The key to lowering gene therapy costs – and getting treatments to where they are most needed – could be making them in middle- low-income countries, argue the authors of a recent commentary in Nature.
Here’s something you may not have heard about gene therapy, the emerging and very costly field of medicine in which doctors bioengineer a gene and release it into the body to essentially replace a faulty original that is causing a genetic disease: The cost is,in fact, so great that four approved gene therapies have already been withdrawn from the market because so few people or healthcare systems can afford them.
The authors of a commentary published July 17 in Nature wonder whether the same thing could happen to two long-awaited gene therapies for sickle-cell anemia that were approved by various countries late last year: Casgevy ((exagamglogene autotemcel) is priced at $2.2 million, and Lyfgenia (lovotibeglogene autotemcel) at $3.1 million.
This devastatingly striking possibility, however unlikely, is not actually the point of the article.It merely draws you in.
Co-authors Evelyn Mwesigwa Harlow, a sickle cell program officer at the Uganda Ministry of Health in Kampala, and Jennifer E. Adair, Ph.D., an associate professor of cell and gene therapy at the Fred Hutchinson Cancer and co-founder of the Global Gene Therapy Initiative (GTTI), of which Harlow is a founding member, have a much more sweeping and important case to make.
GTTI’s mission, according to its website, is fast-tracking gene therapy delivery and access and advocating for the best available treatment for HIV and sickle cell patients.
In their commentary, Harlow and Adair argue that the best way to lower costs and increase access to gene therapies is to produce them in the middle- and lower-income countries whose populations contain the overwhelming majority of patients who need these treatments. Doing so, they say, could even end up lowering prices in wealthy natio.
Focusing mainly on sickle-cell disease — one salient example to illustrate a common theme — they build a point-by-point case for shifting gene therapy development elsewhere:
All of this, Harlow and Adair write, adds up to a perception that the therapies are “less cost-effective and more expensive to produce than they would be if their development, production, marketing and uptake shifted to countries where the relevant diseases are most prevalent.”
The capacity of low- and middle-income countries to pursue research and development is much greater than many people in wealthier countries might assume, they say.
A very high hurdle, of course, would be negotiating agreements around intellectual property, although here, too, they offer various reasons for why and how it might be possible.
If the costs for gene therapy can be brought down from the stratosphere to merely high, they say, parents will do anything they can to save the lives of their children. They point to clinics in Nigeria that have been providing bone-marrow transplants to Africans to treat sickle-cell disease for more than a decade. Hundreds of people in India also have received bone-marrow transplants to cure the disorder.
“In most cases, recipients and their relatives have crowdsourced the $25,000-$50,000 needed per treatment,” they write.
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