Biology, Not Politics
A Deleted FDA Guidance Will Harm Red and Blue
The folks at pharmaceutical companies who set up clinical trials have a big incentive to keep it simple. The more alike everyone is in a drug trial, the more similar the data will be, and the smaller the number of patients required to prove a new drug is safe and effective. But here's the problem: If you only test a new drug on one group (say, young white males), you've only proven the drug works for people like the ones in the trial.
This can have consequences that are not ideal. In the not-too-distant past, for example, artificial heart valves that were developed with only men in the clinical trials did not fit in women’s chests. And historically, many clinical trials to treat conditions that primarily affect older adults (such as cancer) excluded patients over 65, despite the fact that those of us who are older metabolize drugs more slowly and often need lower dosages. FDA is charged with protecting the public health and eventually responded to the high rates of adverse events in patients who were not young white males.
FDA Guidance Documents tell industry what the expectations are at the agency. They are first issued as drafts, then go through a lengthy revision process including comments from industry and consumers before they become final. In 2022, when I was working on clinical protocols for new cancer treatments, FDA published a final guidance called "Inclusion of Older Adults in Cancer Clinical Trials." Up until that time, cancer trials often excluded persons over 65. Finalizing that guidance was an important step forward in protecting public health, and fortunately that guidance is still on the books.
Two years earlier, FDA had published a draft guidance that said clinical trials of all drugs intended for use by older adults should include them in clinical trials. It was an important next step, and this guidance was in review and on its way to becoming final. But this guidance was cancelled this month, without public review or discussion, and is only available on the Wayback Machine. This Guidance also called for inclusion of members of other populations such as women and people of color. The name of this guidance was "Enhancing the Diversity of Clinical Trial Populations." Yes, there’s that word: Diversity.
Elimination of this Guidance Document means that folks at pharmaceutical companies who design clinical trials can return to keeping their lives simple by choosing trial participants who are similar to one another. They can, if they wish, select only young white males for trials (unless they are testing a cancer drug, in which case they must include older persons, or at least older males).
Getting rid of this one Guidance Document can be expected to adversely affect public health over time, as new products are approved based on an unrepresentative subgroup. Pointing this out is not political, it is biological. When clinical trials exclude older persons, or women, or children, or people of color, new drugs may have disproportionate negative effects on people in those groups regardless of whether patients live in red or blue states.
This Guidance is one of the thousands of documents that was removed from US government websites in February without public notice or discussion. Over time, this one change alone will damage public health if it is not reversed. The combined effect of removing thousands of documents is hard to predict.
Pointing this out is not politics, though it does involve public policy. The same document that inspired this change calls for other changes that would affect older persons regardless of how, or whether, we voted. These include making Medicare Advantage the default option, allowing removal of Medicaid coverage for nursing home care, and ending the federal government's ability to negotiate drug prices with pharmaceutical companies.
FDA Guidance Documents are established with care, with attention to the opinions of those affected, and based on evidence such as rates of adverse events. The elimination of this Guidance did not undergo review and seems to ignore the potential consequences. It was driven by politics, not biology.