Gene Therapies To Effectively End Sickle-Cell Are Nearly Here -- But Who Will Get Them, And How Will Our Health System Pay For Them? Questions Abound.

To be sure, this is a watershed moment, after centuries of early-deaths, and more recently, life-long disability -- largely inside the communities of Black African origin, in the US, UK, Europe and elsewhere. . . in addition to Africa itself. This is truly remarkable news. We could see sickle-cell relegated to the dust bin of history the way polio was, for over two thirds of a century. [But that's a separate story, of emerging anti-vaccine non-scientific thought, in the US and EU, for another day.]

The real problem though, lies -- as with all genetic therapies -- in the daunting cost to be "cured": best estimates put the cost of cure, including a year or more out of commission. . . at over a million dollars per patient. In the US, that still makes sense, because the costs of a life-time dealing with the effects of (uncured) sickle-cell, greatly exceed the therapy's likely costs.

But in the developing world, there is not likely to be any way to incent any provider to deliver it, unlike the very cheap COVID-19 vaccines, by comparison. So it is likely that sickle-cell will continue to be scourge, outside North America and Western Europe. Here is a very thoughtful "Science Tuesday" story on it all, in The New York Times, this morning -- and a bit:

. . .“This is what science has been working toward for 50 or 60 years,” said Dr. Lewis Hsu, chief medical officer at the Sickle Cell Disease Association of America and director of the pediatric sickle cell program at the University of Illinois at Chicago.

Sickle cell leads to a steady drip of costs over a lifetime, affecting caregivers as well as patients and often limiting the incomes of both because of time lost from work or an inability to work.

One report examined what private insurance companies paid and found that the lifetime costs of sickle cell were $1.7 million, including $44,000 that patients paid out of pocket. . . .

Treatment starts with intense chemotherapy in a hospital to wipe the bone marrow clean, leaving space for genetically modified red blood cells that result from the treatment. Patients then spend about a month in the hospital waiting for the modified cells to grow. After they are discharged, the patients are immunocompromised for about six months while their immune systems recover.

Insurers are likely to pay for the part of the treatment that involves using a disabled virus to deliver the therapy. But whether they will pay for most of the chemo, hospitalization and other costs is uncertain.

Patients worry about bills from hospitals and doctors. Even if it is mostly covered, co-payments can be an issue.

Will wealthier people get the treatment while others look on longingly, patients ask?. . . .

The achievement of miracle cures -- and, at "miracle prices". . . will increasingly dominate drug delivery debates in the decades to come. Of that, we may all be certain. Onward, but smiling, just the same -- at the progress we (as a species) have made.

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