by Michael Reilly, ASBM Executive Director
It feels impossible these days to go a single week without news of another shocking policy move from the U.S. Food and Drug Administration that upends decades-old scientific consensus or agency norms regarding clinical safety data and drug approval standards.
Consider the most recent example. Earlier this month, the FDA abruptly refused to even review Moderna’s application for its mRNA-based flu vaccine in a decision that stunned the FDA’s own career scientists, industry observers, and public health experts alike. Notably, that refusal was followed not by a reasoned, science-driven explanation but by an equally abrupt reversal after public outcry. Similarly, the agency recently announced it will drop its long-standing requirement of two clinical trials for most drug approvals; only a single study will now be necessary.
Both the vaccine flip-flop and the reduction in data requirements are framed as reforms designed to speed access to innovative treatments while promoting confidence in approved products. Yet in practice, they do the opposite: The regulatory whiplash – the lack of consistency from day to day regarding what data is sufficient to promote confidence in our medicines, for example- erodes confidence in the FDA’s judgment and competence within the medical community and among the public it serves.
Headlines over the past year have included phrases like “chaos at the FDA” and “very unusual discord.” A Washington Post editorial warned that the damage from the Moderna reversal “won’t be easy to repair,” cautioning that drugmakers can no longer be confident the ground rules won’t shift mid-trial. The Hill quoted scientific leaders saying “something is very wrong at the FDA,” while The Wall Street Journal described the agency’s actions as “arbitrary government at its worst.”
Perhaps the most worrisome, if less well known, example of the FDA’s abrupt changes to approval criteria is its rejection of the longstanding global scientific consensus surrounding biosimilars- complex biologic medicines that closely mimic previously-approved products. While safe and effective, they are not identical to the original products in the way generics are.
For decades, regulators in the U.S., the EU, Canada, Australia, and Japan have reflected this indisputable scientific fact by treating biosimilars and generics as distinct categories, requiring tailored evidence packages proportional to biologics’ complexity prior to approval, and different substitution policies.
In other words, more robust data — not less — is required, particularly if biosimilars are to be deemed “interchangeable,” meaning substitutable not only by physicians familiar with their patients’ needs, but by insurers and pharmacy benefit managers.
Yet the FDA has announced it intends to “genericize” biosimilars, with Commissioner Marty Makary himself conflating the two different classes in public statements. This shift has alarmed physicians and patient groups who warn that permitting insurers to switch patients en masse to biosimilars as if they were generics (i.e.; without the supporting data currently required) risks destabilizing treatment for the millions of patients who depend on biologic therapies. There is near-unanimity on the subject among U.S. physians: only 11% of U.S. physicians support treating biosimilars like generics; 88% support keeping the current FDA approval criteria. Yet this highly controversial policy is at risk of soon becoming federal law, through codifying legislation tucked into the administration’s broader health package.
By contrast, in Europe (widely acknowledged as the world leader in biosimilar development and commercialization) most national regulators prohibit generic-style “automatic” substitution of biosimilars by anyone other than the physician. European physicians strongly oppose this practice in their countries, and their U.S. counterparts strongly oppose it here.
And just as troubling, these moves have frequently been accompanied by an unusually high number of senior staff resignations and forced departures. For those who have not served in the Executive Branch, it is vital to understand this is not just routine staffing churn. It is highly unusual to see so many scientific experts leave at the same time, particularly when many cite professional disagreements, internal discord, or policy direction as contributing factors.
It is important here to acknowledge that the current Administration, both through other agencies overseen by HHS and in its work with Congress, has pursued several health initiatives to control medication costs that do not undermine public health or confidence. These include achieving long-sought PBM and insurance reforms, promoting discounted sales of medicines to consumers, and working to rebalance the global economic burden of drug research and development too often borne disproportionately by Americans.
Individual policy missteps by the FDA may harm public health. Yet the cumulative reputational damage to the agency, to the administration’s health agenda and to public confidence will likely be greater than the sum of its parts. In his confirmation hearing, Commissioner Makary stated his goal is “to ensure that the FDA holds to the gold standard of trusted science, transparency, and common sense to rebuild public trust and advance health and safety in the United States and worldwide.” Such trust, we have seen, is hard-won and easily lost. If the FDA is to do so, it must restore coherence, stability, and scientific consistency to its decision-making.
