab, there has been some increased interest in the regulatory and investigational procedures and standards involved in bringing a drug to market. Responding to this, Mikkael A. Sekeres, a professor of medicine and chief physician of the division of hematology at Miami's Sylvester Comprehensive Cancer Center, has penned Drugs and the FDA: Safety, Efficacy, and the Public's Trust, a fairly comprehensive yet accessible description of the FDA approval process for investigational drugs that is woven around a 2011 controversy concerning withdrawal of a breast cancer drug made by Genentech, Avastin. Sekeres' book also includes a brief history of the FDA, focusing on some of the horror stories that motivated public demand for increased drug safety (e.g. thalidomide-related birth defects) and, later, more rapid access to likely effective drugs (e.g. advocacy from HIV and cancer patients). The glue that holds together the information that one could mostly glean from the FDA's website or other sources is Sekeres' broader human interest narrative comprised of anecdotes from treating cancer patients and serving on the Oncologic Drugs Advisory Committee (ODAC). Drugs and the FDA also partially serves as a defense of the FDA's current regulatory program, insisting that it has responded to public demands while remaining faithful to its core mission of ensuring safe and effective drugs reach patients.
Although the central story about the withdrawal of Avastin (Bevacizumab) for the treatment of HER2-negative metastatic breast cancer after lackluster follow-up clinical trial results is awkwardly distributed throughout the work, it is still an illustrative anecdote. It allows Sekeres to introduce the FDA's accelerated approval program, which started in the early 1990s in response to HIV/AIDS and was designed (as the name indicates) to speed up the process for getting drugs to market for serious conditions with unmet needs. Drugs that receive accelerated approvals have to finish or perform follow-up studies that confirm the safety and clinical benefit that was demonstrated by earlier trial results (often called surrogate endpoints). The focus on the accelerated approval program is telling and topical. The current commissioner of the FDA was only able to corral support from the senate finance committee for his nomination by promising reform to this program. It is an unfortunate that the program is a hot-button issue for regulators and legislators given that clinical and financial analyses of the accelerated approval have suggested that it has been a huge success, especially for biomarker-guided target therapies in cancer.
The story of Avastin withdrawal also enables Sekeres to detail the general standards that the FDA relies on for guiding their approval decisions. The FDA essentially assesses the totality of clinical trial data, as opposed to relying only on a couple metrics, and then applies expert clinical judgement with an eye toward balancing benefits and risks. In other words, a drug must be safe-enough and provide a meaningful benefit to patient (preferably survival in serious conditions like cancer) in order to gain full approval. This can be a challenging and sometimes moving bar to clear, which can cause much consternation among patients and pharmaceutical companies. The entire process is time and resource intensive, involving many experts and stakeholders, who are asked to evaluate relevant data and provide guidance that the FDA takes into consideration before making final decisions. The insider look from Sekeres humanizes all of those involved and is for the most part understanding of all the competing interests involved with only mild critique of the financial interests at stake.
Drugs and the FDA is a defense of a frequently assailed institution from a partial insider. Sekeres has a decidedly establishment perspective and a generally positive appraisal of the FDA. It would have been interesting to see a more rigorous evaluation of the FDA performance in a work like this. The most interesting facts and figures in the book typically concern longitudinal perspectives on the FDA decision-making and patient outcomes and needs. I think the book, in trying to be a defense of the FDA's delicate balancing act, reveals some obvious bureaucratic deficiencies, such as ambiguity in approval standards and siloed evaluations of drug applications. The book also doesn't discuss in detail (though does briefly mention) how precision medicine in the genomic era is rapidly changing the indication-based paradigm for drug approvals in cancer. These so-called tumor-agnostic or pan-cancer approvals are premised on particular biomarkers that are identified via laboratory assay, including those that leverage next-generation sequencing technologies. The genomic era will likely lead to important and dramatic changes to what we need from the FDA. A slow and unwieldy regulatory environment may risk stifling innovation and growth. I would have appreciated a deeper look at this trend, especially from an oncologist like Sekeres.
Drugs and the FDA is a quick read that makes a complicated bureaucratic body's work legible to lay readers and supplies a human interest angle to keep readers engaged. Those who have any interest in clinical medicine and pharmaceuticals may want to pick this up as an introduction to this space, though much of the critical information can be quickly located via online sources.
*Disclosure: I received this as an ARC through NetGalley.
Although the central story about the withdrawal of Avastin (Bevacizumab) for the treatment of HER2-negative metastatic breast cancer after lackluster follow-up clinical trial results is awkwardly distributed throughout the work, it is still an illustrative anecdote. It allows Sekeres to introduce the FDA's accelerated approval program, which started in the early 1990s in response to HIV/AIDS and was designed (as the name indicates) to speed up the process for getting drugs to market for serious conditions with unmet needs. Drugs that receive accelerated approvals have to finish or perform follow-up studies that confirm the safety and clinical benefit that was demonstrated by earlier trial results (often called surrogate endpoints). The focus on the accelerated approval program is telling and topical. The current commissioner of the FDA was only able to corral support from the senate finance committee for his nomination by promising reform to this program. It is an unfortunate that the program is a hot-button issue for regulators and legislators given that clinical and financial analyses of the accelerated approval have suggested that it has been a huge success, especially for biomarker-guided target therapies in cancer.
