If a drug maker tested an asthma treatment in children and found it significantly reduced attacks compared with a placebo, this would be considered good news, yes?
But what if the children enrolled in the clinical trial were almost entirely white, even though asthma disproportionately affects Black and Puerto Rican children?
This would dampen my enthusiasm, to say the least. And it would make me wonder why a company would fail to study enough of the children who are most in need.
advertisement Unfortunately, this is not a hypothetical situation.
Two pharmaceutical companies — Sanofi and Regeneron Pharmaceuticals — just published the results of a study of their blockbuster Dupixent medicine for treating children between ages 6 and 11 with moderate to serious uncontrolled asthma.
advertisement The results, published in the New England Journal of Medicine, were impressive: Among the 408 children in the study, there was a 65% average drop in severe asthma attacks over one year versus a placebo. And more children who were given Dupixent reported improved asthma control after six months than in the placebo group.
This convinced the Food and Drug Administration to endorse the drug for treating these youngsters, a move that will expand sales. Last year, Dupixent, which is already approved to treat atopic dermatitis as well as asthma in adults, notched U.S. revenue of $3.2 billion. The authors of an accompanying editorial noted the estimated out-of-pocket cost for the drug can be as high as $45,600 per year.
Of course, this is good news for the drug makers and their investors, but not necessarily for kids in underrepresented racial groups. Why? Nearly 90% of those enrolled in the study were white. (See page 43).
To be blunt, there is a yawning disconnect here. Black children in the U.S. are three times as likely to have asthma compared to white children. And the lifetime prevalence of childhood asthma is highest among Puerto Rican children at nearly 24%, and is 18% among Black children. But the prevalence is lowest among white children at 9.5%, according to the Asthma and Allergy Foundation of America. (See page 38).
There is a reason for these stark differences. As the foundation noted in a lengthy report last year called ‘Asthma Disparities in America,’ ‘racial and ethnic differences in asthma frequency, illness and death are highly connected with poverty, city air quality, indoor allergens, not enough patient education and poor health care.’
Yet the lack of diversity in this multinational trial fails to tell us the extent to which Dupixent may actually help Black and Puerto Rican kids. Sure, some children may benefit, but should we — and can we — generalize the results?
This is especially confounding when the Dupixent study also identified biomarkers for taking a precision medicine approach to treating asthma.
This is hardly surprising, though.
As the accompanying editorial noted, a recent study in the Journal of Allergy and Clinical Immunology found that drugs such as Dupixent may not be suitable for children 12 years and older depending upon their racial or ethnic group — and called for more research to provide equitable treatment for children of color with asthma.
Sadly, such a homogenous clinical trial pool is hardly unusual. Black individuals comprise 13.4% of the U.S. population, but only 5% of those participating in clinical trials, while Hispanic or Latino individuals consist of 18.1% of the population and just 1% of those studied in clinical trials, according to Clinical Research Pathways, which promotes diversity in clinical research.
So what do the companies have to say for themselves?
Not very much, as it turns out.
Sanofi, which was the trial sponsor, maintained both drug makers are ‘committed to increasing diversity in our clinical trials. We continue to make ongoing efforts to further integrate diversity into the design and placement of our clinical trials to ensure that they reflect the real-world, make up of patients living with the diseases being studied, and that we serve all patients in need of our medicines.’
The company also noted that the study population ‘generally reflected the demographic make-up’ of the trial sites. And of the 408 patients in the study, 54 were evaluated in U.S. sites and of those patients, 30% were African American and 30% were Hispanic.
In other words, the drug makers are trying to argue that nearly one-third of the children studied in the U.S. were, in fact, Black or Hispanic. But this is a sleight of hand, because the overall math tells us that just 8% of the total trial participants belonged to these groups.
Statistically significant results aside, a key takeaway from this study is very disappointing.
A lack of diversity in clinical trials is certainly not a new concern, but this latest episode only underscores the need to address the problem immediately. After all, the protocol for this trial was finalized in October 2019, which belies the insistence that the companies are committed to greater diversity.
This study should be a warning light that the pharmaceutical industry can do better.
Until that happens, no one can breathe easier.
https://www.statnews.com/2021/12/08/asthma-diversity-sanofi-regeneron-dupixent/
