What a new genetic therapy should teach us about biomedical ‘progress’

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The therapy, Casgevy, uses gene-editing technology to treat people with sickle cell disease — an inherited blood disorder that afflicts an estimated 100,000 Americans, primarily of African descent. The eye-popping price tag? $2.2 million per person. Meanwhile, 80 percent of the millions who suffer and die of sickle cell disease live in sub-Saharan Africa, where the treatment won’t be accessible for decades due both to its cost and the lack of hospital infrastructure to deliver the complex treatment. Even in the United States, where the therapy is theoretically more accessible, non-White people are more likely to be uninsured or to be insured by Medicaid, which might not cover Casgevy’s exorbitant cost. As happens with many medical technologies when they first emerge, getting treated with Casgevy is likely to be a matter of how rich or poor patients are, whether they have insurance, whether insurance covers the costly treatment — and, foremost, the random chance of where they were born. But it doesn’t have to be this way.

In the United States, we’ve come to accept short-run disparity as a necessary side effect of biomedical progress: The risks and costs of inventing a treatment, running clinical trials and attainting regulatory approval are high, so the prices of new medicines must also be high, if companies are to bring them to market. Yes, when patents expire, competitors arise and prices fall, those drugs eventually can become more accessible. In the meantime, however, the world’s poorest sick people often needlessly suffer and die. With HIV, decades of activism, philanthropic largesse and global support have expanded access to lifesaving antiretrovirals. But treatments are still hard to get in some places. In the intervening years since those drugs first became available to the insured in the United States, millions of people died of AIDS — despite the existence of medicines that could have saved them.

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As more researchers develop genetic therapies, including those that rely on CRISPR’s gene-editing technology, more treatments are poised to command multimillion-dollar price tags — and, in the process, to exacerbate disparities in who suffers from disease or gets cured, who lives or dies. A promising treatment for a disease that primarily afflicts people with less privilege underscores the urgent need to rethink how innovative medicines are brought to market. We don’t have to accept the inequity that has defined the path of past biomedical progress. And we shouldn’t.

One of the key drivers of high costs for genetic therapies is the cost of capital, says Melinda Kliegman, public impact director of the Innovative Genomics Institute, a California-based academic group founded by Jennifer Doudna, one of the two scientists who won the Nobel Prize in chemistry in 2020 for developing CRISPR technology. Venture capitalists who invest in therapies typically look........

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