Blind Spots: When Medicine Gets It Wrong, and What It Means for Our Health, by Marty Makary, M.D. (Bloomsbury Publishing, 288 pp., $28.99)
The U.S. Food and Drug Administration is arguably the world’s most powerful regulatory agency, making decisions that affect trillions of dollars in spending. To run the agency, President Trump has nominated Marty Makary, who, like Health and Human Services Secretary Robert F. Kennedy Jr., has challenged public health orthodoxy. Makary’s recent book, Blind Spots, examines cases where the medical establishment has erred and offers insight into the thinking that might guide his FDA.
Fitting Kennedy’s vision, Makary discusses how “root causes” like pollution and poor nutrition shape health outcomes. His heroes are iconoclasts whose commitment to evidence-based medicine, he believes, has been stifled by institutions that suppress dissent for political reasons. A central challenge for Makary’s prospective FDA tenure will be how a critic of the establishment performs when he’s in charge.
Despite having no experience in government, Makary has influenced a number of salutary health-policy initiatives. A surgical oncologist at Hopkins, he was the lead author of a paper that became the influential “Surgical Safety Checklist,” which reduces surgical mortality by as much as a quarter. Similarly, he has been a leader in the elimination of “never events”—medical errors that should never happen in a hospital. And he has worked for medical price transparency, with his book The Price We Pay calling for “a requirement for all hospitals to post cash prices for shoppable services. . . . because transparency is an American value.”
Blind Spots suggests that transparency will be a priority at Makary’s FDA. For example, Makary highlights how evidence of HIV transmission through blood transfusion was suppressed in the 1980s to protect confidence in blood banks. He quotes the New York Times reporting that scientists “had not shared clues of [an AIDS-like virus] with health officials because they were holding it for publication in scientific journals.” Makary’s emphasizes the importance of sharing data rapidly, both in policy and in academic publications.
Makary’s focus on transparency dovetails with his dislike of paternalism. He criticizes the organ-donation system for not revealing waitlist positions, and the American Medical Association for “lobbying against patients having full access to their medical results in real time[.]”
His anti-paternalism also suggests openness to “challenge studies,” where participants are voluntarily infected to study a disease. (My nonprofit works on challenge studies.) Makary praises two historic examples: Edward Jenner’s smallpox vaccine test in the eighteenth century and Barry Marshall’s self-experiment indicating h. pylori causes ulcers. He separately endorses leading challenge-study researcher Matt Memoli’s universal flu vaccine candidate.
If you read Blind Spots without knowing about Makary’s nomination, you might not predict the FDA as his destination. Only a few of his case studies involve FDA-relevant issues: a story about the misconceived panic over the safety of silicone breast implants and, more briefly, a discussion of oxycontin. In each, he criticizes the FDA for making decisions without proper evidence. In the silicone breast-implant case, a CBS television segment “ignited a nationwide frenzy,” which led to an FDA ban in 1992. The ban was not rescinded until 2006, eight years after the FDA commissioner who originally instituted it conceded that “there’s no evidence that [silicone breast implants] cause systemic disease.”
Time and again, Makary stresses the importance of being rigorous about evidence, and the lives lost to delays in the availability of that evidence. This may imply the desirability of “real-time transparency” between drug developers and the FDA (sharing data with the FDA automatically as the researchers gather it during a clinical trial) and between the government and the public after a drug has been approved (providing a publicly legible readout of real-world evidence on usage, safety, and effectiveness).
Such transparency would require substantial new capacities at the FDA. Systems that connect real-world uses of drugs to patient outcomes would help address declining social trust in medicine. Makary could pursue such capacities by using advances enabled by artificial intelligence, piloting the approach in areas where funding might be available, or collaborating with international partners with easier access to health-care data, such as Israel or the United Kingdom.
A key question for Makary: How can someone who has influenced public policy from the outside transition to effective leadership of an establishment that he has criticized? Scott Gottlieb joined the FDA with the aim of sharing the agency’s decision letters with the public, but he failed to do so. Makary’s predecessor, Robert Califf, made improving the FDA’s use of real-world evidence a key objective, but whether those attempts will bear fruit remains uncertain.
Makary says that his goal in Blind Spots is “to increase public trust by restoring faith in the scientific process.” At the FDA, Makary will need to bring innovation to the problem of administration and institutional design. That task will likely be more challenging than any case he has faced in his career.
Photo by Noam Galai/Getty Images for HBO