When ‘follow the science’ meets data nationalism

Early last year, an FDA advisory committee rejected a new lung cancer drug. The reason? They weren’t too keen on foreign clinical data. 

Later in the year, the developer of a potential variant-proof COVID-19 treatment that might even be effective against other viruses faced the same problem. The FDA rejected authorization, partly because none of the nearly 2,000 clinical trial patients were treated in the U.S.

The FDA’s increasing tendency to follow data only up to the water’s edge, a kind of “data nationalism,” may harm American health and future pandemic preparedness. It causes delays in drug approvals, increases the cost of drug development, and denies patients access to potentially life-saving treatments.

The agency’s intuition is not altogether inexplicable: the agency charged with evaluating the safety and efficacy of medicines available in the U.S. wants data reflecting the U.S. population, with all its diversity. But in the first instance, unless there’s a basis to believe genetic or ethnic differentiation determines the actual treatment pathology, pursuing this otherwise desirable aim actually harms what should be the ultimate criterion: the welfare of patients. 

Practical limitations should make foreign clinical data not only acceptable but desirable. Many disease incidence rates are higher in other countries, making robust clinical trial enrollment more feasible. For example, the rejected lung cancer drug relied on data from China, which has higher incidence and mortality rates than the U.S. because of its smoking patterns, among other factors.

Moreover, in cases where a drug is already approved outside the U.S., clinical data is more readily available. 

Already, in response to the FDA’s decision, at least two drug developers have withdrawn or significantly delayed their plans to seek approval of their similar lung cancer treatments using foreign data. 

Now we’re seeing imminent trials abroad for possible cancer vaccines. If successful, how many American patients will lose out waiting for an unnecessary reproduction of those efforts?

While part of the FDA’s goal is to increase incentives to invest in clinical trial infrastructure and enrollment in the U.S., before the pandemic, some 40 percent of cancer trials failed to enroll a sufficient number of participants. This policy only exacerbates the issue.

The COVID-19 example is even more tragic. The pandemic has highlighted the need for speed and innovation in drug development. However, the FDA’s data nationalism approach to clinical trials risks slowing down the development of effective treatments and vaccines. If the FDA has concerns about data quality and integrity, it should take a more proactive approach to addressing them. This could include working with international partners to establish standards for data collection and analysis, optimizing and increasing resources for international inspections, addressing guidance to clinical trial Institutional Review Boards, and providing a scientific basis to understand when particular patient characteristics are likely to play a meaningful role in medical effectiveness.

The FDA’s insistence on domestic clinical trials puts Americans at risk of losing out on potentially life-saving treatments, especially in the case of future pandemics. As a prominent epidemiologist recently wrote, “Science has been moving at a lightning speed.” But recent history has shown us that a pandemic can move even faster. We should be open to the collection of foreign data because hopefully the development of a vaccine or therapeutic has begun before the disease breaches our shores. There is no reason to wait when we can get ahead of the problem.

The FDA should prioritize patient health over data nationalism, following the science wherever it leads and looking to data from whence it comes. Innovation is a global good, and we only hurt ourselves when we insist otherwise. After all, what good is something like a cancer moonshot that only flies domestic?

Satya Thallam is a Senior Fellow at Lincoln Network. He is a former biotechnology executive, and a former senior policy advisor at the White House and in Congress.