The Trouble With ‘Do Your Own Research’ for Drugs

From MedPage Today

Teaser/Abstract

"Ideally, the approval of a new drug should be exclusively the province of science, but for a more than half-trillion-dollar-a-year industry, it couldn't possibly remain so. The same libertarian posture of the earlier twentieth century — the spirit that opposed government's right to require accurate drug labeling and prevent toxicity — lives on in the insistence by some advocates on the far right that the government shouldn't even be in the business of determining whether a drug works or not."

Source: MedPage Today, Perspectives / Second Opinions column. Published May 22, 2025; updated May 23, 2025. An excerpt from Rethinking Medications: Truth, Power, and the Drugs You Take (Simon & Schuster, 2025). Copyright © 2025 by Jerry Avorn. Reprinted by permission of Simon & Schuster, New York.

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SUMMARY

In this MedPage Today excerpt from Rethinking Medications, Dr. Jerry Avorn directly refutes the proposition — advanced by libertarian advocates and some political figures on the far right — that individual physicians and patients could adequately assess drug effectiveness on their own, without a functioning FDA approval process. The piece identifies the specific informational, methodological, and structural reasons why independent drug evaluation is not feasible for individuals or even for most clinicians, and traces the ideological lineage of the "do your own research" argument from early twentieth-century opposition to drug labeling through its current political expressions in "right to try" legislation and anti-regulatory FDA reform proposals.

BACKGROUND

Dr. Avorn situates the "do your own research" argument within a longer historical pattern: the same libertarian spirit that opposed the government's right to require accurate drug labeling and prevent toxicity in the early twentieth century now manifests in calls to strip the FDA of its authority to assess whether drugs actually work. The argument, as Dr. Avorn characterizes it, holds that physicians and patients could determine drug effectiveness through their individual clinical experiences and marketplace decisions, rendering rigorous government review unnecessary.

KEY FINDINGS

Why independent drug evaluation is not feasible. Dr. Avorn identifies multiple structural reasons why individuals — including clinicians — cannot reliably assess drug effectiveness on their own. First, much of the detailed data the FDA receives from manufacturers is treated as the company's private property and kept secret, meaning any outside reviewer is not working with the complete evidentiary record. Second, evaluating a clinical trial requires technical expertise that goes well beyond reading a published paper: Were the study groups truly comparable at baseline? Was randomization and blinding done appropriately? What statistical methods were used? Were statistically significant differences also clinically meaningful? Were the patients studied comparable to the patients a doctor actually treats, or were they healthier and younger? How does the new drug compare to all relevant existing treatment options, including non-drug alternatives, that were not part of the trial?

Selective publication of favorable results. Beyond the methodological complexity of trial evaluation, Dr. Avorn highlights a systematic distortion in the medical literature produced by selective publication. He cites a landmark analysis by Erick Turner, MD — a psychiatrist who spent several years reviewing new drug applications at the FDA — and colleagues, who reviewed raw data from 74 clinical trials of 12 different antidepressants submitted to the FDA. Nearly a third of those trials had never been published. Virtually all studies showing favorable outcomes made it into medical journals. Of studies with negative or questionable results, nearly all were either never published or appeared with a positive spin on the findings. The result: the published medical literature showed 94% of antidepressant trials found the drugs effective, while only about half of all original studies submitted to the FDA showed the medications worked. In response to this problem, Congress required public disclosure of plans for all clinical trials before launch in 2007 — though disclosure of results remains far from complete.

The von Eschenbach op-ed. Dr. Avorn cites a Wall Street Journal op-ed by Andrew von Eschenbach, MD — appointed FDA Commissioner by President George W. Bush in 2005 — that proposed the FDA should stop assessing drug effectiveness, instead merely ensuring products are not dangerous before releasing them to the marketplace for doctors and patients to sort out which ones work through post-market experience. Dr. Avorn notes the irony: von Eschenbach, whose clinical expertise was as a prostate surgeon, simultaneously prolonged the FDA's years-long refusal to approve greater access to the morning-after contraceptive pill despite its proven safety and effectiveness — suggesting that laissez-faire marketplace logic was not consistently applied.

"Right to try" legislation. Dr. Avorn addresses the nationwide "right to try" movement for unapproved medications, pursued aggressively in several states by conservative legislators. He argues this movement addresses a problem that does not actually exist: the FDA has for many years allowed any physician to request access to an investigational unapproved drug from its manufacturer, approving approximately 99% of such requests. When access problems arise, it is typically the company — not the FDA — that resists making the product available. He raises two additional questions the "right to try" framework does not answer: Should health insurers or government programs pay for drugs that have not been determined to work? And if dangerous side effects occur, should society cover the costs of caring for those consequences?

The "compassionate use" language problem. Dr. Avorn recounts that when he and colleagues wrote a paper for the New England Journal of Medicine about expanded access to unapproved drugs, they used the term "compassionate use." The editors asked them to change it to "expanded access," on the grounds that there is nothing necessarily compassionate about helping people take an untested drug that may not work and could harm them.

IMPLICATIONS

The evidentiary record needed to determine whether a drug works is not publicly available in its entirety, is technically complex to interpret even for experts, and is systematically distorted by selective publication practices that favor positive findings. The proposition that individual physicians and patients could make these determinations independently — whether through personal experience, internet research, or marketplace signals — ignores all of these realities. An independent regulatory body with the authority, expertise, and access to the full body of evidence is not a constraint on freedom; it is the only mechanism by which the question of whether a drug works can be reliably answered at all.