The story of Avastin withdrawal also enables Sekeres to detail the general standards that the FDA relies on for guiding their approval decisions. The FDA essentially assesses the totality of clinical trial data, as opposed to relying only on a couple metrics, and then applies expert clinical judgement with an eye toward balancing benefits and risks. In other words, a drug must be safe-enough and provide a meaningful benefit to patient (preferably survival in serious conditions like cancer) in order to gain full approval. This can be a challenging and sometimes moving bar to clear, which can cause much consternation among patients and pharmaceutical companies. The entire process is time and resource intensive, involving many experts and stakeholders, who are asked to evaluate relevant data and provide guidance that the FDA takes into consideration before making final decisions. The insider look from Sekeres humanizes all of those involved and is for the most part understanding of all the competing interests involved with only mild critique of the financial interests at stake.
Drugs and the FDA is a defense of a frequently assailed institution from a partial insider. Sekeres has a decidedly establishment perspective and a generally positive appraisal of the FDA. It would have been interesting to see a more rigorous evaluation of the FDA performance in a work like this. The most interesting facts and figures in the book typically concern longitudinal perspectives on the FDA decision-making and patient outcomes and needs. I think the book, in trying to be a defense of the FDA's delicate balancing act, reveals some obvious bureaucratic deficiencies, such as ambiguity in approval standards and siloed evaluations of drug applications. The book also doesn't discuss in detail (though does briefly mention) how precision medicine in the genomic era is rapidly changing the indication-based paradigm for drug approvals in cancer. These so-called tumor-agnostic or pan-cancer approvals are premised on particular biomarkers that are identified via laboratory assay, including those that leverage next-generation sequencing technologies. The genomic era will likely lead to important and dramatic changes to what we need from the FDA. A slow and unwieldy regulatory environment may risk stifling innovation and growth. I would have appreciated a deeper look at this trend, especially from an oncologist like Sekeres.
Drugs and the FDA is a quick read that makes a complicated bureaucratic body's work legible to lay readers and supplies a human interest angle to keep readers engaged. Those who have any interest in clinical medicine and pharmaceuticals may want to pick this up as an introduction to this space, though much of the critical information can be quickly located via online sources.
*Disclosure: I received this as an ARC through NetGalley.
January 12, 2024
A summary of the FDA Avastin hearings by a participant. I was hoping for a more expansive explanation of the workings of this dangerous agency that is captive to Big Pharma and insurance companies, but that is not this book. One thing that is clear is that the FDA is a slave to its process and far more concerned about being criticized for approving a drug that has adverse effects than facilitating access to new life saving therapies. Safety Safety Safety and only then do they care (if at all) about efficacy.
From outside the hearings: "Hey Hey FDA, How many people have you killed today"
From outside the hearings: "Hey Hey FDA, How many people have you killed today"
June 15, 2025
Didn’t know the story of avastin or how accelerated approval came out of aids epidemic. Good info
July 2, 2022
I loved this book. The storytelling is great, with excellent writing as it uses the FDA Avastin hearings as a backdrop for the history of the FDA. The writing is conversational, and Dr. Sekeres uses humor when appropriate. The book is also about people and I found some of the stories touching and thoughtful. Since Dr. Sekeres was involved in the hearings, his personal insights were both invaluable to the storytelling and fascinating. This book is well worth reading. Thank you to Netgalley and MIT Press for the digital review copy.
December 18, 2022
I liked this book because it explained the FDA and its processes in a way that was easy to read and understand (not a lot of “doctor speak”). The author explains things by walking you through an actual revocation of a drug that he was a part of, but I think he did a good job of explaining both sides of the argument. He also does a good job of explaining the history of the FDA and how we arrived at the approval process used today. I definitely learned alot that I didn’t know.
January 28, 2023
Excellent, well written book. Examines the history and efficacy of the FDA. Complex ideas are broken down and discussed in a way that makes them easy to understand. Well paced with regular links between various historical events and more recent events like the COVID vaccine development. I will never look at drugs the same way again.
April 21, 2023
Great overview of the Oncology Drug Approval Process!
Balanced look at the opportunities and challenges of our current approval system for cancer drugs. The author presents an interesting and informative review of this process based on his experience on the FTA’s oncology, drug advisory committee worth reading for delay in professional public.
Balanced look at the opportunities and challenges of our current approval system for cancer drugs. The author presents an interesting and informative review of this process based on his experience on the FTA’s oncology, drug advisory committee worth reading for delay in professional public.
September 3, 2024
Well rounded, and rather objective for this particular case of Avastin. Super transparent and informative about what the author knows about the workings of the FDA, but clearly capturing all nuances regarding all products that FDA regulates is not feasible.
May 9, 2023
I dont know that the writing was 4 stars but the insight in to how things worked was really interesting and the store / framing was good.
August 5, 2023
I like the tone of Dr. Sekeres in portraying his thoughts during the hearing and the tragedy that starts it all — the ethylene glycol tragedy
March 21, 2024
Informative parallel storytelling of FDA's origins and FDA's AdCom hearing of Avastin-Breast cancer indication.
September 1, 2024
Should have been condensed. I know now how drugs are tested. And especially this one, with detail upon detail of personalities.
December 1, 2024
Interesting book exploring the role of the FDA. Drug regulation is a complex process and id love to learn more about it
Displaying 1 - 13 of 13 reviews