FDA approved concept. Rubber stamp with FDA and pills on craft paper. 3d illustration.

Book Review: ‘Drugs and the FDA: Safety, Efficacy, and the Public’s Trust’ by Mikkael A. Sekeres

By Matthew Chun

In Drugs and the FDA: Safety, Efficacy, and the Public’s Trust, oncologist Mikkael A. Sekeres tells a captivating story of how the U.S. Food and Drug Administration became the agency it is today and how it makes some of its toughest decisions regarding the regulation of potent drugs.

Sekeres centers his narrative on the controversial 2011 Avastin hearings, in which the FDA reconsidered and ultimately withdrew the breast cancer indication for Genentech’s Avastin drug. Having served on the Oncologic Drugs Advisory Committee (ODAC) tasked with making a recommendation to the FDA based on the hearings and clinical data, Sekeres provides a relatable personal account of the emotion-filled proceedings and the agonizing decision to withdraw approval of a beloved treatment option that never lived up to its promising initial results.

As he recounts his experience as an ODAC member, Sekeres skillfully weaves in historical references to various regulatory failures, including poisoned vaccines, opioid deaths, thalidomide-induced birth defects, and the woefully inadequate response to HIV/AIDS, which shaped the role of the FDA since its inception in 1930. Upon describing how the FDA developed its modern system of checks and balances to ensure drug safety, efficacy, and accessibility, Sekeres then illustrates how the Avastin hearings put all of these processes and values to the test. Among other things, Drugs and the FDA encourages readers to grapple with several important themes that pervade the agency’s decision-making process, including (1) the tension between drug safety and accessibility, (2) the relative weight of expert opinion versus patient autonomy, and (3) the role of democracy and transparency in drug regulation.

The Tension Between Drug Safety and Accessibility

Featuring front and center in the Avastin hearings was the inevitable tension between drug safety and accessibility. On the one hand, the FDA is tasked with approving drugs only if their benefits justify any potential risks to patients. On the other hand, the FDA also serves as a gatekeeper to drugs and can unduly limit drug accessibility by regulating too zealously. So what is the FDA to do when a conditionally approved breast cancer drug causes harsh side effects without living up to its promising initial results of increasing length or quality of life? While the answer may seem obvious, the Avastin hearings demonstrated the difficulties of withdrawing approval for a drug that desperate patients already had relied upon and believed improved their condition, even if the data didn’t demonstrate any statistically significant benefit. Readers are left to decide for themselves whether the FDA properly conceptualized its role in balancing this tension and whether it was a failure or success of the regulatory system for the agency to stick to its guns.

The Relative Weight of Expert Opinion Versus Patient Autonomy

The Avastin hearings further raise questions about the relative weight placed on expert opinion versus patient autonomy in the regulatory process. While ODAC’s expert analysis of the clinical trial data determined that the benefits of Avastin (or rather, lack thereof) did not justify the harms of continued approval, this cost-benefit analysis is, at its heart, a normative decision. So why should patients not be able to make this decision for themselves? The patients who attended, spoke, and protested on behalf of Genentech at the Avastin hearings arguably were well-aware of the drug’s lackluster clinical trial results. And if so, the FDA’s decision to withdraw Avastin’s breast cancer indication could be construed as a direct affront to their ability to make their own informed treatment decisions. Drugs and the FDA thus invites readers to consider whether the FDA’s revocation of Avastin’s breast cancer indication was an appropriate curtailment of patient autonomy.

The Role of Democracy and Transparency in Drug Regulation

Finally, the Avastin hearings demonstrated the ideals of democracy and transparency at work in the FDA’s regulatory process. In his retelling, Sekeres emphasizes the public nature of the hearings and the moving anecdotes told by the protestors and breast cancer patients who came out to plead for Avastin’s continued approval for breast cancer treatment. However, while it may seem right to give the public a voice and a clear view into the agency’s decision-making process, readers are left to reflect on whether the FDA’s proceedings paid anything more than lip service to those values. Yes, members of the public were invited to provide their comments at the hearings. But to what extent did they actually influence the decisions of ODAC? And what does one make of the representativeness of the individuals able to show up for the hearings at the FDA’s Maryland campus during two full weekdays in June? Could they really speak for the breast cancer patients who were too sick from Avastin’s side effects or their underlying cancer to make an appearance? What about the public at large?


Drugs and the FDA: Safety, Efficacy, and Public Trust is a thought-provoking book that confronts readers with all of these difficult questions while keeping them glued to their seats through Sekeres’s skillful and self-reflective retelling of the Avastin hearings. It is a must-read for anyone interested in how the FDA became the trusted drug-regulating agency it is today and how it goes about making the tough life-or-death decisions that Americans rely upon in their everyday lives and in their most vulnerable moments of illness.

Matthew Chun

Matthew Chun is a J.D. candidate at Harvard Law School and patent agent at Fish & Richardson P.C. He holds a DPhil in Engineering Science from the University of Oxford and a B.S. in Mechanical Engineering from the Massachusetts Institute of Technology. At Harvard Law School, Dr. Chun is Managing Editor of the Harvard Journal of Law and Technology and a Student Fellow at the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics. All opinions expressed are solely his own.

Leave a Reply

This site uses Akismet to reduce spam. Learn how your comment data is processed.