Innovation Politics at the FDA

The relationship between innovation and the Food and Drug Administration (FDA) has become a central topic of concern for a whole host of parties: academics, members of the National Academies of Sciences, Engineering, and Medicine, and several former FDA commissioners. Industry typically defines innovation by the raw number of drugs developed in a year, while many scholars (ourselves included) focus on patient value — how much benefit a drug produces relative to its harms, whether a new drug is first-in-class or a follow-on drug. 

Which definition one chooses matters a great deal. While many new drugs are developed each year, scholars have raised serious concerns about the drug innovation pipeline in the U.S., pointing to an underdevelopment of many drugs vital to public health including antibiotics, vaccines, and cancer preventives, not to mention threats to the development of critical information about new drugs given many drugs are approved on the basis of insufficient studies. The lack of development in these critical areas is all the more surprising considering Congress and the FDA have enacted numerous policy changes aimed at speeding pharmaceutical innovation. Could our obsession with innovation actually be undermining innovation?

Consider cancer. Cancer drug development accounts for 60 percent of FDA accelerated approvals (which is a less demanding pathway for approval). PhRMA, the trade group that represents the pharmaceutical industry, and individual pharmaceutical companies like Eli Lilly and Johnson & Johnson, extol accelerated approval’s advancement of innovation. Yet most new cancer drugs approved in the U.S. do not deliver substantial clinically meaningful benefit. The average new cancer drug extends life by just two to three months, at an average list price at launch of $283,000. Therefore it seems clear: Cancer is the biggest area of drug development due to weaker regulatory standards and generous reimbursement at the state and federal level. So, we have investment, but are we really achieving innovation? 

Beyond cancer, the FDA has approved drugs that turned out to be harmful for many patients including Aduhelm (for Alzheimer’s disease), Leqembi (also for Alzheimer’s disease), and Makena (to reduce the risk of preterm birth). Each of these drugs was approved pursuant to the FDA’s accelerated approval pathway. Researchers have likewise raised concerns about other pathways.

We do not mean to impugn all areas of FDA drug review. In some contexts, FDA review is quite robust and effective. In other areas, however, regulatory standards have been significantly lowered, ostensibly to facilitate innovation. This mottled regulatory approach is not just variance or tailoring to different disease contexts; it is actually changing the drug development landscape by incentivizing companies to flock to areas in which FDA regulation has become more porous.

These innovation failures are inherent aspects of a market that centers profit-driven pharmaceutical companies, as considerations of marketability dominate discussions of drug development. This market structure is the result of policy decisions made over decades to center private pharmaceutical companies based on the belief that private corporations are the primary drivers of innovation. It is this belief that has, for instance, led Congress to enact incentives for private pharmaceutical companies to develop certain drugs rather than investing in a public drug development pipeline. Pharmaceutical companies have used their significant power to push for greater use of expedited pathways, lower standards for new drugs, fewer restrictions on contact between industry, physicians, and payers, and greater industry participation during agency drug review.

While industry-driven actions can bear advantages, overall, the pharmaceutical market’s structure reflects a neoliberalvision of the economy in which economic interests are prioritized over political and democratic values. The shift to neoliberalism in the pharmaceutical market is just one facet of a broader embrace of neoliberalism over the preceding decades. Neoliberal advocates cast the market as a value-neutral institution capable of achieving the highest social good, whereas the state was portrayed as subject to capture by special interest groups, or as a barrier to innovation. 

FDA experts have argued, for years now, that FDA regulation is pro-innovation: It incentivizes the production of information about new and existing drugs, ensuring the financial rewards of entering the market is attached to true advances. So, then, why do some believe FDA is anti-innovation? Opposing understandings of innovation highlight a dialogue in which political actors aim to shape popular understandings of policy — or schemas — to achieve their ends, whether that be profit or public health. All this points to the importance of defining innovation so that, hopefully, it will be less of a political tool, and more of a useful point of discussion in the future of FDA. 

Challenging the notion that innovation requires less regulation will be difficult on two fronts. First, public health experts and scholars pushing for a greater focus on patient value face an uphill battle as the government is most responsive to economically powerful interests. Second, given the central role pharmaceutical companies play in the drug development pipeline, challenging the hegemony of these companies can be portrayed as anti-patient, whereas continuing to facilitate pharmaceutical profitability can be seen as vital for patient health. But the solution to each of these two challenges may be the same: forming a coalition of public health experts, scholars, advocates, and patients that is capable of challenging the narratives of the pharmaceutical industry in innovation policy. 

This post draws from ideas in a work-in-progress article, the Legal Political Economy of the FDA, forthcoming in 2027.